Agenda

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Please arrive 24 hours before your presentation start time to upload or check your presentation. 

Speakers:
Jacqueline Hugtenburg
Petra Denig
Jessie Franklin
Julie Lauffenburger
Robert Gross

Course Aim

Adherence to medication is indispensable for achieving clinical goals. Non-adherence is a complex and multifactorial problem leading to poor clinical outcomes, increased morbidity and death rates, and unnecessary healthcare costs. Assessing (non-)adherence is challenging and both subjective and objective measures are applied. This course is aimed at introducing students to the various constructs of adherence behaviour and techniques to evaluate (non-)adherence and data analysis.

Requisites Statement

Entry level.

Course Objectives

     1. To understand the different constructs of adherence behaviour and techniques to assess them.
     2. To describe strengths and limitations of different techniques with respect to validity and implementation.
     3. To select the assessment technique(s) most appropriate for the research objective.
     4. To understand issues linked to the use of (non-)adherence and biomedical outcomes and (non-) adherence itself as an outcome for intervention studies.

Syllabus Outline

1. Principles of adherence.
Students will be introduced to the definitions and taxonomy of adherence to medication, as well as to the various types and causes of non-adherence. Subjective and objective measures used to quantify (non-)adherence, and challenges in the analysis of outcomes will be presented. How adherence research relates to pharmacoepidemiology will also be addressed.

2. Subjective measurements.
Students will be introduced to the use of self-report techniques, such as structured interviews and questionnaires, used in adherence research. A variety of common questionnaires and what they assess will be discussed. An example of analysis of data using techniques from pharmacoepidemiology is presented.

3. Objective measurements: prescription and refill databases.
Students will be introduced to the use of objective measurements based on the use of prescription and refill databases. Techniques and considerations for analysing data taking the definitions and constructs of adherence (initiation, implementation, persistence) into account will be presented.

4. Objective measurements: electronic devices.
Students will be introduced to the use of electronic devices and new developments for measuring adherence.

4. Measurement and modelling techniques.
Examples showing calculations and decisions to make using medication possession-based adherence measures (new users, prevalent users, which medications, follow up, metrics) as well as trajectory techniques to summarize adherence will be presented.

5. Interactive case study.
Using an example of a study among diabetes patients using multiple drugs, students are guided through the subsequent steps in making decisions concerning measurements and analyses of data. The influence of a variety of assumptions and decisions is shown, and students are guided through the process of data preparation and transformation needed for adherence calculations.

6. WRAP UP.
Summarizing the main messages.

Speakers:
Doris Auth
Alicia Gilsenan
Elaine Morrato
Meredith Smith

Course Aim

This half-day course presents an overview of risk management principles, with a focus on best practice methods for designing, implementing and evaluating risk minimization programs. The didactic section will feature experts from regulatory, industry, academia and research organizations who will present the regulatory context, methods, and examples of effective risk minimization program design, implementation and evaluation techniques. The interactive workshop section will provide an opportunity for participants to actively apply what they have learned via a case study approach.

Requisites Statement

The target audience is epidemiologists with basic knowledge of risk management, responsible for developing risk management plans and/or designing, implementing or evaluating risk minimization tools and pharmacovigilance and regulatory professionals who are involved with reviewing, and negotiating risk management plans and their assessments. Prior completion of previous ICPE Introduction to Risk Management Course helpful but not mandatory. Supplementary pre-course materials will be available.

Course Objectives

     1. To describe the current regulatory requirements for risk minimization programs in the United States and Europe;
     2. To present concepts from implementation science and state-of-the-art pragmatic approaches to improve risk minimization program design and implementation;
     3. To provide an overview of research designs and methods to use in evaluating risk minimization program effectiveness;
     4. To provide an opportunity for applied learning in the design and evaluation of risk minimization measures via small-group case study discussion.

Syllabus Outline

Pre-conference readings – to be shared on ISPE on-line community exchange:
• Regulatory guidance statements and resources (e.g., FDA, EMA)
• 2019 case: isotretinoin (back-ground information on indication, teratogenic risk, and setting of use)
Conference syllabus:
1. Overview: The BRACE life-cycle, course learning objectives and case introduction. (Lead: Elaine)

2. Regulatory Requirements for Risk Minimization: US, Europe and Rest of World (Lead: Doris)

Table discussion: Compare and contrast regulatory requirements and risk minimization norms; discuss implications for managing risks and how sponsors must navigate differing regulatory requirements as they strive to develop a harmonized, yet locally tailored, global risk management program

Debrief: Implications for the isotretinoin case (Lead: Elaine)

3. Implementation Science Considerations and Pragmatic Approaches for Designing and Implementing Effective Risk Minimization Programs. (Lead: Meredith)

Table discussion: Apply a framework (RE-AIM) to discuss and design a risk management strategy. Discussion will include: identifying design assumptions and implications for program evaluation.

Debrief: Implications for the isotretinoin case (Lead: Elaine)

4. Considerations for Evaluation of the Effectiveness of Risk Minimization Tools (Lead: Alicia)

Table discussion: Identify the knowledge, process, and outcome measures to assess the effectiveness of a risk management program. Identify potential data sources and research designs.

Debrief: Implications for the isotretinoin case (Lead: Elaine)

5. Session close: Final Q&A and Open Discussion/Sharing- Panel and participants

Speakers:
Amalia M. Issa
Gillian Bartlett
Bruce Carleton
Steve Kimmel
Leah Sansbury
Anke-Hilse Maitland van der Zee

Course Aim

This course, sponsored by the Molecular Epidemiology, Biomarkers, and Pharmacogenomics (MEBP) SIG will focus on the essentials every pharmacoepidemiologist should know about real world applications of pharmacogenomics epidemiology including (1) the role of epidemiology in pharmacogenomics (pre-course video); (2) definitions of genetic terms and basic genetic concepts (pre-course video); (3) quality control and analytical procedures required in a pharmacogenetic analysis; (4) step-by-step planning guide to designing a pharmacogenomic analysis; (5) phenotyping considerations and breaking down examples of pharmacogenomic successes; (6) databases available to study pharmacogenomics; (7) designing a companion pharmacogenomic diagnostic in drug development; (8) designing a prospective observational cohort or case-control study of pharmacogenomics; (9) designing a clinical utility trial: a guide with examples; (10) collecting health related quality of life data using an innovative role playing game.

Requisites Statement

Attendees should have basic knowledge of pharmacogenomics, or have watched the pre-­Course Videos (Available Online only)
• The convergence of Pharmacoepidemiology and Precision Medicine [Gillian Bartlett]
• A Primer on Genetic Terms and Basic Concepts [Geoffrey Liu]

Course Objectives

• To understand basic genetic and pharmacogenomic terminology.
• To understand how pharmacogenomics research relates to the current focus on Precision Medicine (aka Personalized Medicine), using real world examples.
• To better understand the study design and methodologic approaches, their strengths and weakness used in pharmacogenomic epidemiology, specifically in relation to clinical applications.
• To understand the role that pharmacogenomics can play in pharmacovigilance and comparative effectiveness research
• To gain knowledge about some available databases with linkage with genomic information
• To understand the benefits and challenges of pharmacogenomics in pharmacoepidemiology in both research and clinical practice applications.

Syllabus Outline

8:00 am--‐8:10am Welcome and Introduction [Amalia Issa]
• Quick introduction of the topics and faculty members. Course logistics will be briefly discussed.

8:15am--‐8:55am Precision Medicine in the Real World Needs Pharmacogenomic Epidemiology [Leah Sansbury]
• Will provide an overview of pharmacogenomics and how pharmacoepidemiologists can contribute meaningfully to precision medicine, including the methodologies, current status, and future direction.s Will discuss real-world examples, and share the experience of working as a pharmacoepidemiologist in industry and with government.

8:55am--‐9:35am What You Need to Consider When Applying Pharmacogenomic Research into Clinical Practice [Bruce Carleton]
• Will present up-to-date state of the science and describe real-world and up to date clinical applications.

9:35am--‐9:50pm Bio Break

9:50am--‐10:30am Pharmacoepidemiologic Methods in Precision Medicine Research: It’s all about the application[Anke-Hilse Maitland van der Zee]
• Will discuss robust pharmacoepidemiologic methods for use in pharmacogenomics research.
• Will introduce different databases with both rich clinical, treatment, outcomes information and genomic information Will compare the pros and cons of different databases to guide future database selection and research.
10:30am--‐11:00am Clinical Utility Research and Pharmacogenomics: Trials and Tribulations [Stephen Kimmel]
• Will share the roles, responsibilities and contributions of a pharmacoepidemiologist in pharmacogenomic clinical trials and provide up-to-date examples of recent and ongoing trials.

11:00am---11:10am Bio Break

11:10am-12:00 pm Innovative Role Playing Game: How pharmacoepidemiologists can collect real world health related quality of life data for pharmacogenomic applications [Gillian Bartlett and Amalia M. Issa]
• This new interactive and fun feature of the course will provide the attendees with the opportunity to play an interactive innovative role-playing game. Through play and post play discussion, attendees will gain important insights about how to collect useful health related quality of life data for pharmacogenomic applications from patient-participants in real world clinical settings. As always, it will be a highly engaging and interactive experience and discussion with ample active audience participation.

12:00 pm---12:10pm Closing Remarks [Gillian Bartlett]
• Final summary of what we covered in the course, and feedback from attendees and ideas for future courses; Course evaluation (available via ISPE)

Speakers:
Vincent Lo Re, III
Caitlin Knox
Tarek Hammad
Sengwee Darren Toh
Almut G. Winterstein

Course Aim

This ICPE Pre-Conference Course is aimed at early stage pharmacoepidemiologists who have been in the field for less than 3 years. Participation will lead to enhancement in research skills (i.e., grant writing, research presentation, and manuscript writing) and professional development (development of mentor-mentee relationships, enhancing creativity/productivity, professional networking) that are crucial to early success in pharmacoepidemiology.

Educational Objectives

• Establish more effective relationships with mentors for research and career development
• Improve delivery of oral research presentations
• Improve writing skills for research manuscripts
• Enhance success in grant writing
• Develop productive strategies for success in the early stages of careers in pharmacoepidemiology

Target Audience

Early stage pharmacoepidemiologists (i.e., undergraduate/graduate students, postdoctoral fellows, and early career pharmacoepidemiologists) who have been conducting active research in the field of pharmacoepidemiology for less than 3 years

Speakers:
Jaclyn Bosco
Gerald Dal pan
Gianluca Trifirò

Course Aim

This workshop will provide participants with information on the following topics:
• Background on biologics, biosimilars and differences from small molecules
• Naming and traceability issues for biologicals and biosimilars
• Interchangeability, switching, and automatic substitution from a regulatory, methodological, and clinical perspective
• Review country-specific regulatory guidelines, particularly focused on post-marketing monitoring, pharmacovigilance, and risk management requirements for biologics and biosimilars
• Conducting observational studies of biologics and biosimilars, including primary data collection and secondary use of data sources for evaluating patterns of use as well as comparative safety and effectiveness of biologics and biosimilars
• Special emphasis will be given to the methodological issues concerning post-marketing assessment of the interchangeability of biologics and corresponding biosimilars

Requisites Statement

Entry level. Fundamental understanding of pharmacoepidemiology required, but biologic and biosimilars topics will start from the basics.

Course Objectives

     1. To gain an understanding of biologics and biosimilars
     2. To understand evolving regulatory landscape for biologicals, including biosimilars
     3. To learn how to design real-world studies for studying comparative benefit-risk profile of biologics and related biosimilars in post-marketing setting to integrate evidence on biosimilarity from premarketing studies

Syllabus Outline

The course will include lectures from the faculty as well as interactive case studies.

1. Biologics and Biosimilars: What, where, and why?
Students will be introduced to biologics and biosimilars terminology, the big picture, complexities in the marketplace that challenge researchers. Some recommended tips regarding planning pharmacoepidemiologic research studies will also be described.

2. Regulatory requirements and considerations for biologicals and biosimilars
This presentation will discuss the regulatory requirements for biologicals, including biosimilars. Specific emphasis will be placed on the post-marketing requirements and the specific challenges in the design and conduct of post-marketing studies for biologicals, including biosimilars from a regulatory point of view.

3. Methodological considerations for using prospective data and multiple data sources to generate evidence
This presentation will discuss stakeholder needs to generate fit-for-purpose evidence. Students will learn about different study design options and specific considerations when designing a pharmacoepidemiologic study of biologics and biosimilars. The presentation will include examples from published literature.

4. Requirements and potential limitations of observational studies of biosimilars in real world settings through secondary use of healthcare databases.
Overview of observational studies on biologics and biosimilars that have been carried out globally will be given. In particular, potential limitations of different data sources (e.g. claims databases and electronic medical records from various countries (i.e., EU, US, Asia)) in terms of addressing clinically relevant questions concerning biologics and biosimilars will be discussed. Special emphasis will be given on how to study switching practices of biologics/biosimilars and how to generate post-marketing evidence on interchangeability of reference product and corresponding biosimilar. Some examples of database studies will be provided.

Speakers:
Almut Winterstein
Anton Pottegard
Tobias Gerhard

Course Aim

This course will provide participants with a succinct introduction to the basic principles, concepts, and study designs of pharmacoepidemiology. The course includes lectures on (1) cohort studies, (2) case-control studies, as well as (3) confounding and other sources of bias.

Requisites Statement

Entry level.

Course Objectives

     1. Appreciate the basic features of the cohort design and its application in pharmacoepidemiology.

     2. Appreciate the basic features of the case-control design and its application in pharmacoepidemiology.

     3. Recognize the basic types of bias (confounding, selection bias, measurement bias) in pharmacoepidemiological studies. Understand how these biases are introduced and how they can be avoided by design or controlled during analysis.

Syllabus Outline

1. Cohort studies (Almut Winterstein).
Students will be introduced to the basic features of the cohort study design and will appreciate how the design is implemented in pharmacoepidemiological studies.

2. Case-control studies (Anton Pottegard).
Students will be introduced to the basic features of the case-control study design (including self-controlled designs) and will appreciate how the design is implemented in pharmacoepidemiological studies.

3. Bias and confounding (Tobias Gerhard).
Students will be introduced to the three basic types of bias in pharmacoepidemiological studies (confounding, selection bias, and measurement bias). Students will appreciate how these biases are introduced and how they can be avoided by design or controlled during analysis.

Speakers:
Melanie Blank
Cynthia Jones
Rima Izem
Siyan Zhan
Jasmanda Wu, FISPE

Course Description

There are over 7,000 rare diseases which affect more than 350 million people worldwide, only 5% of rare conditions have an FDA approved drug treatment. Over the past years, an increasing number of companies have devoted clinical programs to develop new treatments for patients with rare disorders. This creates the need for rare disease epidemiology, as a sub-specialty within pharmacoepidemiology. This unit is aimed at introducing students to the current regulatory environments for rare disease drug approvals in the US and Europe, the specific methodologic issues arising in rare disease epidemiology, use of electronic medical records and claims databases for rare disease research, as well as practical approaches used to integrate pharmacoepidemiogic methods into rare disease drug development programs.

Requisites Statement

Entry level

Open to ISPE members; members may contact info@pharmacoepi.org if they would like to attend. 

Speakers:
Gillian Bartlett-Esquilant
Bruce Carleton
Andrew Freedman
Geoffrey Liu
Anton Pottegard
Mary Davis
Wei Zhou
Leah Sansbury

Course Aim

This course, co-sponsored by the Molecular Epidemiology, Biomarkers, and Pharmacogenomics and Oncology SIGs, will focus on the essentials every pharmacoepidemiologist should know about study design and real-world applications of cancer personalized medicine including precision medicine, companion diagnostics, biomarkers and the biomarker development pipeline, diagnosis and treatment coding, drug and cancer risk associations and linking databases with genomic information.

Requisites Statement

Entry level. Attendees should have basic knowledge of pharmacogenomics, or have watched the pre-course Videos Available Online only
• The convergence of Pharmacoepidemiology and Precision Medicine [Gillian Bartlett]
• A Primer on Genetic Terms and Basic Concepts [Geoffrey Liu]
• Cancer Terminology and the Science of Cancer Drug Development [Geoffrey Liu]

Course Objectives

To acquire an overview of genomics in cancer research, and how epidemiologists and real-world studies make contributions.
To attain a better understand of methodological issues related to pharmacoepidemiology studies of cancer risk .
To improve knowledge of cancer diagnosis and treatment coding in administrative claims and EHRs.
To understand better target therapy for application in pharmacoepidemiology cancer research designs and oncology drug development, including the use of biomarkers and companion diagnostics.
To understand the benefits and challenges of pharmacoepidemiology study designs in oncology research and their impact on clinical practice applications.

Syllabus Outline

Designing and Analysing Pharmacoepidemiology Studies of Cancer Risk [Anton Pottegard]
• Will provide an overview of general considerations for the planning and conduct of pharmacoepidemiological studies of associations between drug use and cancer development. Will focus on choice of study design and exposure ascertainment, while also covering choice of data sources, ascertainment of cancer outcomes, confounder adjustment and future perspectives in drug-cancer research.

Practical Guide to Cancer Diagnosis and Treatment Coding in Cancer Risk and Treatment Outcome Studies Using Administrative Claims and EHRs [Edgar Simard]
Will share different approaches for coding of cancer diagnosis, treatment and recurrence data in both administrative claims and EHRs. Will discuss the advantages, disadvantages and limitations of different coding approaches.

Designing Cancer Pharmacogenomics Studies of Adverse Events [Bruce Carleton]
• Will provide an overview of germline pharmacogenomics in cancer research, including the history, current status, study design and conduct and future directions. Will share examples of how pharmacogenomics research help cancer patients’ prevention, treatment, survival, and safety.

Current State of Targeted Drug Therapy in Oncology Clinical Practice [Kelly Filipski]
• Will introduce some unique aspects of somatic cancer pharmacogenomics, focusing on somatic biomarkers including mutations, gene or protein expressions, and discovery of new targeted therapies and immunotherapies.

Cancer Pharmacoepidemiology in oncology drug development [Wei Zhou]
• Will introduce the basic concept of oncology drug development, pharmacogenomics and target therapy in oncology, and provide examples of epidemiologist’s contribution to oncology drug development

The expanding roles of cancer pharmacoepidemiologists in Industry, Academia and Government [Leah Sansbury]
• Will share the roles and responsibilities of a cancer pharmacoepidemiologist in industry, government and academia. Will provide real-world examples of what a pharmacoepidemiologist “does” supporting the oncology pipeline in industry.

Precision Medicine in Cancer: Panel Discussion Q and A: [Moderators Gillian Bartlett, Andrew Freedman and Geoffrey Liu]
This is a panel discussion with all course faculty included. This will provide unique opportunities for students to ask questions on the topics included in the course, or any other topics they are interested. The course faculty will also prepare several questions and topics to stimulate the discussions and may debate from different aspects and views.

Speakers
Joshua Gagne
Shirley Wang

Course Aim

There is growing interesting in drug repurposing, which involves finding new uses for old drugs. In addition to various clinical and pharmacological approaches, the methods of comparative effectiveness research (CER) can play an important role in evaluating whether marketed medications may be beneficial for other indications before embarking on large and expensive randomized trials. This year’s course will start with an overview of the role of CER in drug repurposing, describe CER methods for generating evidence to evaluate the benefits of existing medications for new indications, and will discuss the promises and challenges of CER in this space. Multiple case studies will be used to facilitate an interactive group discussion and will address how observational, real-world data can be used to evaluate medications for repurposing.

