strategic initiatives

RWE for Regulatory Decision Making

ISPE Statement on Real-World Evidence (RWE)

ISPE RWE-related Guidance Documents and Policy Statements

 

Relevant RWE Guidance documents from Health Authorities

Relevant RWE Guidance documents from Health Authorities

Region

Health Authority

Documents and links

North America

Food & Drug Administration (FDA)
  • Guidance: Submitting Documents Utilizing Real-World Data and Real-World Evidence to FDA for Drugs and Biologics
  • Guidance: Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices
  • Guidance: Use of Electronic Health Records in Clinical Investigations
  • Framework for FDA’s Real-World Evidence Program

https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence

  • Prescription Drug User Fee Amendments (PDUFA VI)

https://www.fda.gov/industry/prescription-drug-user-fee-amendments/pdufa-vi-fiscal-years-2018-2022

  • 21st Century Cures Act

https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/21st-century-cures-act

 

Health Canada
  • Elements of Real World Data/Evidence Quality throughout the Prescription Drug Product Life Cycle
  • RWE for Drugs Project
  • Overall R2D2 project

https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/announcements/optimizing-real-world-evidence-regulatory-decisions.html

 

Canadian Agency for Drugs and Technologies in Health (CADTH)

https://www.cadth.ca/use-real-world-evidence-single-drug-assessments-environmental-scan

Europe

European Medicines Agency (EMA)
  • Post-authorisation studies

https://www.ema.europa.eu/en/human-regulatory/post-authorisation

 

UK Medicines and Healthcare products Regulatory Agency

https://www.gov.uk/government/organisations/medicines-and-healthcare-products-regulatory-agency

  • Guidance on GxP data integrity

https://www.gov.uk/government/publications/guidance-on-gxp-data-integrity

  • Draft guidance on randomised controlled trials generating real-world evidence to support regulatory decisions

https://www.gov.uk/government/consultations/mhra-draft-guidance-on-randomised-controlled-trials-generating-real-world-evidence-to-support-regulatory-decisions

(ISPE member comments on this draft guidance were provided)

 

UK National Institute for Health and Care Excellence (NICE)
  • Evidence standards framework for digital health technologies

https://www.nice.org.uk/about/what-we-do/our-programmes/evidence-standards-framework-for-digital-health-technologies

Asia

China National Medical Products Administration

http://www.nmpa.gov.cn/WS04/CL2042/

 

Japan Pharmaceuticals and Medical Devices Agency

https://www.pmda.go.jp/english/pnavi_e-01.html

  • Office of Advanced Evaluation with Electronic Data

https://www.pmda.go.jp/english/review-services/reviews/advanced-efforts/0002.html

 

Center for Drug Evaluation, Taiwan

https://www.pmda.go.jp/files/000226215.pdf

 

Singapore Health Sciences Authority

https://www.hsa.gov.sg/docs/default-source/hprg/therapeutic-products/guidance-documents/guidance-for-industry_post-marketing-vigilance-requirements-for-therapeutic-products-v2_01-apr-2020_final---v2.pdf

 

https://www.duke-nus.edu.sg/docs/librariesprovider5/events-doc/2019-dia-core-report-v4.pdf?sfvrsn=7159bfbc_0

 

Japan-Pharmaceuticals and Medical Devices Agency

The guidelines for the conduct of pharmacoepidemiological studies using medical information databases, etc., in evaluation of drug safety

https://www.pmda.go.jp/files/000147250.pdf

 

 

Japan- Pharmaceuticals and Medical Devices Agency

Protocol template and instructions for post-marketing database studies of medicinal products

https://www.pmda.go.jp/files/000222302.pdf

 

 

Japan- Ministry of Health, Labour and Welfare

Ordinance of Revision of Good Postmarket Surveillance Practice

https://www.pmda.go.jp/files/000220720.pdf

 

 

Japan- Ministry of Health, Labour and Welfare

Basic policy on the utilization of health information databases for post-marketing surveillance of medicinal products

https://www.pmda.go.jp/files/000218531.pdf

 

 

Japan- Ministry of Health, Labour and Welfare

Points to consider on ensuring data credibility of databases in post-marketing database studies

https://www.pmda.go.jp/files/000223003.pdf

 

 

Japan- Ministry of Health, Labour and Welfare

Procedures for study planning for post-marketing surveillance of medicinal products

https://www.pmda.go.jp/files/000228612.pdf

 

 

Japan- Ministry of Health, Labour and Welfare

Basic policy on the conduct of validation studies of outcome definitions used in post-marketing database studies

https://www.pmda.go.jp/files/000235927.pdf

 

 

