Use of Real World Evidence in 2023 Novel Drug Approvals

OBJECTIVES: The United States Food and Drug Administration (FDA) was required to evaluate potential use of real world evidence (RWE) to support approval of new indications under the 2016 21^st Century Cures Act. FDA has published a framework and guidances on RWE to support regulatory decision-making.

This study assessed the proportion of novel medications approved in 2023 by FDA’s Center for Drug Evaluation and Research with RWE in applications, quantified the types of RWE, and explored the aspects of RWE supporting efficacy or safety.

METHODS: We identified novel drug approvals and associated clinical reviews on the FDA website and abstracted the drug name, active ingredient, approval date, approved use, review priority, therapeutic area (TA), indicated population and age, and whether RWE supported efficacy or safety. If RWE was present, we extracted the number of studies and a brief description. We noted study design and whether extant data were electronic health records (EHR) or claims.

Approvals were described using counts and proportions by TA and indicated age. Use of EHR and claims were tabulated.

RESULTS: Among 54 approvals with publicly available clinical review, the most common TAs were hematology (20.4%), neurology (14.8%), and oncology (14.8%). Most medications were to treat adults (18+ years; 79.6%).

Only 5 approvals (9.3%) included RWE. Supporting efficacy, 2 applications included external controls (both dermatological), 1 a prospective observational cohort (hematology), and 1 a retrospective cohort (infectious disease). Supporting safety was 1 retrospective cohort (dermatology). Three included patients <18 years and 2 were only 18+. Three approvals included multiple RWE studies for a total of 2 external controls, 2 prospective, and 7 retrospective studies (6 EHR; 3 claims).

CONCLUSIONS: Use of RWE for novel premarket applications remains minimal (<10%) and varied across therapeutic areas, indicated ages, and types of RWE studies.