Requisites Statement
Entry level.

Course Objectives

1. Recognize growing opportunities for comparative effectiveness evidence in the context of drug repurposing
2. Understand biases that commonly occur in comparative effectiveness evaluations and strategies for overcoming these biases
3. Describe challenges to using comparative effectiveness to understand whether approved medical products are effective for other conditions

Syllabus Outline

1. An overview of drug repurposing and the potential role for CER
This lecture will introduce the concept of drug repurposing and provide a high-level overview of various approaches to identifying and evaluating candidate drugs for repurposing. Participants will be provided a high-level overview of CER and how it can be used together with other approaches (e.g., network-based analyses) for repurposing.

2. Introduction to case studies
Course faculty will introduce the two cases studies that will be discussed later in the course (hydroxychloroquine and coronary artery disease; etancercept and Alzheimer’s disease. Participants will be asked to think about the case studies during the subsequent lecture.

3. Frequently observed biases and investigator errors that are avoidable in CER
This lecture will provide an overview of pharmacoepidemiologic approaches to CER. Special attention will be given to challenges and solutions facing the analysis of observational real-world data.

4. Case study 1 and interactive discussion: hydroxychloroquine and coronary artery disease
Course faculty will facilitate an interactive discussion in which participants will be able to apply the learnings of the previous lectures in the context of a real-world case study.

5. Case study 2 and interactive discussion: etancercept and Alzheimer’s disease
Course faculty will facilitate an interactive discussion in which participants will be able to apply the learnings of the previous lectures in the context of a hypothetical real-world case study.

6. Discussion about future directions for CER in drug repurposing
Course faculty will facilitate an interactive discussion about the future of CER in drug repurposing.

Speakers:
Chih-Ying Pratt
Christian Hampp
Jessical Jalbert
Nicole Pratt
Chintan Dave

Course Aim

Pharmacoepidemiologic studies that involve the use of electronic healthcare data have complex and unique characteristics that must be taken into consideration when planning and conducting these types of studies. This highly interactive course will engage participants in the following topics:
     1. Pre-specifying study components of a protocol, including design, analysis, and reporting of the study, along with a science-based rationale for the choices pertaining to these components; and
     2. Translating elements of the study protocol into a statistical analytic program.

Requisites Statement

Intermediate level course. An understanding of basic statistical programming language is required.
The course content is complementary to the “Introduction to Pharmacoepidemiology” class. Prior attendance of the “Introduction to Pharmacoepidemiology” class is encouraged, although not required

Course Objectives

• To introduce a framework for translating study questions into a clear and detailed protocol supported by a rational thought process that is tailored to available data for pharmacoepidemiologic studies using electronic healthcare data
• To develop skills in health care data manipulation to support the conduct of a statistical analysis
• To translate a pharmacoepidemiologic study protocol into a statistical analytic program.

Syllabus Outline

The first part of the class will start with the presentation of an example protocol for a hypothetical case. Using this example, we will walk through a study protocol with substantial inadequacies. A short lecture will follow to address how to write a clear and detailed protocol. Students will form groups to discuss and present their suggestions and approaches to improve the protocol example. The second part of the course includes hands-on session for programing using the previously discussed example protocol.

Speakers:
Jennifer Christian
Priscilla Velentgas
Cynthia Girman
Anne Trontell

Course Aim

Pragmatic randomized clinical trials (pRCTs) require the expertise of pharmacoepidemiologists who understand considerations and methodologic challenges that arise with more “pragmatic” RCTs compared with traditional “explanatory” RCTs. This course will highlight issues such as real-world site and patient identification, enrollment and retention, ethical review issues, approach to randomization, selection bias, and defining the appropriate comparator. In addition, choice of study design including cluster randomization, step wedge design or individual randomization, and associated analytic considerations will be highlighted. Randomizing patients to “standard of care (SOC)”, accounting for changes in therapies over time, and other methodological considerations will also be discussed. Operational challenges and lessons learned from conducting pRCTs will be shared along with case examples.

Requisites Statement

Entry level, though familiarity with basic principles of observational and/or experimental study design and statistical methods is recommended. Recommended pre-course reading material will also be provided.

Course Objectives

• To understand key design considerations for pRCTs and drivers of design choices (including cluster randomized vs. patient level randomization)
• To understand statistical issues for the analyses of pRCTs
• To understand considerations pertaining to primary data collection vs use of secondary data sources
• To identify and address operational considerations and challenges in implementing pRCTs
• To gain insight into current and recent pragmatic trial case examples with regard to their methods, implementation, operational challenges, and intended use as real-world-evidence in support of pre- or post-market treatments

Syllabus Outline

1. Overview and Introduction to Pragmatic Trials-J Christian
To introduce attendees to the rationale and context for conducting pRCTs, a high level overview, explanation of the PRECIS criteria, pivotal studies and articles, and examples of impact with different stakeholders will be discussed.

2. Study designs for pragmatic trials – P Velentgas
The following topics related to study designs and outcome selection for pragmatic trials will be addressed: patient-level and cluster randomized designs, cross-over trials, blinding, inclusion and exclusion criteria, choice of comparators, data collection and outcome selection, with illustrative case examples.

3. Statistical analyses for pragmatic trials – C Girman
Analytic methods related to pragmatic trials will be reviewed, including different considerations related to randomization, sample size estimation for cluster randomized and stepped wedge designs, appropriate analysis methods, and non-inferiority vs. superiority hypothesis testing.

4. Recent case examples of pragmatic trials funded by PCORI – A Trontell
Dr. Trontell will provide an overview of some of the recently completed PCORI-funded pragmatic trials, focusing on unique features of PCORI’s approach and challenges of implementation. Investigator feedback on challenges in pragmatic trials and a patient-centered approach to design of such trials will also be given.

Speaker:
George Gopen, JD, PhD

Course Aim: 
From graduate students to senior practicing pharmacoepidemiologists, our work requires that we communicate in writing. Success in securing funding, sharing research findings, and influencing key decision makers depends on the skill with which we present thoughts to the reader. Those accustomed to reading English prose bring implicit expectations for when and how information will be presented. Violate these unstated expectations and the reader becomes irritated, weary, and struggles to understand the writer’s intended message. The same content presented in alignment with reader expectations allows the reader to focus on the writer’s ideas. As noted in his 1990 publication in American Scientist, “The results are substantive, not merely cosmetic: Improving the quality of writing actually improves the quality of thought.”
Over the last 45 years, Dr Gopen has devoted himself to uncovering these reader expectations and understanding the implications for scientific writing. During that time, he has taught tens of thousands of undergraduates, PhDs, MDs, JDs and MBAs the world over – including many mid-to-senior level clinical scientists. In this workshop, Dr Gopen introduces participants to the rhetorical principles underlying this advanced approach to scientific writing and demonstrates key reader expectations well enough for them to be applied immediately.

Requisites Statement
No required pre-course materials. Designed for all scientific writers from trainees to experienced professionals.

Course Objectives:  
1. Understand the reader’s process of interpretation: to understand how readers go about making sense of words in a sentence.
2. To gain an entirely new perspective on the way the English language functions by seeing it through the eyes of readers.¬¬¬
3. To understand enough individual reader expectations so the participant can make permanent changes to his/her own writing style.

Syllabus Outline
Introduction to the concept of the Reader Expectation Approach
1. Why writing has always been badly taught.
2. The difference in the nature of audience: writing as student versus writing as a professional.
3. Problems that arise when trying to view language from the reader’s perspective.
4. Why the Reader Expectation Approach solves most of these problems.
Discerning the action in an English sentence
1. “Avoid the passive”: bad advice
2. “To make it better, make it shorter”: bad advice
3. The relationship between a sentence’s action and its verb
4. What is right and wrong with “Avoid weak verbs; use strong verbs”
5. The strengths and weaknesses of nominalizations

Agency: How to express it, and how to repress it
Subject-Verb separations: How they function, and why they can cause problems
Whose story is an English sentence telling, according to most readers?
The crucial importance of the backwards link from a sentence to its predecessor
The contents of the “Topic position”: “Whose Story?” + “Backwards Link” = “Context”

The crucial importance of the concept of the “Stress position”
1. Demonstration of the existence of the “Stress position”
2. Grammatical concepts of why colon and semi-colon can create Stress positions, but commas cannot

The flow of thought: Using Topic and Stress positions to ensure the uninterrupted flow of thought in a paragraph
Style: What is it, how to recognize it, and how to improve it.
A method for analysing your own writing style through these Reader Expectation principles
A protocol for applying these principles to all of your future writing

Speakers:
Veronika Wirtz
Lisa Pont
Katja Taxis

Course Aim

This course provides basic theory and practical tips in conducting pharmacoepidemiologic studies when required information is not captured in databases or when large databases are not available. Common study designs used to collect data through observations, medical records or questionnaires will be presented and the strengths and limitations of working without large databases discussed. Hands on experience via a workshop will expose course participants to different study designs and rich discussions on how to answer relevant questions about safety and utilization of medicines in primary and secondary care settings using field studies.

Requisites Statement

Basic knowledge of pharmacoepidemiology required. No mandatory pre-course preparation necessary.

Course Objectives

• To analyze common study designs of pharmacoepidemiologic studies without large databases
• To discuss strengths and limitations of different study designs and data collection methods
• To identify strategies to improve quality and reliability of pharmacoepidemiological studies conducted without large databases

Syllabus Outline

In the first presentation, the students will be introduced to strengths and limitations of different study design and methods of data collections in pharmacoepidemiology. The following presentations highlight examples of research studies that use original data collection to augment the information availability via electronic database. The presentations are followed by a workshop in which students in small group design a study using original data collection to answer to given research question.

1. Katja Taxis, MSc, PhD, Department of Pharmacy, Unit of PharmacoTherapy-Epidemiology & -Economics, University of Groningen, The Netherlands
Common study designs used to collect data through observation, medical records or questionnaires
k.taxis@rug.nl
2. Lisa Pont, BSc, BPharm, PhD, Australian Institute of Health Innovation, Macquarie University, Australia
Field studies to value add in pharmacoepi: focus on efficiency in study design
Lisa.Pont@uts.edu.au
3. Veronika Wirtz, MSc, PhD, Boston University School of Public Health, USA
Course moderator and facilitator
vwirtz@bu.edu

Speakers:
Kevin Haynes
Gillian Hall
Matthew Reynolds
Keith Marsolo
Daniel Beachler
Sudha Raman

Course Aim

Pharmacoepidemiology requires the utilization of database resources to evaluate drug safety. This course will define the different types of data resources available for pharmacoepidemiology and the selection criteria to select the most appropriate resource. The course focuses on the Guidelines for good database selection and use in pharmacoepidemiology research, provides an overview of available resources, addresses the need for outcome validation, and highlights data linkage considerations.

Requisites Statement

Intermediate level course. The course assumes attendees have either taken entry level pharmacoepidemiology courses at ISPE or through didactic course work.

Course Objectives

     1. To review the Guidelines for Good Database Selection and use in Pharmacoepidemiology Research.
     2. To gain an understanding of available pharmacoepidemiology resources
     3. To develop the skills to select an appropriate resource to conduct pharmacoepidemiology research
     4. To examine validation methods for assessing the quality of pharmacoepidemiology data sources
     5. To provide an overview of data linkage of available pharmacoepidemiology resources particularly to patient registry data resources

Syllabus Outline

1. Overview of Database use in Pharmacoepidemiology {30 minutes} Gillian Hall, PhD
Independent Consultant
The presentation will review the ISPE Guidelines for good database selection and use in pharmacoepidemiology research. The focus will be on application of the guidelines in a topically updated portfolio of pharmacoepidemiology studies.

2. Selection of Databases for Pharmacoepidemiology Research {30 minutes} Matthew Reynolds, PhD Vice President, Real World Effectiveness at IQVIA
The presentation will review available resources updated to reflect the dynamic changes to the availability of resources across an international landscape.

3. Database Approaches to Patient Follow-Up for Clinical Trials {30 minutes} Keith Marsolo, PhD
Instructor, Department of Population Health Sciences, Duke University
Facilitated discussion of the capabilities of pharmacoepidemiology databases to collect data for clinical trial investigations for Real World Evidence generation from pragmatic clinical trials.

4. Database Selection Workshop Activity {10 minutes} Kevin Haynes, PharmD, MSCE
An interactive coffee break in which participants will be exposed to several pharmacoepidemiology specific aims and asked to provide potential database resources to address the research questions.

5. Data validation approaches in pharmacoepidemiology {30 minutes} Daniel Beachler, PhD HealthCore, Inc.
An overview of the various approaches available to conduct validation studies. The presentation will focus on the mechanics of medical record validation studies and the impact on bias analyses within pharmacoepidemiology studies.

6. Validation Workshop Activity {15 minutes} Gillian Hall, Daniel Beachler, Kevin Haynes
Participants will work hands on with an on-line tool to determine how to adjust point estimates for outcome misclassification if PPV is known and sensitivity is known or can be assumed.

7. Linking pharmacoepidemiology resources {30 minutes} Sudha Raman, PhD
Assistant Professor, Department of Population Health Sciences, Duke University
The lecture will provide guidance on key aspects of data linkage appropriateness and feasibility necessary to plan useful and sustainable linkages that advance pharmacoepidemiology and patient safety. The presentation will highlight recent advances undertaken across the society.

Please arrive 24 hours before your presentation start time to upload or check your presentation.

Speakers
Cathy Panozzo
Robert T. Chen
Martin Kulldorff
Catherine Cohet
Harry Seifert

Katherine Yih, PhD, MPH:  Harvard Pilgrim Health Care Institute/Harvard Medical School, USA

Course Aim

Vaccines are key tools for global public health.  Increasingly, pharmacoepidemiological studies are providing critical evidence needed for risk-benefit assessment to inform immunization policy, both for mature immunization programs and for low-and middle-income countries (LMIC) for which innovative vaccines against challenging diseases (e.g., dengue, Ebola, malaria, etc.) are under development.  The annual ICPE has had vaccine-specific sessions for >15 years and ISPE has had a Vaccine Special Interest Group (VAXSIG) since 2014.  One of the VAXSIG goals is to offer educational activities on vaccine-related epidemiology, in collaboration with the ISPE Education Committee.  The VAXSIG pre-conference course has been offered since the 2015 ICPE.

Content

This half-day (~4 hours with break) course aims to establish an understanding of some of the ways in which vaccines are monitored for safety after licensure.  The course involves lectures with a combination of presentations and examples, and opportunities for audience participation (questions and discussion).

Requisites Statement

Entry to intermediate level assumes knowledge of basic epidemiologic concepts.  Target audience will be ICPE attendees interested in gaining an understanding of the epidemiology of vaccine safety.

Course Objectives

Upon completing this course, participants will be able to:

• Explain the need for vaccine safety and surveillance studies
• Describe the most common observational vaccine study designs and understand the main study methods and challenges
• Describe in general terms the statistical methods used with the most common observational vaccine study designs, including self-controlled designs
• Understand the key concepts of vaccine safety surveillance in LMIC
• Learn about challenges and solutions for studying vaccine safety during pregnancy
• Understand the unique features of the data-mining method that uses the tree-temporal scan statistic

 Syllabus Outline

Cathy Panozzo, PhD, MPH, Harvard Pilgrim Health Care Institute/Harvard Medical School, USA:  Introduction to the course and VAXSIG (10 minutes)

Robert T. Chen, MD, MA, Brighton Collaboration, a program of Task Force for Global Health, USA:  Introduction to vaccine preventable diseases and vaccine safety (30 minutes)

Martin Kulldorff, PhD, Brigham and Women’s Hospital/Harvard Medical School, USA: Vaccine safety study design overview (40 minutes)

Catherine Cohet, PhD, GSK Vaccines, Belgium:  Vaccine safety study designs for new vaccines in LMIC (40 minutes)

10 min break

Harry Seifert, MD, MSCE, FISPE, GSK Vaccines, USA:  Vaccine safety during pregnancy (40 minutes)

Katherine Yih, PhD, MPH, Harvard Pilgrim Health Care Institute/Harvard Medical School, USA:  Data-mining for vaccine adverse events using the self-controlled tree-temporal scan statistic (40 minutes)

Speakers:
Michele Jonsson Funk
Jennifer Lund
Sudha Raman
Mugdha Gokhale
Til Stürmer

Course Aim 

This half-day interactive course will introduce students to multiple approaches to addressing unmeasured confounders through study design and analysis, the requirements and assumptions of each, and an opportunity to compare these approaches through facilitated discussion of a case-study.

Requisites Statement

Intermediate level: A basic understanding of pharmacoepidemiology study designs, data sources, and confounding are needed for this intermediate-level course.

Course Objectives

     1. Identify common unmeasured covariates in pharmacoepidemiology and the types of bias that can result.
     2. Describe multiple approaches to addressing unmeasured covariates in pharmacoepidemiology through study design and analysis.
     3. Understand the assumptions of each approach, ways of evaluating these assumptions, and when they are likely to be violated.
     4. Compare these methods in a novel setting to identify trade-offs and considerations.

Syllabus Outline

1. Use of study design to control unmeasured confounding
During this group of presentations, Drs Lund, Raman, and Gokhale will introduce three ways in which study design can be used to limit bias due to unmeasured confounding: a) the active comparator new user (ACNU) design, b) self-controlled designs, and c) instrumental variable designs. Faculty will provide foundational knowledge about each approach illustrated through real-world examples in a lecture format with an opportunity for discussion and questions.

2. Analytic approaches to unmeasured confounding
During the second group of presentations, Drs Lund, Stürmer, and Jonsson Funk will introduce three analytic approaches to reduce bias due to unmeasured confounding: a) proxy measures, b) external control for confounding, and c) quantitative bias analysis. Faculty will provide foundational knowledge about each approach illustrated through real-world examples in a lecture format with an opportunity for discussion and questions.

3. Interactive Case Study.
Led by Dr Jonsson Funk, the interactive case study will pose a hypothetical research question and engage students in thinking about the likely unmeasured covariates, considering how each of the approaches might be used in that setting, and weighing the pros/cons of each. This case-study will provide integration across the approaches and give students an opportunity to work through the selection of an appropriate strategy for addressing important unmeasured covariates in light of real-world constraints.

