019: FDA Trends for Accelerated Drug Approvals in Oncology Indications

Statement of Purpose: The regulatory landscape for accelerated approvals for oncology drugs is changing as documented in the March 2023 FDA draft guidance “Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics.” The purpose of this poster is to characterize recent trends in accelerated approvals in oncology with respect to study design, highlight considerations that might be sensitive to changing regulations, and discuss future considerations for drug developers planning to use this pathway.

Description of Methods & Materials: This retrospective analysis used FDA databases to analyze sponsors’ utilization of the accelerated approval pathway, considerations of sample sizes and study designs used in pivotal clinical trials, use of companion diagnostics to select patient populations, time to convert an accelerated approval to a regular approval by completing post marketing requirements, and number of market withdrawals after an accelerated approval.

Data & Results: This analysis found that use of the accelerated approval pathway showed a trend towards a decrease in 2022 compared to 2021 and 2020. Conversion times from accelerated approval to a regular approval are decreasing over time. Results also show a spike in FDA withdrawal of accelerated approvals in the last 2 years. Additionally, we show that study designs differ significantly in accelerated vs regular approval in terms of sample sizes, number of treatment arms and use of companion diagnostics to select patient population. Accelerated approval drugs also utilize multiple other expedited drug pathways and are popular in orphan indications.

Interpretation, Conclusion or Significance: Important changes in regulatory expectations are occurring in oncology drug development. The FDA is modifying and increasing expectations around accelerated approvals. The findings of this analysis are consistent with the FDA’s recent messaging that prolonged duration on the market for drugs with accelerated approval but without confirmatory studies will no longer be acceptable, and with the recommendations for trial designs intended to support accelerated approval described in the March 2023 draft guidance. Drug developers will need to consider the factors discussed in this poster to reevaluate their development strategies for oncology indications.

Disclosures: None