OBJECTIVES:

1. Analyse RWE Role in US FDA Approvals of Novel Drugs and Post Marketing Requirements of RWE Study (Novel Drug Approvals US FDA 2023)
2. Analyse trends and patterns of use of RWE in drug approvals, and derive insights for designing patient-centric trials

METHODS: All novel drug approvals including NDA (New Drug Application) and BLA (Biological License Application) submitted in 2023 by respective sponsors, were identified from Center for Drug Evaluation and Research (CDER) and Drugs@FDA were extracted and analyzed. The submission package data supported with RWE were segregated based on therapeutic areas. These were further categorized among two mutually non-exclusive groups: a. RWE used to support approvals from therapeutic context (e.g., epidemiology, natural history, incidence, or prevalence of the disease) b. RWE used to support safety and/or effectiveness which were further stratified into primary, supportive, not adequate for decision. The approvals were further analyzed for to identify post marketing study (PMS) requirements with recommendation of adopting RWE Study design for PMS.

RESULTS:

• 22 Novel Drug Approvals had a submission package inclusive of RWE of 55 Novel Drug Approvals
• 12 Novel Drug Approvals included RWE study design as part of post marketing requirements

CONCLUSIONS: Understanding the adoption of RWE use in the submission package for Novel Drugs will help sponsors to develop robust RWE based/supported trial design in consultation with regulatory agencies. This can help save patient lives by reducing the cycle time of drug approval and create an affordable access of drugs for patients by reducing development and approval costs.