Speakers:
Gillian Caughey
Veronika Wirtz
Lisa Pont
Solveig Sakshaug
Moretn Andersen
Jaden Brandt

Course Aim

This educational session provides an overview of drug utilisation research (DUR) and presents essential methods used. The session includes interaction with participants, question and answer sessions, and discussion during and at the end of each presentation. At the end of the session, the participant will be exposed to:
• Description of the theoretical framework and practical applications of different methods illustrated using selected examples
• Classification systems used in drug utilisation monitoring and research
• Limitations of data sources and methods
• Interpretation of aggregate and individual-based data, variation and change
• Using drug utilisation research for public health and implications for policy decisions

Requisites Statement

Entry level course. This course is targeted at:
     1. New members of the DUR / health services research (HSR) Special Interest Group (SIG) and graduate students new to      DUR/HSR
     2. Those interested in drug utilisation research who need an overview of the area
     3. Those interested in monitoring of medicine use and quality assessment
     4. Those working in health care organisations involved in payment and reimbursement of medicines, medicines policy areas, or planning of health services

Course Objectives

     1. To provide an overview of Drug Utilisation Research within the context of Pharmacoepidemiology, Health Services Research and Public Health
     2. To describe fundamental principles for classifying and quantifying drug use and to provide an understanding of the methodological challenges
     3. To provide the basic knowledge and understanding of epidemiological measures of drug use and study designs based on individual patient data
     4. To describe methodological and policy issues in Drug Utilisation Research

Syllabus Outline

Veronika Wirtz, Associate Professor, Department of Global Health, School of Public Health, Boston University
Welcome to DUR / HSR SIG from DUR Chair

Course Instructors/Presentations
All presentations will include a working practical example of conducting DUR in practice, relevant to each of the topics.

Lisa Pont, Associate Professor, University of Technology Sydney, Australia, University of Groningen, The Netherlands
Introduction to Drug Utilisation Research: Methodological Framework and Skills Needed

Solveig Sakshaug, Senior Advisor, WHO Collaborating Centre for Drug Statistics Methodology, Norwegian Institute of Public Health, Oslo, Norway
The Anatomical Therapeutic Chemical Classification and the Defined Daily Dose Methodology: Classifying and Quantifying Drug Use.

Morten Andersen, Professor of Pharmacotherapy, Department of Drug Design and Pharmacology, University of Copenhagen.

Jaden Brandt, Faculty of Pharmacy, University of Manitoba
Drug Utilisation Research to Inform Drug Policy-A Canadian Perspective

Speakers:
Brian Bateman
Krista Huybrechts
Andrea Margulis
Ken Rothman

Course Aim

In this half day course, faculty will present and discuss selected analytical considerations that are relevant for drug safety research in pregnancy.

The course will consist of four presentations by faculty, with an interactive approach using mobile technology to engage course participants in the discussion. Up to two publications illustrative of each topic will be proposed as pre-reading materials for course registrants a few weeks before the conference. Students interested in participating in a hands-on exercise on their own computer will be asked to download a spreadsheet for probabilistic sensitivity analyses prior to the conference.

The content of this course has changed from previous years. In 2015, the course provided an overview of issues that are particular to drug safety research in pregnancy. In 2016 the course focused on challenges related to exposure measurement, in 2017 the focus was on maternal and neonatal outcomes, and in 2018 the course presented pros and cons of various study designs. This year, the focus will be on selected analytical aspects of relevance to pregnancy safety research.

Requisites Statement

This is an intermediate-level pharmacoepidemiology course. References for a few suggested readings will be distributed among course registrants a few weeks before the conference. While reading ahead of the course is encouraged, it is not a requirement.

Course Objectives

     1. To be better equipped to critically assess the validity of studies on drug safety in pregnancy
     2. To be able to identify useful analytic approaches to address potential threats to the validity of studies on drug safety in pregnancy, and to recognise the circumstances when they are most appropriate.
     3. To gain an appreciation of the problems with statistical significance testing in the context of drug safety in pregnancy, and become familiar with more appropriate ways to interpret study results.

Syllabus Outline 

1. Overview of threats to the validity of studies on drug safety in pregnancy and analytic approaches to address them
In the first lecture, an overview will be provided of common threats to the validity of studies on drug safety in pregnancy. Analytic approaches that can help to address potential misclassification bias, confounding, and selection bias will be presented. The aim of this first lecture is to provide students a general appreciation of the scope of approaches available before delving into specific issues in the subsequent lectures.
Instructor: Brian Bateman, Brigham and Women’s Hospital, Harvard Medical School.

2. Approaches to mitigate unmeasured confounding
In this session, we will discuss approaches to mitigate unmeasured confounding in pharmacoepidemiology pregnancy safety research. The focus will be on those aspects of unmeasured confounding that are specific to pregnancy research (e.g., variables or characteristics that typically remain unmeasured in pregnancy-related research, unique analytic approaches available to address unmeasured confounding). Planned topics for discussion include propensity score calibration, adjustment with data from externals sources, and sibling analysis. The approaches will be illustrated using pregnancy-related examples.
Instructor: Andrea Margulis, RTI Health Solutions

3. The end of significance testing in pregnancy research
In this lecture, students will learn how statistical significance testing degrades quantitative information into a dichotomy based on the p-value, and how this process fosters misinterpretation of results. The problem will be illustrated using examples from the literature on drug safety in pregnancy and other areas. Students will be able to describe the advantages of estimation over significance testing.
Instructor: Ken Rothman, RTI Health Solutions.

4. Quantitative bias analysis in pregnancy research
In this last lecture, quantitative bias analyses to address misclassification of binary variables and its usefulness in perinatal pharmacoepidemiology will be discussed. Attendees will have the opportunity to conduct a hands-on exercise using the spreadsheet developed by Drs. Fox, Lash, and Greenland.
Instructor: Krista Huybrechts, Brigham and Women’s Hospital, Harvard Medical School.

Speakers:
Joshua Gagne
Gianluca Trifirò
Manfred Hauben
Judy Maro

Course Aim

This half-day course will introduce participants to the principles of surveillance in pharmacovigilance with a focus on quantitative aspects and recent advances in the field. The course will cover classical pharmacovigilance signal detection using spontaneous adverse event reports, quantitative signal detection methods for electronic medical record and claims data, and international initiatives, including work in the US FDA’s Sentinel program aimed at using routinely collected electronic healthcare data to conduct signal detection. Real-world examples will be used across the lecture sessions to illustrate and connect different concepts. Participants will engage in interactive discussions about contemporary issues in and challenges facing pharmacovigilance, such as the role of social media data and the use of the same data for both signal detection and subsequent evaluation.

Requisites Statement

Entry level.

Course Objectives

     1. Explain the need for pharmacovigilance and its component activities with a focus on safety surveillance
     2. Describe the potential data sources (e.g., spontaneous reports, claims, electronic medical records, social media data) for safety surveillance in pharmacovigilance
     3. Understand the array of core analytic methods for pharmacovigilance in spontaneous adverse events reports and signal detection and active monitoring in electronic healthcare databases
     4. Recognize the limitations of each data source and approach and how they complement each other
     5. Appreciate recent advances and controversies in quantitative signal detection

Syllabus Outline

1. Classical pharmacovigilance signal detection using spontaneous adverse event reports
Individual case safety reports have been a main source of drug safety information in the post-approval setting. The first session will cover basic concepts of safety surveillance in pharmacovigilance, describe how spontaneous adverse event reports are generated and collected, and introduce quantitative tools for separating potential signals from noise among large collections of adverse event reports.

2. Lessons learned from more than three decades and safety surveillance system efforts
Many efforts have been undertaken to harness the massive amounts of information contained in routinely collected electronic healthcare data to identify signals of potential drug-induced adverse events. This session will describe what has worked and what has not and the principles for safety signal detection that can be gleaned from these lessons.

3. Novel quantitative methods for identifying drug safety signals in electronic healthcare data
With the expanding availability of electronic healthcare data and the increasing role that they are playing in clinical and regulatory decision-making, new quantitative methods have been developed and evaluated to improve the ability to the performance safety signal detection. This session will focus on technical aspects of these new methods.

4. Interactive discussion of contemporary issues in and challenges facing pharmacovigilance and signal detection
Drug safety signal detection faces many challenges. In this interactive session facilitated by course faculty, participants will be asked to consider all facets of these issues and engage in constructive discussion about the role of signal detection in this context.

Speakers:
John Seeger
Jeremy Rassen

Course Aim

Issues of bias and confounding relate to study design and analysis in the setting of non-random treatment assignment where compared subjects might differ substantially with respect to comorbidities. Failing to address a lack of balance in the covariates between treated and comparison groups can produce confounded estimates of treatment effect.

Requisites Statement

This is an intermediate level course.

Background in epidemiology and logistic regression modelling is assumed.

No precourse work is needed.

Course Objectives

• Discuss how propensity scores are useful for observational research;
• Recognize research conditions where propensity scores offer advantages; and
• Explain how propensity scores may be applied in research (restriction, stratification, matching, modeling, and weighting), and the effect of each application on inference.

Syllabus Outline

Faculty will explain how propensity scores can be used to mitigate confounding through standard observational approaches (restriction, stratification, matching, regression, or weighting).

The advantages and disadvantages of standard adjustment relative to propensity score-based methods will be discussed.

Details of propensity score methodology (variable selection, use, and diagnostics) will also be discussed.

Speakers:
Nancy Dreyer
Nabarun Dasgupta
Jeff Curtis
Christina Mack
Kevin Haynes

Course Aim

This course will provide an overview of the methods and applications for studies that use primary data collection for effectiveness and safety research, including regulatory submissions of real-world data for label approvals or expansions. Topics include data collection from clinicians, electronic health records, or directly from patients without use of clinicians, and will address methodological and logistical challenges with a focus on recruitment and retention. Examples will include working with patient communities, data collection through clinicians, call centers, and digital technologies, and integrating electronic health data, with applications in rheumatology, pregnancy and injury research, among others.

Requisites Statement

Entry level

Course Objectives

1) To understand prospective studies including patient registries can be designed and used to support safety, effectiveness and value, including regulatory use
2) To describe the challenges of recruitment and retention; including strategies for optimization
3) To address challenges and benefits of direct data collection from study subjects without intervention of clinicians, including digital data collection and use of call centers
4) To be familiar with stakeholder expectations and the guides to assess quality in observational studies to enhance critical evaluation skills

Syllabus Outline

1. Principles of patient registries and prospective research [Nancy Dreyer ]
Students will be introduced to the purpose and scope of patient registries and other uses of primary data collection for safety and effectiveness, including regulatory use for approval of new medical products and label expansions as well for evidence of clinicians, patients and payers. Stakeholder expectations will be summarized along with guides to good practice.

2. Automated processing of patient-generated data (for non-techies): [Nabarun Dasgupta]
Examples will be provided about how to use patient-generated data from social media, such as searching Twitter and compiling a dataset with a single line of Stata code. In addition to applied examples, ethical considerations and regulatory expectations of doing research using social data will be addressed as will team/skill configurations required for these activities.

3. Using digital patient-power research networks [Jeff Curtis]
The example of a large and successful PCORI-funded patient powered research network (registry) composed of >18,000 people with rheumatoid arthritis, psoriatic arthritis, and other rheumatic and musculoskeletal conditions will be described. Patient reported outcomes and other active and passive patient generated health data are captured using apps and wearable devices. An in-app electronic consent (eConsent) and linkage to other data sources (e.g. administrative claims data) will be described. Emphasis will be placed on patient engagement and retention, with examples of research activities conducted using these data. See www.ArthritisPower.org

4. Using EHR and Call Centers for Data Collection [Christina Mack]
Principles of good practice for data acquisition and EHR curation will be provided and illustrated. Examples of methods and application of patient registries will be provided, showing the opportunities and challenges of patient reporting via centralized call centers, data curation, shown in the context of sports and orthopedic injury research and external comparator study designs.

5. Randomizing treatments in Integrated Delivery Networks [Kevin Haynes]
Pragmatic randomized trials can be designed and conducted within health systems using electronic health records for follow. The on-going ADAPTABLE trial comparing the cardiovascular benefits of high and low-dose aspirin will be presented along with lessons learned.

Ticket Required.
By invitation only; registration required.

Speakers:
Gillian Caughey
Katja Taxis
Lisa Kalisch Ellett
Petra Denig
Yared Santa-Ana-Téllez

Course Aim

This interactive educational program includes presentations, question and answer sessions and a final discussion with the faculty panel. The session will involve participants in the:
     1. Methods for evaluating the effect of clinical or policy interventions
     2. Interpretation of the evaluation data and dissemination of results
     3. Application of principles derived from Health Services and Implementation Research to the design of a clinical intervention
     4. Use of drug utilisation information from administrative health data to improve medicines use
     5. Use of prescribing data and quality measures to assess prescribing

Requisites Statement

All levels of expertise but specifically this course is targeted at:
     1. Members of the DUR / HSR SIG
     2. Graduate students interested in advanced methodologies
     3. Those interested in quality development and implementation research, knowledge transfer, evaluation of interventions, health and medicines policy areas, health funds and third-party payers
     4. Clinical pharmacoepidemiologists, health service planners, clinical guidelines developers, health practitioners

Course Objectives

     1. To gain an advanced understanding of planning, implementation and evaluation requirements for an effective quality use of medicines intervention
     2. To provide an understanding of advanced drug utilisation methods to evaluate complex interventions and appropriateness of prescribing
     3. To develop an understanding of the significance and applications of DUR to translate research findings at the clinical and policy levels of the health system to improve health outcomes

Syllabus Outline

All presentations will include a working practical example of conducting DUR in practice, relevant to each of the topics.

Katja Taxis, Professor of Pharmacotherapy and Pharmaceutical Care, University of Groningen, The Netherlands
Designing Complex Interventions to Change Drug Utilisation

Lisa Kalisch Ellett, NHMRC-ARC Dementia Research Development Fellow, Quality Use of Medicines and Pharmacy Research Centre, School of Pharmacy and Medical Sciences, University of South Australia, Adelaide, Australia
Evaluating Interventions on Drug Utilisation: Clinical and Policy Perspectives

Petra Denig, Professor, Faculty of Medical Sciences, University of Groningen, Groningen, Netherlands
Assessment of Prescribing using Quality Indicators

Yared Santa-Ana-Téllez, Foundation for the Promotion of Health and Biomedical Research of Valencia Region (FISABIO), Spain
Evaluating Interventions to Promote Rational Drug Utilisation: Advanced methods-time series analysis

Speakers:
Caitlin Knox
Jaclyn Bosco
Katsiaryna Bykov
Olaf Klungel
Eric Tchetgen
Alec Walker

Course Aim

This half-day interactive course will touch on multiple advanced pharmacoepidemiology methods with each lecture providing the necessary tools for the attendees to apply the discussed principles to their own research.

Requisites Statement

Advanced level: An advanced level of understanding of pharmacoepidemiology study designs, data sources, and biases are needed for this advanced-level course.

Course Objectives

1. To review and discuss methods to estimate effects across multiple (>2) exposure groups
2. To describe main challenges and solutions for conducting drug-drug interaction outcome studies in electronic healthcare data.
3. To provide a gentle introduction to principles and applications of Doubly Robust Inference in Epidemiological Practice to address concerns about confounding adjustment and selection bias from missing data
4. Acquaint participants with the uses of conditional power

Syllabus Outline

This course will be moderated by Drs. Caitlin Knox and Jaclyn Bosco and the presentations include the topics below, which will be followed by Q&A and a discussion with the audience: 

Multi-group comparisons
Dr. Olaf Klungel

Multiple treatment options are often available to choose from, e.g. between classes of drugs, between different drugs within a class, or different dosages. To inform these decisions reliable estimates of the safety and effectiveness are needed. This lecture will review and discuss the different approaches to estimate treatment effects across >2 exposure groups, including methods such as (multinomial) regression and propensity score matching. Methods will be discussed based on real-life examples. Drug-

Drug interaction research
Dr. Katsiaryna Bykov

The lecture will introduce students to basic pharmacology of drug-drug interactions, specific methodological issues arising when conducting observational drug-drug interaction studies in electronic healthcare data and analytic approaches to address them. The advantages, assumptions, and limitations of various approaches will be illustrated using empirical examples and simulations. 

Double robustness
Dr. Eric Tchetgen

Double robustness refers to an appealing property of certain statistical methods that combine two separate regression models for the purpose of conducting inferences in epidemiologic applications, such that only one of the two models needs to be correct for valid inference, without necessarily knowing which of the two models is correct.  Therefore, doubly robust estimators (DR) offer two separate chances to obtain valid inferences from a single analysis, and therefore, are considerably more robust than non-doubly robust methods such as maximum likelihood estimators. In this course, we will describe a number of DR estimators for epidemiologic practice, including DR methods for confounding adjustment in estimating causal effects, that combine propensity score methods (e.g. inverse-probability weighting) and standard regression analysis such that valid inferences are obtained if either but not necessarily both estimators properly adjust for confounding.  Other key applications will be described in the context of instrumental variable analysis and adjustment for selection bias due to missing data. Simulation studies and several epidemiologic applications will be presented to illustrate the appealing robustness and efficiency properties of DR estimation

Conditional power
Dr. Alec Walker

Conditional power is the probability of a study achieving a numerical objective, as calculated under different hypotheses about the "truth" when the study is partially complete.  Participants will see how the need for conditional power calculation arises when there are unexpected events affecting study progress, and they will learn a straightforward technique for making the calculations.

Speakers:
Sebastian Schneeweiss
Jeremy Rassen
Nicolle Gatto
Xavier Kurz
Olaf Klungel
Natalia Petrusik-Ivleva

Course Aim

It is widely recognized that sensitivity analyses of study design choices and analytic assumptions help to interpret the robustness of pharmacoepidemiology studies. To encourage increased use of well-described techniques, this workshop will provide an introduction and demonstrations of a range of sensitivity analyses typically applied in pharmacoepidemiology with hands-on exercises. The course is interactive and participants will be able to conduct their own sensitivity analyses during the course – bring your own computer!

Part 1: Sensitivity analyses of study design choices will introduce typical variations in study designs, including variations in exposure risk window length, variations in covariate assessment window length, duration of minimum induction period, and variations in follow-up model (fixed time vs. as treated analysis). Using brief lectures followed by exercises, participants will make choices about sensitivity analysis assumptions and are able to observe the results of their choices in terms of changing parameter estimates and 95% confidence intervals. Two example case studies using claims data will illustrate the concepts. The audience will suggest variations in design choices and predict the impact to the results. Course faculty will implement these assumptions in real-time and discuss changes in study findings with the audience.

Part 2: Quantitative confounding bias analysis will focus on testing the influence of external assumptions or outside data on our understanding of residual confounding in a specific study setting. Using an Excel spreadsheet, participants will be guided through a “rule-out approach” and an “array approach” to residual confounding based on structural assumptions. We will also illustrate a simple algebraic approach to assessing the impact of residual confounding if more detailed information from electronic health records or registry data becomes available in a subset of the larger claims-based cohort.

This 4-hour pre-conference course focuses on principles and concepts, not on mathematical details. The course faculty includes members from academia, industry and regulatory to teach a broad perspective on the uses and interpretation of sensitivity analyses.

Educational objectives

Upon completing this course, participants will be able to:
• Recognize the value and applicability of sensitivity analysis in pharmacoepidemiology research
• To discuss the findings of sensitivity analyses of study design choices and understand the impact of varying study design assumptions in practical examples
• To explain quantitative confounding bias analysis and understand their interpretation in specific examples
• To develop strategies on how to plan for and communicate results of sensitivity analyses from the perspective of various stakeholders

Target audience

• This course is designed for scientists, regulators, coverage decision makers conducting and interpreting pharmacoepidemiology research studies using healthcare databases
• This beginner-to-intermediate-level course assumes knowledge of basic pharmacoepidemiologic methods

Speakers:
Mary E Ritchey
Daniel Prieto Alhambra
Christina Mack
Theodore Lystig
Colin Anderson-Smits

Course Aim

Over the past few years, there has been increased focus on generating real-world evidence for medical products. Thus, medical product epidemiology is gaining visibility outside of ISPE. Medical device epidemiology uses methodologies similar to those for medicines. However, alternative strategies are often implemented due to the nature of the products, data sources, and situational use. With the changing regulatory and reimbursement environments, it is more important than ever for pharmacoepidemiology researchers to better understand the methods and practice of medical device epidemiology, both for study of medical devices and combination products. Methods discussed will be applicable across therapeutic products will provide deeper understanding of measuring benefits and risks in clinical context.

