PHP39 - Are FDA Accelerated Approvals Unconditional Approvals?

Abstract

The Food and Drug Administration (FDA) initiated the Accelerated Approval program in 1992. This enabled early market authorisation of treatments for diseases with severe unmet needs based on surrogate or interim endpoint data. Post-marketing confirmatory trials are mandatory to verify the clinical benefit. However, only 50% (24/48) of oncology drugs that received accelerated approval had been converted to regular approval by the end of 2013, with an average delay of 53 months (Macaulay, ISPOR Quebec, 2014). This research provides an analysis on the approval status of oncology drugs that have received accelerated approval since 2014. Accelerated approvals for oncology drugs were identified from www.fda.gov (up to 11/30/2017). In total, 89 oncology drug:indication pairings received accelerated approvals, with a per annum increase over time (range: 3 [1995]–15 [2017]). Overall, 38/89 (43%) accelerated approvals have been converted to regular approvals with an average delay of 45 months (range: 5–150); 48/89 (54%) accelerated approvals have not been converted to regular approval despite being on the market for an average of 53 months (range: 2–156) due to lack of confirmatory trial data. 3/89 accelerated approvals (3%) were voluntarily withdrawn due to failed confirmatory trials; two of which were subsequently approved in the same cancer type for a different patient population and one remains permanently withdrawn. An increasing number of oncology drugs have received accelerated approval based on early clinical data. However, the majority have not been converted to regular approval despite being on the market for up to 14 years. Given that some drugs have been withdrawn following failed confirmatory trials, the timely completion and submission of confirmatory trial data should become a strict, non-negotiable requirement, where the FDA should implement an audit to determine the reason for the delay and, if deemed necessary, review the license.