
FDA Reviews Gains and Risks of Accelerated Approval Process
Is FDA approving too many new drugs and added indications too quickly based on surrogate endpoints that fail to pan out?
Is FDA approving too many new drugs and added indications too quickly based on surrogate endpoints that fail to pan out? While patient advocates and the research community have been strong backers of early access to treatments that demonstrate potential effectiveness for serious and life-threatening conditions that lack effective treatment, some consumer advocates and research experts believe that FDA has been lax in approving medicines with insufficient supporting data.
Now the agency is reviewing the record of its accelerated approval (AA) program following the recent withdrawals of certain key indications for several leading cancer therapies based on the failure of postapproval studies to document extended benefits of treatment. That development has raised questions about the broader FDA policy, including frequent difficulties in conducting postapproval clinical trails and the relatively few therapies that have had approvals withdrawn by the agency.
These issues will be reviewed by FDA’s Oncologic Drugs Advisory Committee (ODAC) at a
These monoclonal antibodies target PD-1/PD-L1 proteins to boost immune response against cancer, raising questions about the broader efficacy of such treatment. The confirmatory studies aimed to document overall improved survival compared to standard-of-care chemotherapy, after gaining accelerated approval based on tumor response in early studies. The ODAC meeting will examine the status of efforts by Genentech, Merck, and BMS to confirm additional indications for their therapies, and, in the process, will scrutinize the AA program more publicly for a range of cancer types.
The meeting is part of a broad, ongoing effort by FDA’s Oncology Center of Excellence (OCE) to review AA policies and the role of surrogate endpoints in evaluating new therapies. Sponsors are anxious to know whether the agency is focusing on this small subset of treatments or on the use of surrogate endpoints and other accelerated research strategies more broadly. Over its 30-year history, FDA’s AA program has led to speedy approvals of dozens of products, primarily for oncology and hematology in recent years. Although FDA may withdraw an accelerated approval for a product, the manufacturers did so voluntarily in the above cases, and for some dozen other therapies in previous years. There are only two cases where the agency initiated a withdrawal, following a complex and time-consuming process.
OCE’s review is expected to tap a
The ODAC meeting will provide an opportunity for sponsors to provide updates on ongoing studies for their therapies and to examine whether further testing could revise the withdrawal actions. A long-debated issue is industry’s poor record in conducting and reporting results from postapproval studies, and FDA appears to be taking a harder line on requiring confirmatory trials to support clinical benefit.
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