This course provides a foundation for medical device epidemiology and its current applications in actual clinical practice. It will comprise of a 4-hour session that will incorporate didactic lectures and interactive case studies that highlight novel medical devices and combination products.

Requisites Statement

Entry level course. Topics covered will span entry-level, intermediate, and advanced topics in epidemiology.

Course Objectives

     1. Understand existing data sources for medical device epidemiological research and how to use them
     2. Explore methodologic considerations of research involving medical devices and combination products
     3. Recount real-world examples of medical device epidemiology, in the context of comparative effectiveness research
     4. Examine the dynamic regulatory environment for medical devices and how it differs from medications
     5. Discuss the role of patients in medical device research, including understanding of benefits and risks
     6. Learn about real-world medical devices and combination products in an interactive forum

Syllabus Outline

1. Overview of medical device epidemiology.
Students will be introduced to the language and regulation of medical devices and combination products. Emphasis will be placed on regulatory expectations as related to the role of epidemiologists. An update of changes related to real-world evidence within North America and Europe will be emphasized.

2. Overview of methods for medical device epidemiology.
Students will discuss conceptual methods and study designs applicable to medical device epidemiology, including identification of exposures, comparators, and outcomes; assessing provider effects and learning curves; and, biases specific to epidemiology of medical devices.

3. Data sources for medical device epidemiology.
Students will become familiar with data sources typically used for medical device epidemiology, with an emphasis on European data. Examples databases and research will be described.

4. Advances in medical device surveillance.
Students will hear about surveillance for medical devices, including coordinated registry networks, use of real-world data for expansion of indications, and leveraging already-collected data to better understand safety and effectiveness of medical devices and combination products.

5. Benefit-risk assessment for medical devices and combination products.
Students will be introduced to how benefit-risk balance is assessed for medical devices and combination products. Differences between US and European expectations will be outlined. Similarities and differences to expectations for medicines will be highlighted.

6. Cases studies.
Students will participate in two separate case study experiences:
• The first case study will engage students in building a device registry suitable to address a regulatory commitment
• The second case study will engage students in identifying gaps within the benefit-risk profile for a combination product and constructing a methodologically robust study to address the gap in research

Speakers:
Tamar Lasky, FISPE
Daniel B. Horton
Steven Bird
Mehmet Burcu

Course Aim

The increasing use of medications by children and the history of excluding children from clinical trials have created the need for pediatric pharmacoepidemiology, a sub-specialty within pharmacoepidemiology. Unique challenges in studying children, accessing data, defining outcomes, and designing pediatric studies will be discussed. This half-day course will introduce participants who have a good understanding of pharmacoepidemiology to the specific methodologic issues arising in pediatric pharmacoepidemiology and operational approaches used to study medications in children.

Requisites Statement

Entry level, with some knowledge of epidemiology, pharmacoepidemiology and pediatrics.

Course Objectives

Participants will gain an understanding of key issues in pediatric pharmacoepidemiology including:
• Considerations unique to pharmacoepidemiology in pediatric populations, including limitations in generalizing from adult populations, definitions of age sub-groups, and variables related to growth and development
• Selection and measurement of outcomes in pediatric pharmacoepidemiology
• Examples of pharmacovigilance and regulatory action in pediatric populations
• Impact of policy on drug utilization in pediatric populations

Syllabus Outline

1. Why Pediatric Pharmacoepidemiology?
Students will be introduced to the purpose and scope of pediatric pharmacoepidemiology as a sub-specialty of pharmacoepidemiology, reasons that few drugs have been adequately studied in children, and why information cannot always be extrapolated from adults to children. It will then address special issues around measurement of age in children and definition of age sub-groups.

2. Growth and Development in Pediatric Pharmacoepidemiology
Following a clinical overview on assessing childhood growth and development, the session will review examples and implications of studying growth and development as key outcomes and as aspects of medication exposure (including dosage) and effect modifiers.

3. Selecting and Measuring Useful Treatment Outcomes in Pediatric Drug Studies
Current guidance for the selection of outcomes in pediatric efficacy and safety studies will be discussed, including an overview on appropriate, reproducible and clinically meaningful outcome measures.

4. Pediatric pharmacovigilance, the case example of deferasirox and kidney injury
The process from evaluation of safety data to regulatory action will be described. The presenter will discuss safety signal identification, assessment of case reports and the published literature, pooled analyses of clinical trial laboratory data, clinical adverse event data, drug plasma concentration data, and resultant label changes.

5. Assessing the impact of policy on drug utilization in children and adolescents
With a focus on the assessment of prior authorization policies on pediatric use of medications, the session will review real-world examples and discuss study design and other methodological considerations.

6. Interactive discussion
Course faculty and attendees will divide into breakout groups to critique a current example of pediatric pharmacoepidemiologic research. Faculty will facilitate small group discussions and provide additional points for discussion.

Speakers
Gerald Dal Pan
June Raine
Danica Marinac-Dabic
Stephen Evans

Course Aim

Medical product regulation increasingly relies on pharmacoepidemiologic analyses to assess the safety of medical products once they are marketed. This course is aimed at describing how pharmacoepidemiologic analyses are used in regulatory decision making, and the challenges that regulators face when using such analyses.

Requisites Statement

Intermediate level

Course Objectives
1. Understand the role of pharmacoepidemiologic analyses in regulatory decision making.
2. Understand the strengths and limitations of pharmacoepidemiologic analyses in regulatory decision making.

Syllabus Outline

More details coming soon!

Speakers:
Rebecca Noel
Tarek A. Hammad
Juhaeri Juhaeri
Brett Hauber

Course Aim

Benefit-risk assessment is now a fundamental science for understanding and evaluating the development, approval, and continued marketing of drugs and devices. This course is aimed at introducing attendees to core concepts and tools of both the science and practice of benefit-risk assessment.

Requisites Statement

This is an introductory to mid-level course designed to acquaint attendees with the current state of the art in the use of benefit-risk assessment methods and tools.

Course Objectives

• Develop an understanding of quantitative benefit-risk assessment methods and software tools
• Develop an understanding of how to successfully structure and present benefit-risk information for drug development decision-making and regulatory interactions, including the use of visual tools
• Develop an understanding of how pharmacoepidemiology and benefit-risk assessment intersects with patient engagement, by developing an understanding of the use of preference methods

Syllabus Outline

Tarek A. Hammad, MD, PhD, MSc, MS, FISPE
• Tarek Hammad will provide attendees with an overview of ICH expectations and analytical considerations for the development and presentation of benefit-risk information in new drug applications

Juhaeri Juhaeri, PhD
• Juhaeri Juhaeri will provide attendees with an overview of leading quantitative methods (e.g., MCDA, SMAA) for the development and presentation of benefit-risk assessment information

Brett Hauber, PhD
• Brett Hauber will provide attendees with an overview of leading methods (e.g., discrete choice, direct elicitation, threshold, contingent valuation ) for the elicitation, analysis and presentation of patient preference information

Full meeting registration includes food, drink and entertainment.

Open to All participants

Open to all participants

Open to all participants.

Please arrive 24 hours before your presentation start time to upload or check your presentation. 

Alison Bourke, FISPE, ISPE President

Daniel B. Horton, Chair, 2019 ICPE Local Host Committee

Vincent Lo Re. FISPE, Chair, 2019 ICPE Scientific Program Committee

With the 21st Century Cures Act, U.S. Congress directed the FDA to explore how real-world evidence (RWE) can be used to support approval of new indications for existing drugs and to satisfy post-approval study requirements.  To address this mandate, FDA committed to conducting pilot studies and developing guidance on how RWE can contribute to the assessment of safety and effectiveness in regulatory submissions.  In this keynote address, the FDA speaker will discuss the current application of RWE in regulatory decision making, the development of evidence to support fitness for use, future directions, and the role of pharmacoepidemiology in the generation and evaluation of RWE.  A panel of pharmacoepidemiologists will provide their own perspectives on the promises and challenges of using RWE to assess the safety and effectiveness of drugs and biologics. 

Keynote Speaker

• Jacqueline Corrigan-Curay, JD, MD, FDA/CDER/OMP Director

Moderator:

• Vin Lo Re, FISPE, Associate Professor of Medicine & Epidemiology, Perelman School of Medicine, Center for Clinical Epidemiology & Biostatistics, University of Pennsylvania

Panelists

• Brian Strom, FISPE, Chancellor, Rutgers Biomedical & Health Sciences; Executive Vice President for Health Affairs; University Professor; Rutgers, the State University of New Jersey
• Solomon Iyasu, Vice President and Head of Pharmacoepidemiology, Merck & Company, Inc.
• June Raine, Director, Vigilance and Risk Management, Medicines and Healthcare products Regulatory Agency

Drugs and Germs: Dirty Details about Pharmacoepidemiology and the Microbiome [5]:  Room 202 AB

Daniel B Horton, Peter J Turnbaugh, Scott E Plevy, Soko Setoguchi, Tobias Gerhard, James D Lewis. (United States)

How Do We Assess When Electronic Health Records or Claims Databases are Fit for a Specific Research or Regulatory Purpose? [2]:  Room 201 A

Cynthia J Girman, Mary E Ritchey, David Martin, Wei J Zhou, Nancy J Dreyer. (United States)

Pregnancy Registries and Pregnancy Outcome Studies in Healthcare Databases: Trends and Complementary Research Approaches [4]:  Room 201 C

Steve Gao, Krista Huybrechts, Leyla Sahin, John Seeger, Daina Esposito, Christina D. Chambers. (United States)

What Does Replicable and Robust Non-Randomized Database Evidence Look Like? [1]:  Ballroom B

Shirley Wang, Jeremy Rassen, Simone Pinheiro, Jesse Berlin, Sebastian Schneeweiss. (United States)

How Do We Assess When Electronic Health Records or Claims Databases are Fit for a Specific Research or Regulatory Purpose? [2]:  Room 201 A

Cynthia J Girman, Mary E Ritchey, David Martin, Wei J Zhou, Nancy J Dreyer. (United States)

When Artificial Intelligence Meets Pharmacoepidemiology [3]:  Room 201 B

Dorothee B Bartels, Jim Slattery, Antoine Pariente, John Rigg. (Germany)

Pregnancy Registries and Pregnancy Outcome Studies in Healthcare Databases: Trends and Complementary Research Approaches [4]:  Room 201 C

Steve Gao, Krista Huybrechts, Leyla Sahin, John Seeger, Daina Esposito, Christina D. Chambers. (United States)

Drugs and Germs: Dirty Details about Pharmacoepidemiology and the Microbiome [5]:  Room 202 AB

Daniel B Horton, Peter J Turnbaugh, Scott E Plevy, Soko Setoguchi, Tobias Gerhard, James D Lewis. (United States)

What Does Replicable and Robust Non-Randomized Database Evidence Look Like? [1]:  Ballroom B

Shirley Wang, Jeremy Rassen, Simone Pinheiro, Jesse Berlin, Sebastian Schneeweiss. (United States)

When Artificial Intelligence Meets Pharmacoepidemiology [3]:  Room 201 B

Dorothee B Bartels, Jim Slattery, Antoine Pariente, John Rigg. (Germany)

Open to all participants; lunch available.

• Education       Marrriott/304-305
• Finance          Marriott/302-303
• Membership    Marriott/306

Adherence; Asian Pharmacoepidemiology Network (AsPEN);  Biologics & Biosimilars; Comparative Effectiveness Research (CER); & Geriatric Pharmacoepidemiology

Moderators: Deborah Layton & Jesper Hallas, FISPE

1:30 PM               

Modeling Effect Heterogeneity in the Presence of Heterogeneous Confounding: A Plasmode Simulation [24]

Mitchell M Conover, Virginia Pate, Michele Jonsson Funk. (United States)

1:45 PM               

A Little off the Sides: Propensity Score Trimming in the Presence of Heterogeneity [25]

Michael Webster-Clark, Robert Glynn, Til Stürmer. (United States)

2:00 PM               

Propensity Score Trimming - Lessons from a Study of Antipsychotic Augmentation for Depression [26]

Tobias Gerhard, Daniel Horton, Mark Olfson, T Scott Stroup, Soko Setoguchi, Brian L Strom. (United States)

2:15 PM               

Residual Confounding and Treatment Effect Heterogeneity: An Applied Example [27]

Mitchell M Conover, Virginia Pate, Michele Jonsson Funk. (United States)

2:30 PM               

Novel Approaches for Defining Binary Confounders in Claims Data: A Simulation Study [28]

John G Connolly, Sebastian Schneeweiss, Robert J Glynn, Joshua J Gagne. (United States)

2:45 PM               

An Empirical Evaluation of a Propensity Score Stratification Based Weighting Approach in Settings of More Than Two Treatment Groups [29]

Rishi J Desai, Kazuki Yoshida, Krista Huybrechts, Jessica Franklin. (United States)

Moderators: Christina Chambers & Marleen VanGelder

1:30 PM               

Comparative Safety of Antiepileptic Drugs and Risk of Major Congenital Malformations [18]

Jacqueline M Cohen, Maarit K Leinonen, Silje Alvestad, Marte-Helene Bjørk, Carolyn E Cesta, Kristjana Einarsdóttir, Anders Engeland, Mika Gissler, Óskar Hálfdánarson, Øystein Karlstad, Helle Kieler, Kari Klungsøyr, Mette Nørgaard, Lars Pedersen, Randi Selmer, Helga Zoega, Kari Furu. (Norway)

1:45 PM               

Use of Oral Fluconazole during Pregnancy and Risks of Spontaneous Abortion, Stillbirth and Major Congenital Malformation: An International, Multi-Centre Cohort Study [19]

Colin Dormuth, Brandace Winquist, Anat Fisher, Fangyun Wu, Pauline Reynier, Matthew Dahl, Zhihai Ma, Xinya Lu, Jianguo Zhang, Colette Raymond, Kristian B. Filion, Robert W Platt, Carolina Moriello, J. Michael Paterson, CNODES Investigators. (Canada)

2:00 PM               

Oral Fluconazole Use in Pregnancy and the Risk of Birth Defects [20]

Yanmin Zhu, Brian T Bateman, Sonia Hernandez-Diaz, Kathryn J Gray, Helen Mogun, Krista F Huybrechts. (United States)

2:15 PM               

The Impact of In-Utero Prescription Opioid Exposure on Congenital Malformations [21]

Loreen Straub, Krista F Huybrechts, Sonia Hernandez-Diaz, Helen Mogun, Brian T Bateman. (United States)

2:30 PM               

Risk of Neonatal Abstinence Syndrome Following Prenatal Exposure to Methadone vs Buprenorphine [22]

Mollie Wood, Milada Mahic, Marte Handal, Svetlana Skurtveit, Ingunn Olea Lund, Sonia Hernandez-Diaz. (United States)

2:45 PM               

Paternal Exposure to Finasteride - before and during Pregnancy [23]

Jon T Andersen, Thomas B Jensen, Henrik Horwitz, Stine S Clausen. (Denmark)

Moderators: Joris Komen & Marie de Bruin

1:30 PM               

Use of Stimulants in Adults and Risk of Cardiovascular Events: A Multi-Design Approach [30]

Mina Tadrous, Simon Greaves, Michael Paterson, Jennifer Watt, David N Juurlink, Diana Martins, Muhammad M Mamdani, Tara Gomes. (Canada)

1:45 PM               

Mirabegron and the Risk of Arrhythmias: A Population-Based Cohort Study [31]

Mina Tadrous, Rano Matta, Simon Greaves, David N Juurlink, Muhammad Mamdani, Tara Gomes. (Canada)

2:00 PM               

Risk of Major Cardiovascular Events Associated with Varenicline and Nicotine Replacement Therapy: An Australian Population-Based Study [32]

Alys Havard, Stephanie K Choi, Clara K Chow, Duong T Tran, Kristian B Filion. (Australia)

2:15 PM               

Cardiovascular Safety of Prucalopride for Chronic Constipation: A Multinational Population-Based Cohort Study [33]

Alicia Gilsenan, Joan Fortuny, Miguel Cainzos-Achirica, Oscar F. Cantero, Robert W. Flynn, Luis Garcia-Rodriguez, Abenah Harding, Bianca Kollhorst, Pär Karlsson, Love Linnér, Thomas M. MacDonald, Ingvild Odsbu, Estel Plana, Ana Ruigómez, Tania Schink, Ryan Ziemiecki, Elizabeth B. Andrews. (United States)

2:30 PM               

Cardiotoxicity of Aromatase Inhibitors in Post-Menopausal Women with Breast Cancer: A Population-Based Cohort Study [34]

Farzin Khosrow-Khavar, Nathaniel Bouganim, Kristian B. Filion, Samy Suissa, Laurent Azoulay. (Canada)

2:45 PM               

Diagnosis of Immune Checkpoint Inhibitor Associated Myocarditis: A Systematic Review of Case Reports and Observational Studies [35]

Richeek Pradhan, Amit Nautiyal, Sonal Singh. (United States)

 Moderators: Daniel Arhinful & Kimberly Brodovicz

1:30 PM

Scientific Evidence Supporting Benefits of Novel Therapeutic Agents Approved by the US Food and Drug Administration, 2015-2017 [6]

James Heyward, G. Caleb Alexander, Thomas J. Moore. (United States)

1:45 PM

Cost-Effectiveness of First Line Tyrosine Kinase Inhibitor Initiation Strategies for Chronic Myeloid Leukemia [7]

Joehl T. Nguyen, Ashley L. Cole, Ashley A. Leech, William A. Wood, Stacie B. Dusetzina. (United States)

2:00 PM

Healthcare Resource Use and Expenditures in Patients Newly Diagnosed with Paroxysmal Supraventricular Tachycardia in the United States [8]

Naomi C. Sacks, Katie Everson, Philip L. Cyr, Madison T. Preib, David R. Wood, Sean D. Pokorney. (United States)

2:15 PM

The Economic Impact of the Introduction of Infliximab-Biosimilar: Preliminary Results from a Study on Rheumatologic Patients in Tuscany, Italy [9]

Valentina Lorenzoni, Leopoldo Trieste, Marta Mosca, Irma Convertino, Marco Tuccori, Ersilia Lucenteforte, Sara Ferraro, Luca Leonardi, Giuseppe Roberto, Nicoletta Luciano, Corrado Blandizzi, Rosa Gini, Giuseppe Turchetti. (Italy)

2:30 PM

Antithrombotic Stewardship [10]

Albert Dreijer, Marieke JHA Kruip, Jeroen Diepstraten, Suzanne Polinder, Rolf Brouwer, Nanne Croles, Esther Kragten, Frank WG Leebeek, Patricia van den Bemt. (Netherlands)