Japan- Ministry of Health, Labour and Welfare

Basic policy on utilization of patient registries in filing marketing authorization (for public comment)

https://public-comment.e-gov.go.jp/servlet/PcmFileDownload?seqNo=0000211435

 

 

Japan- Ministry of Health, Labour and Welfare

Points to consider on ensuring data credibility in patient registries in filing marketing authorization (for public comment)

https://public-comment.e-gov.go.jp/servlet/PcmFileDownload?seqNo=0000211436

 

 

Taiwan-Food & Drug Administration

Basic policy on the use of RWE to support drug development

https://www.fda.gov.tw/Tc/newsContent.aspx?cid=3&id=26255

 

 

Taiwan- Food & Drug Administration

Guidelines of clinical investigation using EHRs

https://www.fda.gov.tw/Tc/newsContent.aspx?cid=3&id=26558

 

 

Taiwan- Food & Drug Administration

Key considerations for pragmatic trials, study design for RWE

https://www.fda.gov.tw/Tc/newsContent.aspx?cid=3&id=26558

 

 

China- National Medical Products Administration

Guidelines for Real-World Evidence to Support Drug Development and Review (trial implementation)

 

China- National Medical Products Administration

Guideline on Using Real-World Evidence to Support Drug Research & Development and Evaluation (Interim)

 

China- National Medical Products Administration

Guidelines for Real-World Data Used to Generate Real-World Evidence (for public comment)

 

China—Center for Drug Evaluation

Draft Instructions on Guidelines for Real-World Data Used to Generate Real-World Evidence (for public comment)

Australasia

Australian Therapeutic Goods Administration
  • The TGA has adopted many of the EU GVP modules, which are outlined

https://www.tga.gov.au/publication/joint-tga-medicines-australia-guidelines-design-and-conduct-company-sponsored-post-marketing-surveillance-studies

https://www.tga.gov.au/publication/risk-management-plans-medicines-and-biologicals


 

Relevant RWE data sources

This is not a comprehensive list of data sources and these are not ISPE endorsed, but merely comprise some examples.

Region

Source

Description and links

North America

HealthData.gov

An electronic index of US government datasets, managed by the U.S. Department of Health and Human Services Office of the Chief Technology Officer

https://healthdata.gov/

 

NIH

An online guide which contains information on finding, using, and analyzing health and population data

https://www.nihlibrary.nih.gov/resources/subject-guides/health-data-resources

 

CIHR

A sample of data and analytic platforms, as well as examples of Canadian data training hubs and resources

https://cihr-irsc.gc.ca/e/49941.html

 

Government of Canada

Resources on the state of Canadians' health, including statistics, facts, reports and data

https://www.canada.ca/en/services/health/data.html

 

FDA

MyStudies app: Open Source software for collecting real world data from patients to link to other sources e.g. electronic health records

 https://www.fda.gov/drugs/science-and-research-drugs/fdas-mystudies-application-app-technical-background

 

Europe

European Network of Centres for Pharmacepidemiology and Pharmacovigilance (ENCePP)

ENCePP Resources database- an electronic index of available EU research organisations, networks and data sources

http://www.encepp.eu/encepp/resourcesDatabase.jsp

 

 

WHO

European health information gateway

https://gateway.euro.who.int/en/datasets/

 

European Union

Health data resources

https://ec.europa.eu/health/indicators_data/data_en

Asia

Taiwan National Health Insurance Research Database

https://nhird.nhri.org.tw/en/

Australasia

AIHW

Data collections

https://www.aihw.gov.au/about-our-data/our-data-collections

 

NSW Government

Links and information on available datasets

https://www.medicalresearch.nsw.gov.au/data/

 

Ministry of Health New Zealand

A central location for accessing New Zealand health data and health statistics collected and produced by the Ministry of Health and wider health sector

https://www.health.govt.nz/nz-health-statistics

 

Other Resources

Description

Link

Video on Advancing the Use of Real World Evidence for Regulatory Decision Making (DIA)

https://globalforum.diaglobal.org/issue/september-2019/advancing-the-use-of-real-world-evidence-for-regulatory-decision-making/?_ga=2.3906416.1733483813.1597321879-328470703.1597321879

 

FDA’s Real World Evidence Program Moving Forward (Article; DIA)

https://globalforum.diaglobal.org/issue/december-2019/fdas-real-world-evidence-program-moving-forward/?_ga=2.138564083.1733483813.1597321879-328470703.1597321879

 

Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes (Video; Duke Margolis)

https://healthpolicy.duke.edu/events/leveraging-randomized-clinical-trials-generate-real-world-evidence-regulatory-purposes

 

Real-World Evidence Collaborative (Article; Duke Margolis)

https://healthpolicy.duke.edu/projects/real-world-evidence-collaborative