RWE Program Action Items

Issue guidance on how to assess the reliability and relevance of RWD from medical claims and EHRs used to generate RWE regarding drug product effectiveness; examine how to assess the reliability and relevance of registry data and international electronic health care data

Review and, where applicable, publish guidance on potential gaps in RWD sources and strategies to address them

Develop guidance on considerations for designing clinical trials that include pragmatic design elements and that generate evidence of effectiveness for regulatory decisions; explore pragmatic approaches to each stage of a clinical trial, including recruitment and enrollment of patients, strategies for facilitating interventions, and approaches to assessing outcomes

Consider issuing guidance on the use of RWD to generate external control arms

Issue guidance about observational study designs using RWD, including whether and how these studies might provide RWE to support product effectiveness in regulatory decision-making; consider reporting requirements for such studies used to support effectiveness determinations

Consider whether existing guidance documents on use of electronic data in clinical investigations adequately address concerns relevant to different study designs using RWD to generate RWE for drug product effectiveness determinations and whether additional guidance on use of electronic source data is needed

Finalize the guidance, “Use of Electronic Records and Electronic Signatures in Clinical Investigations Under 21 CFR part 11 – Questions and Answers,” and consider its applicability to different study designs; issue additional guidance as needed on regulatory considerations raised by different study designs using RWD to generate RWE submitted to support drug product effectiveness

Source: Framework for FDA’s Real-World Evidence Program

Real-World Evidence: US FDA Framework Emphasizes Data Fitness And Study Quality

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