2:45 PM

Privacy versus Public Good: The Methodological Approach of the Belgian Healthdata.be Platform to Protect Individual Privacy in Health Research [11]

Kaatje Bollaerts, Maria Alexandridou, Simeon Wanyama, Kris Vranken. (Belgium)

Moderators: Andrew Zullo & Rachel Sobel, FISPE

1:30 PM               

Effects of Bisphosphonate Use on Knee Osteoarthritis Progression: An Analysis of the Osteoarthritis Initiative Study [36]

Kaleen N Hayes, Vasily Giannakeas, Andy Kin On Wong. (Canada)

1:45 PM               

The Effect of Beta Blocker Use on Bone Outcomes in the Framingham Osteoporosis Study [37]

Christine W Lary, Kathleen Nevola, Alexandra C Hinton, Theresa I Shireman, Katherine Motyl, Karen L Houseknecht, Frances L Lucas, Sarah Hallen, Andrew R Zullo, Sarah D Berry, Douglas P Kiel. (United States)

2:00 PM               

Sodium-Glucose Co-Transporter 2 Inhibitors and the Risk of Fractures among Patients with Type 2 Diabetes [38]

Devin Abrahami, Antonios Douros, Hui Yin, Oriana H Yu, Laurent Azoulay. (Canada)

2:15 PM               

Immunosuppression, Fractures, and Post-kidney Transplant Outcomes among Older Recipients [39]

Mara McAdams DeMarco, Megan Salter, Sunjae Bae, Nadia Chu, Dorry Segev. (United States)

2:30 PM               

Benzodiazepines and Risk of Fractures in Young People [40]

Greta A Bushnell, Tobias Gerhard, Stephen Crystal, Mark Olfson. (United States)

2:45 PM               

Long-Term Budesonide Treatment and Risk of Osteoporotic Fractures in Patients with Microscopic Colitis [41]

Mette Reilev, Ole K Bonderup, Gunnar L Nielsen, Martin T Ernst, Jesper Hallas. (Denmark)

Moderators: Seoyoung Kim & Maribel Salas, FISPE

1:30

Infant Infections after Maternal Anti-TNF Treatment in Pregnancy [12]

Gabriella Broms, Helle Kieler, Anders Ekbom, Mika Gissler, Karin Hellgren, Maarit K Leinonen, Lars Pedersen, Marcus Schmitt-Egenolf, Henrik Toft Sørensen, Fredrik Granath. (Sweden)

1:45 PM

The Association between Atopic Eczema and Cancer in England and Denmark: Two Cohort Studies [13]

Kathryn E Mansfield, Sigrún A Jóhannesdóttir Schmidt, Bianka Darvalics, Amy Mulick, Katrina Abuabara, Angel Wong, Henrik Toft Sørensen, Liam Smeeth, Krishnan Bhaskaran, Isabel dos Santos Silva, Richard J Silverwood, Sinéad M Langan. (United Kingdom)

2:00 PM

The Association between Partner Bereavement and Incident Psoriasis or Atopic Dermatitis: A Matched Cohort Study [14]

Angel YS Wong, Harriet J Forbes, Amy Mulick, Kathryn Mansfield, Richard J Silverwood, Henrik T Sørensen, Liam Smeeth, Sigrún AJ Schmidt, Sinéad M Langan. (United Kingdom)

2:15 PM

Risk of Liver Disease in Patients with Psoriasis, Psoriatic Arthritis, and Rheumatoid Arthritis Treated with Methotrexate [15]

Joy Wan, He Zhang, Alexis Ogdie, Alexander Egeberg, Joel M Gelfand. (United States)

2:30 PM

Prediction of Cardiovascular Events in Rheumatoid Arthritis Patients Using Protein Biomarkers and Clinical Factors [16]

Jeffrey R Curtis, Fenglong Xie, Lang Chen, Eric Sasso, Elena Hitraya, Jerry S Lanchbury, Darl Flake, Alexander Gutin, Cheryl Chin, Cynthia Crowson. (United States)

2:45 PM

Induction Immunosuppression Practices in Liver Transplantation Are Variable and Predict Post-Transplant Outcomes [17]

Therese Bittermann, Rebecca A Hubbard, James D Lewis, David S Goldberg. (United States)

Heterogeneity and Validity in National and International Multi-Database Pharmacoepidemiologic Studies (MPES): Lessons Learned in North America, Europe, and Asia [43] :  Room 201 A

Edward Chia-Cheng Lai, Kenneth Man, Darren Toh, Robert Platt, Jesper Hallas, Soko Setoguchi. (Taiwan)

Machine Learning to Improve Causal Inference in Pharmacoepidemiology [45]:  Room 201 C

Jason Roy, Nandita Mitra. (United States)

Machine Learning to Improve Causal Inference in Pharmacoepidemiology [45]:  Room 201 C

Jason Roy, Nandita Mitra. (United States)

Measuring, Mapping, and Meshing of Genotype and Phenotype Data across Large Datasets: Challenges of Combining and Mining Genotype and Phenotype Data [46]  Room 202 AB

Leah Sansbury, Meg Ehm, Marylyn Richie, Amalia Issa, Colin Palmer, Edgar Simard. (United States)

The Ascension of Real-World Evidence: Can (and Should) We Establish Thresholds for Validity? [42]:  Ballroom B

Nicolle M Gatto, Cynthia Girman, Stephan Lanes, David Martin, Robert F Reynolds, Sebastian Schneeweiss, Jim Slattery. (United States)

Heterogeneity and Validity in National and International Multi-Database Pharmacoepidemiologic Studies (MPES): Lessons Learned in North America, Europe, and Asia [43] :  Room 201 A

Edward Chia-Cheng Lai, Kenneth Man, Darren Toh, Robert Platt, Jesper Hallas, Soko Setoguchi. (Taiwan)

Thinking Globally While Acting Locally: Developing Time-on-Treatment Data in International Settings [44]: Room 201 B

Judith C. Maro, Kristian B. Filion, Achim Wolf, Kevin Haynes, Michael D. Nguyen. (United States)

Machine Learning to Improve Causal Inference in Pharmacoepidemiology [45]:  Room 201 C

Jason Roy, Nandita Mitra. (United States)

Measuring, Mapping, and Meshing of Genotype and Phenotype Data across Large Datasets: Challenges of Combining and Mining Genotype and Phenotype Data [46]  Room 202 AB

Leah Sansbury, Meg Ehm, Marylyn Richie, Amalia Issa, Colin Palmer, Edgar Simard. (United States)

The Ascension of Real-World Evidence: Can (and Should) We Establish Thresholds for Validity? [42]:  Ballroom B

Nicolle M Gatto, Cynthia Girman, Stephan Lanes, David Martin, Robert F Reynolds, Sebastian Schneeweiss, Jim Slattery. (United States)

Thinking Globally While Acting Locally: Developing Time-on-Treatment Data in International Settings [44]: Room 201 B

Judith C. Maro, Kristian B. Filion, Achim Wolf, Kevin Haynes, Michael D. Nguyen. (United States)

Moderators: Hedvig Nordeng & Alys Havard

5:15 PM               

Risk of Pregnancy Loss in Patients Exposed to Mycophenolate Compared to Azathioprine: A Retrospective Cohort Study [53]

Thuy N. Thai, Amir Sarayani, Xi Wang, Yasser Albogami, Sonja Rasmussen, Almut G. Winterstein. (United States)

5:30 PM               

Exposure to Antidepressants during Pregnancy and the Risk of Attention Deficit Hyperactivity Disorder in Offspring: Findings from a Nationwide Cohort Study with and without Sibling Design [54]

Buket Öztürk, Lars Pedersen, Vera Ehrenstein, Henrik T Sørensen. (Denmark)

5:45 PM               

Does the Type of Antihypertensive Medication Used in Pregnancy Affect Risk of Pre-Eclampsia? [55]

Sascha Dublin, Mary A Akosile, Lu Chen, Lyndsay A Avalos, T Craig Cheetham, Thomas Easterling, Victoria L Holt, Sylvia E Badon, Romain S Neugebauer, Kristi Reynolds, Rod L. Walker, Susan M Shortreed. (United States)

6:00 PM               

Prenatal Antidepressant Exposure and Perinatal Outcomes: An Analysis Utilizing Longitudinal Clustering Methods [56]

Gretchen Bandoli, Christina Chambers, Kristin Palmsten. (United States)

6:15 PM               

Incident Exposure to Benzodiazepine in Early Pregnancy and the Risk of Spontaneous Abortion [57]

Odile Sheehy, Anick Bérard, Jin-Ping Zhao. (Canada)

6:30 PM               

Ondansetron Use in Early Pregnancy and Miscarriage [58]

Elizabeth A Suarez, Kim Boggess, Stephanie M Engel, Jennifer L Lund, Til Stürmer, Michele Jonsson Funk. (United States)

Moderators: Christine Ulbricht & Sudha Raman

5:15 PM               

François Montastruc, Rui Nie, Simone Loo, Soham Rej, Sophie Dell'Aniello, Joelle Micallef, Samy Suissa, Christel Renoux. (Canada)

5:30 PM               

Changes in the Population Characteristics of U.S. Youth Receiving Psychotropic Polypharmacy: 1996 to 2016 [72]

Chengchen Zhang, O'Mareen Spence, Gloria Reeves, Susan dosReis. (United States)

5:45 PM               

Trends in Antipsychotic-Related Polypharmacy among U.S. Youth from 1996 to 2016 [73]

Chengchen Zhang, O'Mareen Spence, Gloria Reeves, Susan dosReis. (United States)

6:00 PM               

Off-Label Use of Antipsychotics among US Commercially-Insured Youth [74]

Kyungwan Hong, O'Mareen Spence, Susan dosReis. (United States)

6:15 PM               

Risk of Psychosis with Amphetamine versus Methylphenidate in Attention Deficit Hyperactivity Disorder [75]

Lauren V Moran, Dost Ongur, John Hsu, Victor M Castro, Roy H Perlis, Sebastian Schneeweiss. (United States)

6:30 PM               

The Occurrence of Suicide-Related Outcomes among Patients Initiating Tegaserod in Routine Clinical Practice [76]

John D Seeger, Sherry G Quinn, Heather Norman, Judy Wong. (United States)

Moderators: Andrew Freeman, FISPE, & Cynthis De Luise

5:15 PM               

Cholesterol-Lowering Medication and Breast Cancer Recurrence in Postmenopausal Women Treated with Adjuvant Aromatase Inhibitors: A Danish Population-Based Cohort Study [77]

Karl S. Harborg, Uffe Heide-Jørgensen, Thomas P. Ahern, Maj-Britt Jensen, Marianne Ewertz, Deirdre Cronin-Fenton, Signe Borgquist. (Denmark)

5:30 PM               

Association between Hydrochlorothiazide Exposure and Skin, Lip and Oral Cancer: A Series of Population-Based Nested Case-Control Studies [78]

Daniel R Morales, Alexandra Pacurariu, Xavier Kurz, Jim Slattery. (United Kingdom)

5:45 PM               

Voriconazole and Cutaneous Squamous Cell Carcinoma Risk among Lung Transplant Recipients [79]

Monica D'Arcy, Ruth Pfeiffer, Donna R Rivera, Sarah Aaron, Elizabeth Yanik, Isaac Brownell, Edward Cowen, Matthew Triplette, Greg Hess, Eric A Engels. (United States)

6:00 PM               

Detection of Prevalent Cancer among New Users without Routine Care [80]

Monica D'Arcy, Til Stürmer, Michele Jonsson Funk. (United States)

6:15 PM               

Automatic Detection of Drug-Drug Interactions in Cancer Patients: A French Population-Based Cohort Study [81]

Camille Morival, Clément Mathieu, Antoine Pariente, Mathieu Molimard, Amandine Gouverneur, Pernelle Noize. (France)

6:30 PM               

Treatment Pattern and Real World Effectiveness on Non Small Cell Lung Cancer Patients Harboring Major Single and Double EGFR Mutations [82]

W. Venus So, Xing Yang, Mathias Leddin, Lin-Chi Chen, Yvonne A Nagel. (United States)

Moderators: Charles Leonard & Edward Lai

5:15 PM               

Analysis of Sodium-Glucose-Cotransporter-2 Inhibitor Use in Patients with Type-1 Diabetes Mellitus and Rates of Diabetic Ketoacidosis [65]

Christian Hampp, Richard Swain, Casie Horgan, Elizabeth Dee, Yandong Qiang, Sarah Dutcher, Andrew Petrone, Rong Chen Tilney, Judith C Maro, Catherine A Panozzo. (United States)

5:30 PM               

Clinical Outcomes in Patients with Type 2 Diabetes Newly Initiating SGLT2 or DPP4 Inhibitors [66]

Sruthi Adimadhyam, Danielle Isaman, Andrew Weckstein, Emma Payne, Edward Yu, Anthony Louder, Shannon L. Reynolds. (United States)

5:45 PM               

Incidence Rate of Fournier’s Gangrene in Type-2 Diabetic Patients Using Second-Line Anti-Diabetic Drugs [67]

Sara Eapen, Sebastian Schneeweiss, Aditya Rajan, Natalia Petruski-Ivleva, Saira A Jan. (United States)

6:00 PM               

SGLT-2 Inhibitors and the Risk of Community-Acquired Pneumonia in Patients with Type-2 Diabetes [68]

Vanessa Brunetti, Pauline Reynier, Laurent Azoulay, Oriana H. Yu, Pierre Ernst, Robert W Platt, Kristian B Filion. (Canada)

6:15 PM               

Risk of Inflammatory Bowel Disease with Sodium Glucose Co-Transporter-2 Inhibitors Compared to Dipeptidyl Peptidase-4 Inhibitors in Patients with Type 2 Diabetes [69]

Ghadeer K. Dawwas, Steven M. Smith. (United States)

6:30 PM               

Sodium-Glucose Co-Transporter 2 Inhibitors and the Risk of Venous Thromboembolism in Patients with Type 2 Diabetes: A Cohort Study [70]

Niklas Schmedt, Dirk Enders, Felix Müller, Jochen Walker, Edeltraut Garbe, Antonios Douros. (Germany)

Moderators: Kenneth Man & Vicki Osborne

5:15 PM               

Use of Psychotropic Drugs in Children and Adolescents with Tic Disorders in Denmark: A Nationwide Drug-Utilization Study [59]

Lotte Rasmussen, Julie M Thrane, Niels Bilenberg, Anton Pottegård. (Denmark)

5:30 PM               

Patterns of Drug Utilisation over Age in Asthmatic Children [60]

Esmé Baan, Astrid Heeremans, Leila Karimi, Hettie M Janssens, Miriam Karimi, Johan C de Jongste, Lies Lahousse, Katia Verhamme. (Netherlands)

5:45 PM               

Methylphenidate Treatment during Childhood Is Continued in Adulthood in Half of the Study Population [61]

Chii Chyi Cheung, Katia M Verhamme, Ron M Herings, Loes E Visser, Bruno H Stricker. (Netherlands)

6:00 PM               

Unintended Consequences of a National Policy Intervention to Curb Inappropriate Alprazolam Prescribing in Australia [62]

Andrea L Schaffer, Nicholas A Buckley, Sallie Pearson. (Australia)

6:15 PM               

Prescribing Systemic Steroids for Acute Respiratory Tract Infections in US Outpatient Settings [63]

Kueiyu Joshua J Lin, Evan Dvorin, Aaron S Kesselheim. (United States)

6:30 PM               

Characteristics of Febuxostat and Allopurinol Initiators and Utilization Patterns in Real-World Settings [64]

Marie Bradley, Efe Eworuke, Austin Cosgrove, Laura Hou, Darren Toh. (United States)

Moderators: Helga Gardarsdottir, FISPE, & Eric Wittbrodt

5:15 PM               

Using Negative Control Outcomes to Assess the Comparability of Osteoporosis Treatment Groups [47]

Leah J. McGrath, Leslie Spangler, Jeffrey R. Curtis, Vera Ehrenstein, Henrik Toft Sørensen, Bradley Saul, Sara Levintow, M. Alan Brookhart, Brian D. Bradbury. (United States)

5:30 PM               

Can Adjustment Fail? Confounder Misclassification in Single-Arm Studies with Real World Comparators [48]

Michael Webster-Clark, Mitchell Conover, Michele Jonsson-Funk, Til Stürmer. (United States)

5:45 PM               

Confounding by Indication in Drug-Cancer Association Studies: A Nested Case-Control Study of the Association between Calcium Channel Blockers and Renal Cell Carcinoma [49]

Kasper Bruun Kristensen, Laurel A Habel, Joshua J Gagne, Søren Friis, Jesper Hallas, Klaus Kaae Andersen, Anton Pottegård. (Denmark)

6:00 PM               

Visualization Tool of Variable Selection for Inverse Probability Weighted Estimator in High-Dimensional Covariates [50]

Ya-Hui Yu, Ashley I Naimi, Lisa M Bodnar, Kristian B Filion, Robert W Platt. (Canada)

6:15 PM               

A General Propensity Score for Signal Detection Using Tree-Based Scan Statistics [51]

Shirley V Wang, Joshua J Gagne, Judith C Maro, Sushama Kattinakere, Danijela Stojanovic, Efe Eworuke, Elande Baro, Rita Ouellet-Hellstrom, Michael Nguyen, Sandra DeLuccia, Ella Pestine, Martin Kulldorff. (United States)

6:30 PM               

Approaches to Overcome the Immeasurable Time Bias in Cohort Studies: Beta-Blockers and Risk of Mortality in Patients with Heart Failure [52]

In-sun Oh, Han Eol Jeong, Kristian B. Filion, Ju-Young Shin. (Korea, Republic of)

Open to all participants.

Adherence SIG:  Room 201 A

Asian Pharmacoepidemiology Network (AsPEN) SIG:  Room 201 B

Benefit Risk Assessment, Communication and Evaluation (BRACE) SIG:  Room 201 C

Biologics and Biosimilars:  Room 202 AB

Comparative Effectiveness Research (CER):  Room 204 A

Database:  Room204 B

Digital Epidemiology:  Room 204 C

Drug Utilization Research:  Room 203 A

Geriatric Pharmacoepidemiology:  Room 203 B

Medical Devices:  Room 101 AB

Medicines in Pregnancy & Lactation:  Room 101

Molecular Epi/Biomarkers/Pharmacogenetics:  Room 103

Oncology:  Room 104

Pediatrics:  Marriott/301

Rare Disease:  Marriott/302-302

Vaccines:  Marriott/304-305

By invitation only

Registration required -- limited capacity.
Organized by the Student Council. Please contact SISPEchair@gmail.com for more information, and to RSVP.

Open to all participants

Open to all participants

Please arrive 24 hours before your presentation start time to upload or check your presentation. 

