FDA Defends Accelerated Approval Program
FDA finds itself defending the accelerated approval process in the wake of Biogen’s controversial approval for its Alzheimer’s therapy Aduhelm (aducanumab), and a new BMJ report that since the program’s inception in 1992, nearly half (112) of the 253 drugs authorized have not been confirmed as clinically effective. “Of these 112 drugs approved in the past 28 years, a fifth (24) have been on the market for more than five years and some for more than two decades — often with a hefty price tag,” the report shows.
In defense of the program, FDA points to its recently released data showing that of the 115 current accelerated approvals that have not been converted to full approval, 84 were granted since the beginning of 2018 (i.e. 3.5 years ago). The agency notes that confirmatory studies take time to complete, and it believes it is important that sponsors set achievable clinical trial target timelines.
“We work actively with sponsors to ensure that confirmatory studies are completed in a timely manner and expect sponsors to commit all resources needed to move trials forward as effectively as possible, with the aim of completing trials as soon as is feasible, while assuring the quality of the data and the robustness of the results,” an FDA spokesman told FDA Webview 8/2. “Because the FDA continues to use this pathway to accelerate access to drugs for serious and life-threatening diseases for which there is an unmet medical need, at any point in time there will be drugs that are not converted because the confirmatory trials are ongoing.”
Meanwhile, FDA Oncology Center of Excellence director Richard Pazdur told a 7/29 Friends of Cancer Research Web cast (25:38 minute mark) that the accelerated approval program is under attack by critics. Acknowledging that there is room for improvement, Pazdur took issue with critics focusing on the negatives of the program (about 10 indications withdrawn from the 155 approved since 1992. He said more focus should be given to the successes of the program — “the very important drugs that have been approved, years before their confirmatory studies have been completed.”
To focus just on the failed trials is short sighted, according to Pazdur. A failed trial does not mean a failed drug, he said, adding that there are many reasons why a drug can fail, such as a trial not being appropriately designed, not enrolling enough patients, selecting the wrong patient population and choosing the wrong endpoint.
To improve the program, Pazdur continued, would involve addressing “some of the difficulties, such as removing drugs from the market when they don’t meet their primary endpoint or when we believe the standards of medicine have changed.” He also suggested another avenue involving “discussions with companies on a comprehensive drug development plan aimed at looking at what are the confirmatory studies and the accelerated approval studies before the drug is even filed, rather than having these discussions during the process of the accelerated approval review.”