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Nonrandomized Real‐World Evidence to Support Regulatory Decision Making: Process for a Randomized Trial Replication Project
Author(s) -
Franklin Jessica M.,
Pawar Ajinkya,
Martin David,
Glynn Robert J.,
Levenson Mark,
Temple Robert,
Schneeweiss Sebastian
Publication year - 2020
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.1633
Subject(s) - randomized controlled trial , food and drug administration , replication (statistics) , replicate , medicine , legislation , process (computing) , risk analysis (engineering) , computer science , surgery , political science , statistics , mathematics , virology , law , operating system
Recent legislation mandates that the US Food and Drug Administration issue guidance regarding when real‐world evidence ( RWE ) could be used to support regulatory decision making. Although RWE could come from randomized or nonrandomized designs, there are significant concerns about the validity of RWE assessing medication effectiveness based on nonrandomized designs. We propose an initiative using healthcare claims data to assess the ability of nonrandomized RWE to provide results that are comparable with those from randomized controlled trials ( RCT s). We selected 40 RCT s, and we estimate that approximately 30 attempted replications will be completed after feasibility analyses. We designed an implementation process to ensure that each attempted replication is consistent, transparent, and reproducible. This initiative is the first to systematically evaluate the ability of nonrandomized RWE to replicate multiple RCT s using a structured process. Results from this study should provide insight on the strengths and limitations of using nonrandomized RWE from claims for regulatory decision making.