Moderators: Cynthia Girman, FISPE, & Mina Tadrous

8:00 AM               

Data-Driven Identification of Indication for Treatment in Electronic Medical Records Using Cluster Analysis in Combination with a Self-Controlled Cohort Analysis [411]

Tomas Bergvall, Birgitta Grundmark, Alison Bourke, Niklas Norén. (Sweden)

8:15 AM               

Evaluation Framework to Guide Model Selection and Cohort Definition in Causal Inference [412]

Yishia Shimoni, Sivan Ravid, Ehud Karavani, Peter Bak, Marie Ng, Sharon Hensley Alford, Denise Meade, Ya'ara Goldschmidt. (United States)

8:30 AM               

A R-Based Application to Perform Distributed Regression Analysis with Vertically Partitioned Data [413]

Qoua Her, Yuji Samizo, Thomas Kent, Mia Gallagher, Aleksandra Slavkovic, Sengwee Toh. (United States)

8:45 AM               

Privacy-Protecting Estimation of Adjusted Risk Ratios Using Modified Poisson Regression in Multi-Center Studies [414]

Di Shu, Jessica G Young, Sengwee Darren Toh. (United States)

9:00 AM               

Relying on RE-LY to Transport Effect Estimates to Patients in Routine Care [415]

Michael Webster-Clark, Til Stürmer, Jessie K Edwards, Charles Poole, Ross Simpson, Jennifer L Lund. (United States)

9:15 AM               

An Online Tool for Correcting Risk Ratio or Cumulative Incidence Estimates for Bias Due to Outcome Misclassification [416]

Gillian C Hall, Stephan Lanes, Kaatje Bollaerts, Xiaofeng Zhou, Vinay Mehta, Germano Ferreira, Rosa Gini. (United Kingdom)

Moderators: CHAIR PENDING

8:00 AM               

Opioid Prescription Use after Vaginal Delivery and Subsequent Persistent Opioid Use and Misuse [435]

Yanmin Zhu, Krista F Huybrechts, Rishi J Desai, Alexis Krumme, Loreen Straub, Raisa Levin, Helen Mogun, Sonia Hernandez-Diaz, Brian T Bateman. (United States)

8:15 AM               

Postpartum Opioid Prescribing and the Risk of Persistent Opioid Use [436]

Sarah S Osmundson, Jae Young Min, Andrew D Wiese, Robert E Hawley, Stephen W Patrick, Marie R Griffin, Carlos G Grijalva. (United States)

8:30 AM               

Prevalence and Predictors of ADHD Medication Use in a Cohort of Pregnant Women [437]

Maxim Lemelin, Takoua Boukhris, Odile Sheehy, Anick Bérard. (Canada)

8:45 AM               

Interrupted Time Series Analysis to Assess Prescription Filling around Conception and Implications for Misclassification of Medication Use in Pregnancy [438]

Jacqueline M Cohen, Randi Selmer, Kari Furu, Øystein Karlstad. (Norway)

9:00 AM               

Trajectories of Antipsychotic Use before and during Pregnancy and Related Maternal and Birth Characteristics [439]

Andrea L Schaffer, Helga Zoega, Duong T Tran, Nicholas A Buckley, Sallie Pearson, Alys Havard. (Australia)

9:15 AM               

Medication Use Patterns in Pregnant Women with Migraine [440]

Rohini K. Hernandez, Sonja Nakasian, Lisa Bollinger, Brian Bradbury, Susan Jick, Paul Muntner, Eric Ng, Victoria Chia. (United States)

Moderators: Rianne van den Ham & Lourens Bloem

8:00 AM               

Clopidogrel Drug Interactions and Serious Bleeding: High-Throughput Screening Using the Self-Controlled Case Series Design [417]

Charles E. Leonard, Meijia Zhou, Colleen M. Brensinger, Warren B. Bilker, Samantha E. Soprano, Thanh Phuong Pham Nguyen, Young Hee Nam, Jordana B. Cohen, Sean Hennessy. (United States)

8:15 AM               

Statins and Risk of Venous Thromboembolism [418]

Nils Skajaa, Szimonetta K. Szépligeti, Erzsébet Horváth-Puhó, Waleed Ghanima, John B. Hansen, Henrik T. Sørensen. (Denmark)

8:30 AM               

Antithrombotic Treatment Preceding Ischemic Stroke, Intracranial Hemorrhage, and Gastrointestinal Bleeds and Mortality in Patients with Atrial Fibrillation [419]

Joris J Komen, Tomas Forslund, Aukje K Mantel-Teeuwisse, Olaf H Klungel, Björn Wettermark, Paul Hjemdahl. (Netherlands)

8:45 AM               

Pharmacoepidemiologic Screening of Potential Oral Anticoagulant Drug Interactions and Thromboembolic Events [420]

Meijia Zhou, Charles E. Leonard, Colleen M. Brensinger, Warren B. Bilker, Stephen E. Kimmel, Todd E. Hecht, Sean Hennessy. (United States)

9:00 AM               

Non-Vitamin K Antagonist Oral Anticoagulants and Angioedema: A Cohort and Case-Crossover Study [421]

John Connolly, Sebastian Schneeweiss, Robert J Glynn, Joshua J Gagne. (United States)

9:15 AM               

Exposure to Dicloxacillin and Flucloxacillin during Warfarin Use and Risk of Ischemic Stroke and Systemic Embolism [422]

Maja Hellfritzsch, Lars Christian Lund, Zandra Ennis, Tore B Stage, Mette Bliddal, Peter B Jensen, Daniel P Henriksen, Martin T Ernst, Morten S Olesen, Anne Broe, Kasper B Kristensen, Per Damkier, Jesper Hallas, Anton Pottegård. (Denmark)

Moderators: Tania Schink & Anne-Marie Meyer

8:00 AM               

Comparative Performance of CIDACS-RL Using Administrative Data Sources [423]

George C Barbosa, Mohamed S Ali, Bruno Araujo, Maria Y Ichihara, Sandra Reis, Samila Sena, Julia M Pescarini, Rosemeire L Fiaccone, Leila D Amorim, Robespierre Pita, Marcos E Barreto, Liam Smeeth, Mauricio L Barreto. (Brazil)

8:15 AM               

Swedish National Registries versus Electronic Medical Records: Comparing the Completeness of Systemic Therapy Data Using Data from the Scan-Leaf Study  [424]

Pia Horvat, Anne Mette-Kejs, Quratul Ann, Valeria Lascano, Benjamin Bray, Dony Patel, Simon Ekman, Laure Lacoin, Ariadna Juarez-Garcia, Joseph Kim. (United Kingdom)

8:30 AM               

Validation of Diagnostic Algorithms to Detect Type 1 Diabetes Mellitus Disease Using Administrative Data from a General Population from Southern Italy [425]

Ylenia Ingrasciotta, Andrea Fontana, Valentina Ientile, Salvatore Crisafulli, Maria Paola Bertuccio, Maurizio Pastorello, Vincenzo Provenzano, Alessandro Scorsone, Gianluca Trifirò. (Italy)

8:45 AM               

Validity of Claims-Based Definitions for Rheumatoid Arthritis, Selected Cancers and Infectious Diseases in Japan: Results from Validate - J Study [426]

Cynthia de Luise, Haoqian Chen, Edward Nonnenmacher, Naonobu Sugiyama, Ryota Hase, Mitsuyo Kinjo, Daisuke Suzuki, Kayoko Katayama, Takakazu Higuchi, Sadao Jinno, Yoshiya Tanaka, Toshitaka Morishima, Soko Setoguchi. (United States)

9:00 AM               

ICD-9 TO ICD-10 Mapping for Research in Biologics and Biosimilars Using Administrative Healthcare Data [427]

Mengdong He, Adrian J. Ortiz, James Marshall, Aaron B. Mendelsohn, Jeffrey R. Curtis, Charles E. Barr, Catherine M. Lockhart, Seoyoung C. Kim. (United States)

9:15 AM               

External Validation of an Algorithm to Identify Patients with High Data-Completeness in Electronic Health Records for Comparative Effectiveness Research [428]

Joshua K. Lin, Yinzhu Jin, Robert J. Glynn, Sebastian Schneeweiss. (United States)

Moderators: Paul Coplan, FISPE,  & Silvia Alessi-Severini

8:00 AM                

Incidence Rates of and Risk Factors for Opioid Overdoses in New Users of Prescription Opioids among US Medicaid Enrollees: A Cohort Study [429]

Young Hee Nam, Warren Bilker, Francesco DeMayo, Mark Neuman, Sean Hennessy. (United States)

8:15 AM               

Risk of Opioid Overdose Associated with Concomitant Use of Opioids and Muscle Relaxants [430]

Yan Li, Yu-Jung Wei, Joshua Brown, Gary M. Reisfield, Chris Delcher, Almut G. Winterstein. (United States)

8:30 AM               

The Association between Prescription Opioid Use and Bone Fracture: A Self-Controlled Case Series Study [431]

Emily Peach, Fiona Pearce, Janice Fuller, Andrew Cooper, Li-Chia Chen, Roger Knaggs. (United Kingdom)

8:45 AM               

The Proportion of Patients in the United States Receiving Postsurgical Opioids Exceeding Recommended Thresholds Increased between 2006 and 2015 [432]

Jessica C Young, Brooke A Chidgey, Til Stürmer, Michele Jonsson Funk. (United States)

9:00 AM               

Healthcare Utilization Associated with Chronic Opioid Use among Hospitalized Patients [433]

Siyana Kurteva, Daniala Weir, Todd Lee, Michal Abrahamowicz, Robyn Tamblyn. (Canada)

9:15 AM               

High-Risk Opioid Utilization and Mortality among Hemodialysis Patients [434]

Matthew Daubresse, G. Caleb Alexander, Deidra C. Crews, Dorry L. Segev, Mara A. McAdams-DeMarco. (United States)

Moderators: Stanley Edlavitch & Paul Stang, FISPE

8:00 AM               

Baclofen Decreases Alcohol Consumption in Real World Clinical Populations [441]

Christopher T. Rentsch, David A. Fiellin, Kendall J. Bryant, Amy C. Justice, Janet P. Tate. (United Kingdom)

8:15 AM               

Long-Term Effect of Interferon and Glatiramer Acetate in Real-World Settings Use on Multiple Sclerosis Disability Progression: Input of Time-Dependent Propensity Score [442]

Mathilde Lefort, Yohann Foucher, Rémi Lenain, Sandra Vukusic, Gilles Edan, Emmanuelle Leray. (France)

8:30 AM               

Ethnic Inequalities in Prescribing Quality for Patients with Dementia: A Cohort Study Using Electronic Health Records [443]

Mary E Jones, Claudia Cooper, Cini Bhanu, Rosalind Raine, Gillian Manthorpe, Kate Walters, Irene Petersen. (United Kingdom)

8:45 AM               

Nested Case-Control Study of Antidepressant Drug Use and Subdural Hematoma Risk [444]

David Gaist, Luis A García Rodríguez, Stine M Hald, Maja Hellfritzsch, Frantz R Poulsen, Bo Halle, Jesper Hallas, Anton Pottegård. (Denmark)

9:00 AM               

Co-Occurring Neuropsychiatric Diagnoses and Use of Psychotropic Drugs in Children with Autism Spectrum Disorder [445]

Svetlana Skurtveit, Yury Kiselev, Marte Handal, Vidar Hjellvik, Ted Reichborn-Kjennerud, Camilla Stoltenberg, Pål Suren, Alexandra Havdahl. (Norway)

9:15 AM               

Concurrent Use of Neurocognitively-active Medications and Alcohol Increases Risk of Community-Acquired Pneumonia [446]

Christopher T. Rentsch, Janet P. Tate, Kirsha S. Gordon, Kathleen M. Akgün, Supriya Krishnan, Alice Tseng, Julie A. Womack, Maria C. Rodriguez-Barradas, Cynthia L. Gibert, Kendall J. Bryant, E. Jennifer Edelman, Amy C. Justice. (United Kingdom)

In parallel with the global adoption of risk management planning, significant progress has been made in recent years in the area of risk minimization measures and evaluation of their effectiveness. Experts in the field now fully recognize the importance of integrating risk minimization and effectiveness as part of the evaluation of the benefit-risk profile throughout the therapeutic product lifecycle in order to ensure that better safety outcomes are met for the patient. However adoption of such measures has also been accompanied with certain challenges. Examples include how to harmonize the risk minimization programs with regional or country-specific requirements, when should effectiveness be evaluated and what is an acceptable threshold for success. In addition, more and more, it is recognized that evaluation of effectiveness should occur throughout the life cycle of a therapeutic product, and information is to be collected in real-world settings. This information in turn will inform on whether changes are needed to the product label or other terms of marketing, including removal of regulatory requirements.

Considering that 2019 coincides with the 10th anniversary of the global adoption of risk management planning (i.e., ICH E2E) worldwide, in this hot topic session we propose to have 3 speakers each, in 20 minutes, providing their perspective supported with case examples to illustrate real-world challenges and proposed solutions. These talks will be followed with a 30 minute moderated discussion with the audience and a podium discussion with the panelists in order to generate a discussion and ultimately share best practices that can be used by all those who are implicated in the area of risk management planning.

Moderator

• Rania Mouchantaf, PhD,  Manager at the Marketed Health Products Directorate, Health Canada.

 Speakers

• Doris Auth, PharmD

           Doris Auth, Associate Director, Division of Risk Management, Office of Surveillance and Epidemiology/CDER/FDA

10 years of REMS: Evaluation Challenges and Opportunities

• Meredith Smith, PhD, MPA

       Meredith Smith. Global Risk Management Officer, Global Patient Safety, Labeling & Pediatrics,  Amgen, Inc.

Advancing the field of risk minimization through application of implementation science: Current status and future state

• Yola Moride, PhD, FISPE

        Full Professor , Faculty of Pharmacy, Université de Montréal

Evaluation of effectiveness of risk minimization interventions: Methodological landscape and lessons learned

 Panelists

• June Raine, MD, MSC, Director of Vigilance and Risk Management, Medicines and Healthcare products Regulatory Agency UK

• Soonyoung Hahn, PhD, President, Korea Institute of Drug Safety and Risk Management

Open to all participants; lunch available.

• Development:  Marriott/304-305

• Global Development:  Marriott/302-303

• History & Archives: Marriott/308

• Publications & Communications:  Marriott/307

• Public Policy:  Marriott/301

Spotlight Poster Session B:   Benefit Risk Assessment, Communication and Evaluation (BRACE); Databases; Drug Utilization Research; Medical Devices; & Rare Disease

Moderators: CHAIR PENDING

1:00 PM               

Patient-, Disease- and Treatment Related Factors That Impact Values Attached to Drug Effects: A Preferences Study among Patients with Type 2 Diabetes [447]

Sonia Roldan, Douwe Postmus, Sieta De Vries, Peter Mol, Hans Hillege. (Netherlands)

1:15 PM               

Impact of Medicines Regulatory Risk Communications in the United Kingdom on Prescribing and Clinical Outcomes: Systematic Review, Re-Analysis and Meta-Analysis [448]

Daniel R Morales, Bruce Guthrie, Tobias Dreischulte, Chris Weatherburn. (United Kingdom)

1:30 PM               

Risk Minimisation Studies Outcomes and Process Indicators [449]

Esther Artime, Nawab Qizilbash. (Spain)

1:45 PM               

Ivabradine Drug Utilization Study in Five European Countries: A Pass to Assess Effectiveness of Risk-Minimization Measures [450]

Linda Salem, Alexandre Malouvier, Jon Blathford, Elena Rivero-Ferrer, Nicolas Deltour, Emmanuelle Jacquot. (France)

2:00 PM               

Comparative Effectiveness of Risk Mitigation Strategies to Prevent Fetal Exposure to Mycophenolate [451]

Amir Sarayani, Yasser Albogami, Mohannad Elkhider, Juan M. Hincapie-Castillo, Babette A. Brumback, Almut G. Winterstein. (United States)

2:15 PM               

Post-Authorisation Amendments to Additional Risk Minimisation Measure Requirements of Medicines Authorised in the EU: A Cohort Study [452]

Reynold D Francisca, Emna Baba, Christina E Hoeve, Inge M Zomerdijk, Miriam C Sturkenboom, Sabine M Straus. (Netherlands)

Moderators: Philip Hunt

1:00 PM               

Effect of a Novel Pharmacist-Delivered Behavioral Intervention for Patients with Poorly-Controlled Diabetes: The Enhancing Outcomes through Goal Assessment and Generating Engagement in Diabetes Mellitus Pragmatic, Database-Randomized Controlled Trial [453]

Julie Lauffenburger, Roya Ghazinouri, Saira Jan, Sagar Makanji, Christina Ferro, Jennifer Lewey, Eric Wittbrodt, Jessica Lee, Nancy Haff, Constance Fontanet, Niteesh Choudhry. (United States)

1:15 PM               

Reduced Healthcare Utilization in Patients Using Empagliflozin: An Interim Analysis from the Empagliflozin Comparative Effectiveness and Safety (EMPRISE) Study [454]

Mehdi Najafzadeh, Ajinkya Pawar, Anouk Déruaz-Luyet, Kimberly G Brodovicz, Anastasia Ustyugova, Lily G Bessette, Nileesa Gautam, Sebastian Schneeweiss, Elisabetta Patorno. (United States)

1:30 PM                The Effectiveness of Glucagon-Like Peptide-1 Receptor Agonists in Preventing Hospitalizations for Chronic Lower Respiratory Disease [455]

Yasser Albogami, Kenneth Cusi, Michael Daniels, Yu-Jung Jenny Wei, Almut Winterstein. (United States)

1:45 PM               

Risk Factors for Progression to Vision Threatening Diabetic Retinopathy [456]

Brian L. VanderBeek, Yafeng Li, J. Clay Bavinger, Yinxi Yu. (United States)

2:00 PM               

Dipeptidyl Peptidase-4 Inhibitors and Risk of Inflammatory Bowel Disease: Real World Evidence in US Adults [457]

Tiansheng Wang, Jeff Y Yang, John B Buse, Virginia Pate, Huilin Tang, Edward Barnes, Robert S Sandler, Til Stürmer. (United States)

2:15 PM               

Dipeptidyl Peptidase-4 Inhibitors and the Risk of Bullous Pemphigoid among Patients with Type 2 Diabetes: A Population-Based Cohort Study [458]

Antonios Douros, Julie Rouette, Hui Yin, Oriana Hoi Yun Yu, Kristian B. Filion, Laurent Azoulay. (Canada)

Moderators: Catherine Cohet & Patricia Saddier

1:00 PM               

Pneumococcal Vaccine Safety Surveillance Using Three Statistical Methods: Disproportionality Analysis, Tree-Based Scan Statistics, and Empirical Bayes Geometric Mean [483]

Hyesung Lee, Ju Hwan Kim, Ju-Young Shin. (Korea, Republic of)

1:15 PM               

Early Season Self-Controlled Risk Interval Analyses of Guillain-Barré Syndrome Risk Following Influenza Vaccination Using Claims Delay Adjustment [484]

Mao Hu, Silvia Perez-Vilar, Michael Wernecke, Deepa Arya, Jeffrey Kelman, Steve Chu, Tom MaCurdy, Richard Forshee. (United States)

1:30 PM               

Comparative Effectiveness of High-Dose versus Standard-Dose Influenza Vaccine among Patients on Chronic Hemodialysis [485]

Anne M. Butler, J. Bradley Layton, Vikas R. Dharnidharka, John M. Sahrmann, David J. Weber, Leah J. McGrath. (United States)

1:45 PM               

Effect of Age on Relative Effectiveness of High-Dose versus Standard-Dose Influenza Vaccines among US Medicare Beneficiaries Ages 65 Years and Older [486]

