
FDA Continues to Grapple with Accelerated Approval Issues
Scrutiny of accelerated approvals has generated a range of reform proposals to accelerate the conduct of confirmatory studies.
The approval of certain critical medicines based on preliminary clinical evidence continues to raise questions about reliance on too-weak efficacy data and delays in sponsors submitting confirmatory evidence from postapproval trials. FDA has approved dozens of valuable therapies under the program, many of them able to confirm efficacy through subsequent clinical studies. However, the controversial approval in 2021 of Biogen’s Aduhelm to treat Alzheimer’s Disease by the Center for Drug Evaluation and Research (CDER) continues to haunt the program, triggering widespread scrutiny of accelerated approvals and generating a range of reform proposals to accelerate the conduct of confirmatory studies.
FDA’s Oncologic Drugs Advisory Committee (ODAC) added to the debate with its April 2021 meeting that challenged several “dangling approvals” for cancer therapies. That prompted some manufacturers to withdraw earlier approvals for certain indications and others to renew confirmatory study efforts. The agency
Legislative reform?
Meanwhile, Congressional leaders have proposed measures to reform the accelerated approval program, as discussed in an article in the
At the same time, the cancer community and numerous patient disease groups are anxious to retain the accelerated approval program as important for enhancing patient access to therapies likely to have important benefits. These advocates also recognize that FDA should be able to remove those products unable or unwilling to confirm initial efficacy to retain credibility for the program. The
Stricter oversight of the accelerated approval program appears likely, as product costs and benefits remain in the spotlight. An analysis by
About the author
Jill Wechsler is Washington editor for BioPharm International.
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