Commercial health plan spending on oncology drugs approved via the accelerated approval pathway and later withdrawn from the market.

Abstract

11134 Background: The U.S. Food and Drug Administration (FDA) introduced the Accelerated Approval Program in 1992 to expedite the approval of drugs that can be used to treat certain conditions and fill an unmet medical need. Clinical trials conducted to achieve accelerated approval status often use surrogate outcomes as primary endpoints to reduce the time it takes to get FDA approval. Drug manufacturers must conduct further research to verify and report the projected clinical benefit and safety. The FDA may issue traditional approval to previously accelerated approved drug or may request the manufacturer to withdraw a drug from the market based on the clinical benefit and/or safety demonstrated in the confirmatory trial(s). We report members impacted, total drug claims, and total drug cost incurred by a commercial health plan associated with the coverage of oncology drugs approved by the FDA and later withdrawn from the market between 2020 to 2023. Methods: Fifteen unique indications associated with oncology drugs that received accelerated approval and later withdrawn by the FDA were identified between 2020 to 2023. A cohort of patients enrolled in commercial health plan was further identified using the ICD-10 code related to the drug indication being withdrawn. To calculate total paid amount for drugs, de-identified administrative claims data submitted on behalf of this cohort were sorted and summed in this time period using either J-code for drugs covered under medical benefits or national drug code (NDC) for drugs covered under pharmacy benefits. Results: Between 2020 to 2023, 8 oncology drugs were withdrawn because of later failing to demonstrate clinical benefit in the confirmatory trials, 2 drugs were withdrawn due to safety concerns, and 5 drugs were withdrawn due to drug manufacturer unable to complete a confirmatory trial. In this population, we identified 18,430 unique members with 103,743 administrative claims. The total paid amount was $1,392,553,995 in this time period. The average drug cost per administrative claim was $13,423 and the average drug cost per member was $75,559. Conclusions: Drug costs established by manufacturers are not differentiated based on the approval status - accelerated or regular. While drugs approved via accelerated approval pathway may provide benefit to certain patient population, the cost associated with accelerated approved drugs needs more attention. Indication-specific pricing model may help alleviate the cost burden associated with drugs approved via accelerated approval pathway. [Table: see text]