Yun Lu, Yoganand Chillarige, Hector Izurieta, Yuqin Wei, Wenjie Xu, Michael Lu, Heng-Meng Sung, Arnstein Lindaas, Michael Wernecke, Thomas MaCurdy, Jeffrey Kelman, Richard A Forshee. (United States)

2:00 PM               

Risk of Pneumonia after Varicella Vaccination [487]

Chia-Hung Liu, Yi-Chen Juan, Yen-Yum Yang, Yi-Chun Yeh, Wan-Ting Huang, K Arnold Chan. (Taiwan)

2:15 PM               

No Increased Risk of Kawasaki Disease among Young Children in 28 Days after PCV13 Vaccination [488]

Meghan A. Baker, Bethany Baer, Martin Kulldorff, Lauren Zichittella, Rebecca Reindel, Sandra DeLuccia, Hana Lipowicz, Katherine Freitas, Robert Jin, W. Katherine Yih. (United States)

Moderators: Daniel Prieto-Alhambra, FISPE, & Mollie Wood

1:00 PM               

Methods to Account for Time-Varying Medication Use When Developing a Model for Patient Prognosis [471]

Romin Pajouheshnia, Noah Schuster, Rolf Groenwold, Linda Peelen. (Netherlands)

1:15 PM               

Using the Reverse Waiting Time Distribution with Random Index Dates to Estimate Prescription Durations with Seasonal Variation [472]

Katrine Bødkergaard Nielsen, Randi Marie Selmer, Jesper Hallas, Lars Jøran Kjerpeseth, Anton Pottegård, Eva Skovlund, Henrik Støvring. (Denmark)

1:30 PM               

Exploring Longitudinal Trajectories to Model Paternal Exposure in Evaluating Risk of Adverse Outcomes in Their Offspring [473]

Minna Vehkala, Ruben Hermans, Silvia Narduzzi, Deborah Layton. (United Kingdom)

1:45 PM               

Using Machine Learning Algorithms to Predict Medication Initiations and Discontinuations [474]

Hoa V Le, Chi Truong. (United States)

2:00 PM               

Assessing Differences in Treatment Effects: Interactions with Propensity Score Splines [475]

David J Graham, Jiemin Liao, Elande Baro, Rongmei Zhang, Michael Wernecke, Yuqin Wei, Mao Hu, Sandia Akhtar, Thomas E MaCurdy, Jeffrey A Kelman, Frank E Harrell. (United States)

2:15 PM               

A Challenge of Real World Data: How to Assign Individuals to a Treatment Strategy When Their Data Are Consistent with Several Treatment Strategies at Baseline [476]

Xabier Garcia de Albeniz, John Hsu, Miguel A Hernan. (Spain)

Moderators: Vera Ehrenstein

1:00 PM               

Development and Validation of Algorithms to Estimate Live Birth Gestational Age in Medicaid Analytic Extract Data [477]

Yanmin Zhu, Thuy N Thai, Brian T Bateman, Sonia Hernandez-Diaz, Jessica M Franklin, Loreen Straub, Almut G Winterstein, Kathryn J Gray, Yandong Qiang, Wei Hua, Lockwood G Taylor, Krista F Huybrechts. (United States)

1:15 PM               

Utility of Prenatal Tests to Estimate Pregnancy Start in Sentinel [478]

Lockwood G Taylor, Steven T Bird, Danijela Stojanovic, Sengwee Toh, Elnara Fazio-Eynullayeva, Andrew Petrone, Jennifer G Lyons. (United States)

1:30 PM               

Estimation of the Beginning of Pregnancies Ending in Stillbirths [479]

Tania Schink, Ulrike Haug. (Germany)

1:45 PM               

Validation of ICD-9-CM Coding Algorithms to Identify Non-Live Birth Outcomes in an Automated Database [480]

Yanmin Zhu, Kathryn Gray, Brian T Bateman, Sonia Hernandez-Diaz, Loreen Straub, Rebecca M Reimers, Beryl Manning-Geist, Elizabeth Yoselevsky, Yandong Qiang, Lockwood G Taylor, Wei Hua, Krista Huybrechts. (United States)

2:00 PM               

Immortal Time Bias in Comparative Drug Safety Studies in Pregnancy [481]

Kristian B Filion, Colin Dormuth, Pauline Reynier, J. Michael Paterson, Robert W Platt, Samy Suissa. (Canada)

2:15 PM               

Comparing the Use of Propensity and Disease Risk Scores in Signal Detection for Prematurity on Medico-Administrative Database [482]

Romain Demailly, Sylvie Escolano, Françoise Haramburu, Pascale Tubert-Bitter, Ismaïl Ahmed. (France)

Methods in Geriatric Pharmacoepidemiology - Moderators: Mary Ritchey, FISPE, & Lisa Pont, FISPE,

1:00 PM               

Identifying Drug Classes Whose Initiation May Be a Proxy for Frailty [459]

Henry T Zhang, Robert J Glynn, Til Stürmer. (United States)

1:05 PM               

Validation of a Five-Year Mortality Prediction Model among US Medicare Beneficiaries [460]

Rachael K Ross, Tzy-Mey Kuo, Michele Jonsson Funk, Jennifer L Lund. (United States)

1:10 PM               

The Impact of Polypharmacy Definitions on the Estimation of Polypharmacy Prevalence in Older Individuals: A Population-Based Evaluation [461]

Marie-Eve Gagnon, Houssem Missaoui, Bernard Candas, Guillaume Boucher, Marc Simard, Caroline Sirois. (Canada)

1:15 PM               

Measuring Frailty in Administrative Claims Data: Comparative Performance of Four Claims-Based Frailty Measures in the United States Medicare Data [462]

Dae Kim, Elisabetta Patorno, Ajinkya Pawar, Hemin Lee, Sebastian Schneeweiss, Robert Glynn. (United States)

1:20PM               

Panel Discusssion: Methods in Geriatric Pharmacoepidemiology

Machine Learning for Risk Prediction - Moderators: Mary Ritchey, FISPE, & Olaf Klungel, FISPE

1:30 PM               

Predictive Modeling in Patients with Heart Failure: A Comparison of Machine Learning and Traditional Statistical Approaches [463]

Rishi J Desai, Shirley Wang, Muthiah Vaduganathan, Sebastian Schneeweiss. (United States)

1:35 PM               

Performance of Machine Learning Algorithms for Hysterectomy Risk Prediction among Women with Endometriosis in the United States [464]

Bradley Saul, Ryan D. Kilpatrick, Leah J. McGrath, Diane Reams, Lani Wegrzyn, Stephanie E. Chiuve, M. Alan Brookhart. (United States)

1:40 PM               

Inferring Mortality in Claims Data [465]

Dana Y Teltsch, Feng Zhang, Amanda M Kelly, Dawn G Albright. (United States)

1:45 PM               

Federated Learning for Predicting Clinical Events with Distributed Patient Data Sets [466]

Yoonyoung Park, Issa Sylla, Amar K Das, Olivia Choudhury. (United States)

1:50pm                 

Panel Discusssion: Machine Learning for Risk Prediction

Opioid Method Research - Moderators: Mary Ritchey, FISPE, & Vin Lo Re, FISPE

2:00 PM               

Using Machine Learning to Predict Risk of Opioid Overdose in Medicare [467]

Weihsuan Jenny Lo-Ciganic, James L Huang, Hao H Zhang, Jeremy C Weiss, Yonghui Wu, Chian K Kwoh, Julie M Donohue, Jerry Cochran, Adam J Gordon, Daniel C Malone, Courtney C Kuza, Walid F Gellad. (United States)

2:05 PM               

Dual-Trajectories of Opioid and Gabapentinoid Use and Risk of Subsequent Drug Overdose among United States Medicare Beneficiaries [468]

Lili Zhou MS, Sandipan Bhattacharjee PhD, C. Kent Kwoh MD, Patrick J. Tighe MD, Gary M. Reisfield MD, Daniel C. Malone PhD, Marion Slack PhD, Wei-Hsuan Jenny Lo-Ciganic PhD. (United States)

2:10 PM               

Estimates of Prolonged Opioid Use Following Surgery Vary by Orders of Magnitude across Various Definitions [469]

Jessica C Young, Jennifer M Wu, Virginia Pate, Michele Jonsson Funk. (United States)

2:15 PM               

Concurrent Use of Opioids and Strong Metabolic Inhibitors and the Risk of Serious Infections [470]

Andrew D Wiese, Marie R Griffin, C. Michael Stein, William Schaffner, Robert A Greevy, Edward F Mitchel, Carlos G Grijalva. (United States)

2:20pm                 

Panel Discusssion: Opioid Methods Research

Replicating Clinical Trial Data Using RWD:  Ballroom B

Data Diversity in Multi-Database Pharmacoepidemiologic Studies and Its Role in Outcome Misclassification: A Curse or a Blessing?:  Room 201 A

Characterizing Patient Experience Data in Medical Product Development: What Is It, What Is Expected, and How Can You Contribute Based on Anticipated Regulatory Guidance?  Room 201 B

Existing Databases for Drug Utilization Research in Latin American and African Countries:  Room 201 C

Introducing Lactation Real World Data to ISPE:  Room 202 AB

• Academic Council:  Room 202 AB
• Government/Regulatory Council:  Room 106 AB
• Industry/Service Provider Council:  Room 204 C
• Student Council:  Room 107 B

Join your colleagues for an evening of food, music, networking and dancing.
The Kimmel Center  300 S. Broad Street is a 10-minute walk from the Philadelphia Marriott Downtown

Please arrive 24 hours before your presentation start time to upload or check your presentation. 

Open to all participants

Moderator: Douglas Steinke

8:30 AM               

Treatment Patterns of Checkpoint Inhibitors in Metastatic Melanoma [817]

Marina A Machado, Cristiano S Moura, Jeffrey R Curtis, Marie Hudson, Sasha Bernatsky. (Canada)

8:45 AM               

Measuring Treatment Intensification in Patients with Type 2 Diabete: An Electronic Health Records Based Study [818]

Robert J Romanelli, Zachary A Marcum, Qiwen Huang, Jakob S Knudsen, Dominick L Frosch. (United States)

9:00 AM               

Trends in Blood Pressure Thresholds for Initiation of Antihypertensive Treatment in Patients with Type 2 Diabetes [819]

Martina Ambroz, Sieta T deVries, Grigory Sidorenkov, Klaas Hoogenberg, Petra Denig. (Netherlands)

9:15 AM               

Incidence of Direct Oral Anticoagulation Use for Non Valvular Atrial Fibrillation and Characteristics of Users in Six European Countries (2008-2015): A Cross-National Drug Utilization Study [820]

Luisa Ibanez, Mònica Sabaté, Xavier Vidal, Elena Ballarín, Marietta Rottenkolber, Sven Schmiedl, Andreas Heeke, Consuelo Huerta, Elisa Martin Merino, Dolores Montero, Luz M Leon-Muñoz, Christiane Gasse, Nicholas Moore, Cécile Droz, Régis Lassalle, Mia Aakjaer, Morten Andersen, Marie Louise De Bruin, Rolf Groenwold, Rianne Van den Ham, Patrick Souverein, Olaf Klungel, Helga Gardarsdottir. (Spain)

9:30 AM               

Assessment of the Calcium Channel Blocker, Lower Extremity Edema, Loop Diuretic Prescribing Cascade: A Prescription Sequence Symmetry Analysis [821]

Scott M Vouri, Xinji Jiang, Carl Pepine, Laurence Solberg, Daniel C. Malone, Almut Winterstein. (United States)

9:45 AM               

Appropriate Prescribing in Nursing Homes Demonstration Project: A Pragmatic, Cluster-Randomized Trial [822]

Mina Tadrous, Kinwah Fung, Laura Desveaux, Tara Gomes, Monica Taljaard, Noah Ivers. (Canada)

Moderator: John Seeger, FISPE

8:30 AM               

Staying on Treatment Matters: Estimating Effects of Dabigatran vs Warfarin in Medicare [841]

Michael Webster-Clark, Til Stürmer, Charles Poole, Ross Simpson, Jessie K Edwards, Jennifer L Lund. (United States)

8:45 AM               

Comparative Effectiveness and Safety of Concomitant Use of Antiplatelets and Anticoagulants in Acute Coronary Syndrome Patients with Atrial Fibrillation [842]

Ghadeer K. Dawwas, Eric Dietrich, David Winchester, Almut Winterstein, Richard Segal, Haesuk Park. (United States)

9:00 AM               

Safety and Effectiveness of Direct Oral Anticoagulants versus Vitamin K Antagonists, Results from Three Italian Regions [843]

Ursula Kirchmayer, Silvia Narduzzi, Flavia Mayer, Marco Tuccori, Olivia Leoni, Valeria Belleudi, Antonio Addis, Marina Davoli. (Italy)

9:15 AM               

Comparative Effectiveness and Safety of Direct Oral Anticoagulants in Non-Valvular Atrial Fibrillation: A Multi-Center Observational Cohort Study [844]

Madeleine Durand, Mireille E Schnitzer, Menglan Pang, Greg Carney, Sherif Eltonsy, Kristian B. Filion, Anat Fisher, Min Jun, I fan Kuo, Alexis Matteau, J. Michael Paterson, Jacqueline Quail, Christel Renoux, for the CNODES Investigators. (Canada)

9:30 AM               

Comparative Effectiveness and Safety of Direct Acting Oral Anticoagulants and Warfarin in Cancer Patients with Venous Thromboembolism [845]

Ghadeer K. Dawwas, Eric Dietrich, Kyle Davis, Haesuk Park. (United States)

9:45 AM               

Risk of Major Bleeding Associated with the Use of Individual Direct Oral Anticoagulants Compared to Vitamin K Antagonists in Patients with Non-Valvular Atrial Fibrillation: A Meta-Analysis of Results from Multiple Population-Based Cohort Studies Using a Common Protocol in Europe and Canada [846]

Hendrika van den Ham, Patrick Souverein, Olaf Klungel, Robert Platt, Pierre Ernst, Sophie Dell’Aniello, Sven Schmiedl, Birgit Grave, Marietta Rottenkolber, Consuelo Huerta, Elisa Martin Merino, Luz León-Muñoz, Luz León-Muñoz, Dolores Montero, Morten Andersen, Mia Aakjær, Marieke De Bruin, Helga Gardarsdottir. (Netherlands)

Moderators: Jennita Reefhuis & Mette Norgaard

8:30 AM               

Concomitant Use of Quinolones and Stimulants and the Risk of Cardiovascular Adverse Events: A Comparative Safety Study [835]

Adel A. Alrwisan, Yu-Jung Wei, Patrick Antonelli, Babette Brumback, Almut Winterstein. (Saudi Arabia)

8:45 AM               

An Unnecessary Risk: Lack of Benefit of Fluoroquinolone Antibiotics in Uncomplicated Exacerbations of Chronic Obstructive Pulmonary Disease [836]

Pierre Ernst, Matthew Dahl, Dan Chateau, Nick Daneman, Jacqueline Quail, Ingrid S. Sketris, Anat Fisher, Jianguo Zhang, Shawn Bugden, for the CNODES Investigators. (Canada)

9:00 AM               

Excess Risk of Tendon Rupture Due to Fluoroquinolones with and without Concomitant Corticosteroid Use [837]

Rebecca Persson, Susan Jick. (United States)

9:15 AM               

Early Childhood Antibiotics Use and the Risk of Attention-Deficit/Hyperactivity Disorder: A Population-Based Cohort Study [838]

Amani Hamad, Silvia Alessi-Severini, Salah Mahmud, Marni Brownell, I fan Kuo. (Canada)

9:30 AM               

Comparative Safety and Cost Assessment of Prescribing Systemic Steroids for Acute Respiratory Tract Infections [839]

Kueiyu Joshua J Lin, Sebastian Schneeweiss, Evan Dvorin, Mehdi Najafzadeh, Aaron S Kesselheim. (United States)

9:45 AM               

Antibiotic Use and Colorectal Cancer Recurrence and Mortality: A Danish Nationwide Prospective Cohort [840]

Veronika Fedirko, Deirdre Cronin-Fenton, Anders Hammerich Riis, Henrik Toft Sørensen, Timothy L. Lash. (United States)

Moderators: Nicolle Gatto, FISPE, & Tobias Gerhard, FISPE

8:30 AM               

Evaluation of Bias in Case-Crossover Analyses of Persistent Drug Exposures [829]

Katsiaryna Bykov, Shirley V. Wang, Joshua J. Gagne. (United States)

8:45 AM               

Empirical Assessment of Case-Based Methods for the Identification of Drug-Related Health Outcome of Interest in the French Nationwide Healthcare Database (SNDS) [830]

Nicolas Thurin, Régis Lassalle, Patrick Blin, Marine Pénichon, Martijn Schuemie, Joshua J Gagne, Jeremy A Rassen, Jacques Benichou, Alain Weill, Cécile Droz-Perroteau, Nicholas Moore. (France)

9:00 AM               

Using Active Comparators in Self-Controlled Studies [831]

Jesper Hallas, Suzanne Cadarette, Nicole Pratt, Joseph A Delaney, Heather Whitaker, Malcolm Maclure. (Denmark)

9:15 AM               

Development and Application of a Hybrid Matching Algorithm to Refine the Prevalent New-User Cohort Design for Comparative Drug Effect Studies [832]

Chenyi Yang, Shihchen Kuo, Chun-Ting Yang, Edward Chia-Cheng Lai, Huang-Tz Ou. (Taiwan)

9:30 AM               

Classes and Prevalence of Prevalent New Users: An Example in Medicare [833]

Michael Webster-Clark, Til Stürmer, Jessie K Edwards, Ross Simpson, Charles Poole, Jennifer L Lund. (United States)

9:45 AM               

Comparative Performance of Trend-in-Trend Design and Instrumental Variable Methods in an Active Comparator New User Setting [834]

Phyo T Htoo, Michele Jonsson-Funk, Gang Fang, Virginia Pate, John Buse, Jessie K Edwards, Nabarun Dasgupta, Til Stürmer. (United States)

Moderator: Ju-Young Shin

8:30 AM               

Impact of the Publicly Funded Herpes Zoster Immunization Program on Burden of Disease in Ontario, Canada [847]

Mina Tadrous, Daniel McCormack, Diana Martins, Tara Gomes, Jeff Kwong, Sarah Buchan, Tony Antoniou. (Canada)

8:45 AM               

Trends in Human Papillomavirus Vaccination Uptake in Girls and Boys in the United States: Real-World Evidence from 2003 to 2016 [848]

Szu-Ta Chen, Krista F. Huybrechts, Brian T. Bateman, Sonia Hernández-Díaz. (United States)

9:00 AM               

Vaccination before First Symptom of Central Demyelination  [849]

Lamiae Grimaldi-Bensouda, Nicolas Morisot, Pegdwende O Dialla, Clémentine Nordon, Lucien Abenhaim. (France)

9:15 AM               

Incidence of Outcomes Relevant to Vaccine Safety Monitoring in Europe Using Two Distinct Data Sources: National Healthcare Registries (Sweden) and Electronic Medical Records (UK) [850]

Lina Titievsky, Nils Ekstrom, James Stark, Michael Törnblom, Dan Scott, Rob Maroko, Cassandra Hall-Murray, Peter McMahon, Leif Jörgensen, Mats Rosenlund. (United States)

