Evaluating Trends in the Use of RWE in FDA New Drug Approvals
Speaker(s)
York M1, Franklin M2, Rojubally A3, Patel M4, Gdovin Bergeson J5
1Franklin Pharmaceutical Consulting, Rock Hill, SC, USA, 2Franklin Pharmaceutical Consulting, Cary, NC, USA, 3Franklin Pharmaceutical Consulting, Surrey, BC, Canada, 4Franklin Pharmaceutical Consulting, Westport, CT, USA, 5Franklin Pharmaceutical Consulting, Columbia, SC, USA
Presentation Documents
OBJECTIVES: Real-world evidence (RWE) continues to be a widely discussed component of new drug applications (NDAs) within the US Food and Drug Administration (FDA). This study sought to evaluate the use of RWE in recent FDA drug approvals by assessing variables such as frequency, purpose, and acceptability.
METHODS: Publicly available FDA application review files for NDA approvals during the year 2022 were systematically reviewed for the use of RWE. Sources included the FDA’s 2022 CDER Drug and Biologic Calendar Year Approvals and the FDA’s Drugs@FDA: FDA-Approved Drugs. Search terms included RWE, real-world evidence, external control, non-interventional, registry, observational, retrospective, and cohort studies. Results were classified by therapeutic area (TA) and FDA submission classification to evaluate current trends in RWE use. RWE in approvals was categorized by frequency (percentage of approvals containing RWE), purpose (benchmark/disease history, formal comparison, or post-authorization request), and acceptability (did the FDA consider the RWE acceptable as support for the approval).
RESULTS: Of the 92 NDA approvals in 2022, 36 (39.1%) contained RWE. The top 3 TAs with submissions containing RWE were oncology, nerve disorders, and skin conditions. RWE was most often included in applications classified as priority (75%), priority orphan (46%), and standard orphan (45%). Over 70% of approvals classified as “new molecular entity” included RWE. Within the approvals, the purpose of the RWE was primarily as a benchmark/disease history indicator (67%) or in post-authorization studies (30%) to support safety and/or efficacy outcomes. The RWE was accepted in 31% and dismissed in 36% of new drug approvals.
CONCLUSIONS: In 2022, RWE was commonly included for consideration by the FDA in new drug approvals. The purpose of the RWE varied across the TAs evaluated and submission classifications, impacting the acceptability of the RWE. Continued evaluation of the US FDA’s acceptance of RWE in NDAs is warranted.
Code
RWD60
Disease
No Additional Disease & Conditions/Specialized Treatment Areas