RWE for Regulatory Decision Making

ISPE Statement on Real-World Evidence (RWE)

• ISPE's Position on Real-World Evidence (RWE) Feb 2020

ISPE RWE-related Guidance Documents and Policy Statements

• ISPE Comments on the Framework for FDA's Real World Evidence Program July 2019
• ISPE's Comments on the Core Recommendations in the Summary of the Heads of Medicines Agencies (HMA) - EMA Joint Big Data Task Force April 2019
• ISPE's response to The Magic of Randomization versus the Myth of Real-World Evidence. N Engl J Med. 2020 Feb 13;382(7):674-678. doi: 10.1056/NEJMsb1901642. PMID: 32053307 was published in the July 23, 2020 issue of N Engl J Med: Winterstein AG, Girman CJ, Pottegård A. Randomization versus Real-World Evidence. N Engl J Med. 2020 Jul 23;383(4):e21. doi: 10.1056/NEJMc2020020. PMID: 32706549

Relevant RWE Guidance documents from Health Authorities

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

United Kingdom

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

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

Europe

European Medicines Agency (EMA)

• post-authorisation

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

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