9:30 AM               

Advance: Towards Near-Real Time Monitoring of Vaccination Coverage, Benefits and Risks Using European Electronic Health Record Databases [851]

Kaatje Bollaerts, Tom De Smedt, Chris McGee, Hanne-Dorthe Emborg, Maria Alexandridou, Marco Villa, Talita Duarte-Salles, Myint Tin Tin Htar, Simon de Lusignan, Claudia Bartolini, Rosa Gini, Lina Titievsky, Miriam Sturkenboom, Vincent Bauchau. (Belgium)

9:45 AM               

Variation in Rotavirus Vaccination Coding in US State Medicaid Data [852]

J. Bradley Layton, Anne M Butler, M. Alan Brookhart, Catherine A. Panozzo. (United States)

Moderators: Aaron Kamauu & Andres Gomez-Caminero

8:30 AM               

Identifying Signals of Warfarin Drug-Drug Interactions: A Translational Biomedical Informatics Approach Using Real World Clinical Evidence [823]

Meijia Zhou, Lei Wang, Charles E. Leonard, Colleen M. Brensinger, Warren B. Bilker, Stephen E. Kimmel, Todd E. Hecht, Lang Li, Sean Hennessy. (United States)

8:45 AM               

Drug Safety Signal Detection in a Chinese Regional Healthcare Database Using the Tree-Based Scan Statistic and Comparison to 3 Other Statistical Methods [824]

Hailong Li, Lingli Zhang, Siyan Zhan. (China)

9:00 AM               

Contribution of Natural Language Processing in Predicting Risk of Pleural and Pericardial Effusions in Small Cell Lung Cancer by Line of Therapy [825]

Rui Jiang, Ravi Potluri. (United States)

9:15 AM               

Text Mining to Identify Drug-Drug Interactions (DDIS) in Elderly Patients Admitted to Emergency Department [826]

Iris Pujade, Perrine Duthoit, Grégoire Evrard, Alicia Bel-Létoile, Clément Goehrs, Vianney Jouhet, Sébastien Cossin, Antoine Pariente, Frantz Thiessard, Francesco Salvo, Louis Letinier. (France)

9:30 AM               

Nationwide Adverse Drug Reaction Screening Using High Process Computing [827]

Te-yuan Chyou, Prasad S Nishtala. (New Zealand)

9:45 AM               

Detecting Rare Diseases: A Case Study Using Machine Learning Semi-Supervised Networks to Identify Under-Diagnosed Patients [828]

Yunlong Wang, Christina Mack, Fan Zhang, Yong Cai, Emily Zhao. (United States)

10:30 AM            

Analysis of Data from Distributed Pharmacoepidemiologic Networks [853]:  Ballroom B

Romin Pajouheshnia, Helga Gardarsdottir, Robert Platt, Darren Toh, Olaf Klungel. (Netherlands)

10:30 AM            

Causal Inference in Pharmacoepidemiology [854]:  Room 201 A

Laura Pazzagli, Elizabeth A. Stuart, Miguel Hernán, Sebastian Schneeweiss. (Sweden)

10:30 AM            

Pharmacoepidemiology and Rare Cancer Research [855]:  Room 201 B

Wei Zhou, Leigh Marcus, Anton Pottegård, Bruce Carleton, Andrew Freedman, Hans-Georg Eichler. (United States)

10:30 AM            

Emerging Solutions to Addressing Challenges in Measuring Adherence to Multiple Medications [856]: Room 201 C

Julie Lauffenburger, Caroline Walsh, Sarah-Jo Sinnott, Jessica Franklin, Petra Denig. (United States)

10:30 AM            

Mixing It Up: Implementing Qualitative & Mixed Methods in Pharmacoepidemiology Research [857]:  Room 202 AB

1. Elle Saine, Julia E Szymczak, Irene Petersen, Rachel E Sobel. (United States)

Open to ISPE members; members may contact info@pharmacoepi.org if they would like to attend. 

Moderator: Aaron Winn

1:30 PM               

Pharmacy-Based Predictors of Non-Adherence, Non-Persistence, and Re-Initiation of Antihypertensive Drugs among Patients on Oral Diabetes Drugs in the Netherlands [876]

Sofa Dewi Alfian, Petra Denig, André Coelho, Eelko Hak. (Netherlands)

1:45 PM               

The Social Determinants of Non-Adherence to Antihypertensive Medications [877]

Macarius Donneyong, Paul Juarez, Teng-Jen Chang, Shawnita Sealy-Jefferson, Pamela Salsberry, Bo Lu, Wansoo Im, John Jackson, Michael Fischer. (United States)

2:00 PM               

Increased Out-of-Pocket Costs Lead to Decreased Adherence and Delayed Treatment Reinitiation of Branded Diabetes Drugs in Medicare Patients [878]

Danielle S Chun, Mugdha Gokhale, Stacie B Dusetzina, Virginia Pate, John B Buse, Emily Gower, Til Stürmer. (United States)

2:15 PM               

Impact of a Two-Tier Intervention on Statin Prescribing and Adherence Levels Among Latino Patients with Diabetes [83]

Toney Duong, Jose Hernandez. (United States)

2:30 PM               

Does Nonadherence with Antidepressants Differ by Treatment Indication? [880]

Jenna Wong, Siyana Kurteva, Robyn Tamblyn. (United States)

2:45 PM               

A Group Based Trajectory Modeling to Study Adherence Patterns of Atypical Antipsychotics in Pediatrics and Adolescents: A Retrospective Cohort Study Using Texas Children Health Plan [881]

Swarnava Sanyal, Tyler J Varisco, Soham Yande, Hua Chen. (United States)

Moderators: Jodi Segal, FISPE, & Christian Hampp, FISPE

1:30 PM               

Improved Real-World Outcomes for Type 2 Diabetes Patients on Basal-Bolus Insulin Therapy Switching to Insulin Glargine 300 U/ml vs. Insulin Glargine 100 U/ml and Insulin Detemir [858]

Jasmanda Wu, Timothy S. Bailey, Zsolt Bosnyak, Jukka Westerbacka, Rishab A. Gupta, Arjun A. Menon, Charlie Nicholls, Lawrence Blonde. (United States)

1:45 PM               

Metformin Use and Risk of Benign Prostatic Hyperplasia [859]:  

Mette Nørgaard, Bianka Darvalics, Reimar W Thomsen. (Denmark)

2:00 PM               

Comparative Cardiovascular and Hypoglycemic Safety of Glimepiride in Type 2 Diabetes: A Population-Based Cohort Study [860]

Antonios Douros, Sophie Dell’Aniello, Oriana Yu, Samy Suissa. (Canada)

2:15 PM               

Major Adverse Cardiovascular Events Associated with Sodium Glucose Co-Transporters versus Other Antidiabetes Drugs: Population-Based Cohort Study [861]

Colin Dormuth, Xiaoting Ding, Ellen Reynolds, Greg Carney. (Canada)

2:30 PM               

Comparative Risk Evaluation for Cardiovascular Events Associated with Empagliflozin vs. Dapagliflozin: A Multi-Institutional Cohort Study [862]

Shih-Chieh Shao, Kai-Cheng Chang, Ming-Jui Hung, Yuk-Ying Chan, Hui-Yu Chen, Yea-Huei Kao Yang, Edward Chia-Cheng Lai. (Taiwan)

2:45 PM               

Empagliflozin Selected Cardiovascular and Safety Outcomes in Routine Care: First Results from Theempagliflozin Comparative Effectiveness and Safety (EMPRISE) Study [863]

Elisabetta Patorno, Ajinkya Pawar, Jessica M Franklin, Mehdi Najafzadeh, Anouk Déruaz-Luyet, Kimberly G Brodovicz, Steven Sambevski, Lily G Bessette, Martin Kulldorff, Sebastian Schneeweiss. (United States)

Moderator: Amanda Golembesky

1:30 PM               

Real-World Data on the Use of Biologics in the Treatment of Colorectal Cancer: Comparison between Three European Databases [864]

Katja Anita Oppelt, Josephina G. Kuiper, Ylenia Ingrasciotta, Valentina Ientile, Ron M. Herings, Gianluca Trifirò, Ulrike Haug. (Germany)

1:45 PM               

Drug Survival of Targeted Therapies for Plaque Psoriasis: Evidence from a Large U.S. Claims Database [865]

Nathaniel Hendrix, David Veenstra. (United States)

2:00 PM               

Risk of Serious Infections among New Users of Interleukin-17, Interleukin-12/23 Inhibitors, Compared to Tumor Necrosis Factor (TNF)-alpha Inhibitors, for the Treatment of Psoriasis and Psoriatic Arthritis [866]

Xintong Li, Kathleen M Andersen, Hsien-Yen Chang, Jeffrey R. Curtis, G. Caleb Alexander. (United States)

2:15 PM               

Risk of Heart Failure Following Exposure to Non-TNFi Compared to TNFi Biologics in US Patients with Rheumatoid Arthritis [867]

Thomas Bo Jensen, Nicole Tsao, Ajinkya Pawar, Rishi Desai, Seoyoung Kim. (United States)

2:30 PM               

Risk of Diabetes Treatment Intensification Associated with Use of Abatacept versus Other Biologic Drugs in Patients with Rheumatoid Arthritis and Diabetes Mellitus [868]

Sarah Chen, Hemin Lee, Yinzhu Jin, Jun Liu, Seoyoung Kim. (United States)

2:45 PM               

Intravitreal Anti-Vascular Endothelial Growth Factor Drugs and Risk of Serious Non-Ocular Hemorrhage [869]

Janet Sultana, Francesco Giorgianni, Giulia Scondotto, Valentina Ientile, Pasquale Cananzi, Olivia Leoni, Sebastiano Walter Pollina Addario, Giovanbattista de Sarro, Adele de Francesco, Maria Rosalia Puzo, Christel Renoux, Gianluca Trifiro'. (Italy)

Moderators: Jennifer Lund & Thierry Trenque

1:30 PM               

Heatwaves and Heat-Sensitizing Medications in Vulnerable Older Adults [870]

J. Bradley Layton, Wenhong Li, Lily Wang, Jiacan Yuan, Joshua P Gilman, Daniel B Horton, Soko Setoguchi. (United States)

1:45 PM               

Opioid Intensification versus Adjuvant Gabapentin Initiation and Risk of Hospitalization for Falls and Fractures in United States Nursing Home Residents [871]

Danni Zhao, Anne Hume, Shao-Hsien Liu, Christine Ulbricht, Matt Alcusky, Giovanni Gambassi, Kate L Lapane. (United States)

2:00 PM               

Comparative Safety and Effectiveness of Apixaban and Rivaroxaban versus Warfarin among US Nursing Home Residents [872]

Matthew Alcusky, David D McManus, Marc Fisher, Anne L Hume, Jennifer Tjia, Kate L Lapane. (United States)

2:15 PM               

Statin Use in Older Danes: An Analysis of Discontinuation Patterns  [873]

Wade Thompson, Dorte E Jarbøl, Peter Haastrup, Jesper Bo Nielsen, Anton Pottegård. (Denmark)

2:30 PM               

Does Use of Acetylcholinesterase Inhibitors Prevent or Delay the Prescribing of Psychotropic Medications in People with Dementia? Analyses of the Swedish Dementia Registry (Svedem) [874]

Edwin C Tan, Kristina Johnell, J Simon Bell, Sara Garcia-Ptacek, Johan Fastbom, Peter Nordström, Maria Eriksdotter. (Australia)

2:45 PM               

Association Rule Analysis to Evaluate Frequent Drug Combinations Associated with Acute Kidney Injury in Older Adults [875]

Prasad Nishtala, Te-yuan Chyou. (United Kingdom)

Moderator: David Miller, FISPE

1:30 PM               

Current Practice in Reporting Exposure in Pharmacoepidemiological Studies [882]

Mirjam Hempenius, Kim Luijken, Anthonius Boer, Olaf H Klungel, Rolf H Groenwold, Helga Gardarsdottir. (Netherlands)

1:45 PM               

Cause-Specific and All-Cause Mortality Associated with Proton Pump Inhibitors: A Cohort Study [883]

Jeremy P Brown, John Tazare, Kathryn Mansfield, Kevin Wing, Adrian Root, Laurie Tomlinson, Krishnan Bhaskaran, Elizabeth Williamson, Liam Smeeth, Ian Douglas. (United Kingdom)

2:00 PM               

Survival after Stevens-Johnson Syndrome: A UK-Based Cohort Study [884]

Carole Marxer, Noel Frey, Michael Bodmer, Andreas Bircher, Susan S. Jick, Christoph R. Meier, Julia Spoendlin. (Switzerland)

2:15 PM               

Real World Effects of Medications for Chronic Obstructive Pulmonary Disease: A UK Population-Based Non-Interventional Cohort Study with Validation against Randomised Trial Results [885]

Ian Douglas, Kevin Wing, Elizabeth Williamson, James Carpenter, Lesley Wise, Sebastian Schneeweiss, Liam Smeeth, Jenni Quint,  (United Kingdom)

2:30 PM               

Safety of Colobreathe: Findings from the UK Cystic Fibrosis Registry [886]

Sigal Kaplan, Diana Bilton, Susan C Charman, Andrew Lee, Noreen Caine. (Israel)

2:45 PM               

Where Should I Submit My Manuscript? Applying Machine Learning to Enhance Communication of Medical Research [887]

Jeremy Rassen. (United States)

Moderator: Soko Setoguchi, FISPE

1:30 PM               

Propensity Score Stratification and Inverse Probability Weighting Compared to Surgical Randomised Clinical Trial Results in Device Epidemiology [888]

Victoria Y Strauss, Albert Prats-Uribe, Klara Berencsi, Andrew Carr, Andrew Judge, Alan Silman, David W Murray, Nigel K Arden, Irene Petersen, Rafael Pinedo-Villanueva, David J Beard, J Mark Wilkinson, Ian Douglas, Jose M Valderas, Sarah Lamb, Daniel Prieto-Alhambra. (United Kingdom)

1:45 PM               

The Performance of Preference-Based Instrumental Variables to Emulate a Randomised Clinical Trial of Comparative Medical Device Effectiveness [889]

Albert Prats-Uribe, Klara Berencsi, Andrew Carr, Andrew Judge, Alan Silman, David W Murray, Nigel K Arden, Irene Petersen, Rafael Pinedo-Villanueva, David J Beard, J Mark Wilkinson, Ian Douglas, Jose M Valderas, Sarah Lamb, Victoria Y Strauss, Daniel Prieto-Alhambra. (United Kingdom)

2:00 PM               

Development and Validation of Patient-Level Prediction Models for Adverse Outcomes Following Total Knee Arthroplasty in Osteoarthritic Patients [890]

Ross Williams, Jenna Reps, Ying HE, Peter Rijnbeek, Anthony G Sena, Daniel Prieto-Alhambra, Patrick Ryan. (Netherlands)

2:15 PM               

Medications, Device Use, and Non-Device Associated Urinary Tract Infections in Hospitalized Patients [891]

Paula D Strassle, Emily E Sickbert-Bennett, Lauren M DiBiase, Jennifer L Lund, Michael Klompas, Paul Stewart, David J Weber. (United States)

2:30 PM               

Cardinality Matching versus Propensity Score Matching for Causal Inference in Medical Device Research: An Applied Analysis of Minimally-Invasive versus Open Elective Thoracic Segmentectomy [892]

Stephen Johnston, Iftekhar Kalsekar, David Wei, Annie Lied-Lied, Jose Zubizarreta. (United States)

2:45 PM               

Self Controlled Case Series in Surgical Epidemiology: Checking the Underlying Assumptions in an Empirical Example [893]

Danielle E Robinson, Ian Douglas, Victoria Y Strauss, Daniel Prieto-Alhambra. (United Kingdom)

Uncontrolled study arms in Phase II/III trials and long-term trial extensions provide an efficient mechanism to gain efficacy and safety data.  They may also avoid ethical issues in the use of long-term placebo, feasibility or cost issues around choice of a relevant, effective and safe comparator, while meeting the need for sufficient exposure data for regulatory filing. 

While uncontrolled studies offer benefits, there are also risks. The lack of an internal control group poses challenges to the evaluation and interpretation of any unexpected serious adverse events (SAEs) that may occur. The occurrence of even a small number of SAEs, or otherwise clinically important AEs, may engender enough uncertainty with respect to the risk/benefit profile that, additional data may be required pre-submission, lead to cautionary labeling, or prompt further post-marketing study requirements.

This Hot Topics Session will cover issues related to uncontrolled, pre-authorization product development trials and extensions to randomized controlled trials. The session will propose the use of observational, real-world evidence (RWE) patient cohorts as a potential method to provide an external control group for these Phase II/III uncontrolled, clinical trials and/or open-label, long-term extensions that may mitigate some of the risks. The 20-minute presentations and 30-minute discussion will include:

1. scope of the use of single-arm, uncontrolled trials in product development and examples where uncontrolled arms have posed a regulatory review issue; 
2. methodologic and analytic challenges of using external control cohorts as comparators for safety and/or effectiveness for uncontrolled trials and/or trial extensions, including types of databases, collection of data, analytic issues and ability to control for confounding; and
3. challenges in interpreting uncontrolled trial data in regulatory filings and regulatory perspectives on use of real world data as external controls to assist in regulatory decision–marking. 

Moderators

• Nancy Santanello, FISPE, Pharmacoepidemiology Consultant

• Barry Gertz, Managing Director, Blackstone Life Sciences

Speakers

• Joanne Waldstreicher, Chief Medical Officer, Johnson and Johnson, Raritan, NJ

• Peter Stein, Director, Center for Drug Evaluation and Research (CDER), Office of New Drugs (OND), FDA, Silver Spring, MD

• Stephen Evans, FISPE, Professor of Pharmacoepidemiology, London School of Hygiene and Tropical Medicine, London, UK

• Vincent Lo Re, FISPE, Chair, ICPE 2019 Scientific Program Committee

  • Review of Abstract Admission Categories
    • Vera Ehrenstein
    • Daniel B Horton
    • Elaine Morrato
    • Mary Ritchey, FISPE
    • Rachel Sobel, FISPE
    • Walter Straus
  • Newcomer Track
    • Charles Leonard
    • Ian Douglas
  • Moderator Subcommittee
    • Mary Ritchey, FISPE
    • Krista Huybrechts, FISPE
  • Lisa Pont, FISPE
  • Spotlight Poster Sessions
  • J. Bradley Layton
  • Jennifer Lund

Spotlight Poster Awards

1. Bradley Layton, Chair, Spotlight Poster Sessions

2019 Meetings

Mid-Year Meeting – Orlando

Juan Hincapie-Castillo, 2019 Mid-Year Program Planning Committee

36^th ICPE – Berlin

            Tobias Gerhard, FISPE, Chair ICPE 2020 Scientific Program Committee

            Frank Andersohn, Chair, ICPE 2020 Local Host Committee

Drawing for ICPE 2020 Registrations

                        Tobias Gerhard, FISPE, Chair ICPE 2020 Scientific Program Committee

Adjournment of the ICPE 2019

          Vincent Lo Re, FISPE, Chair, ICPE 2019 Scientific Program Committee