Drugs

Recent Accelerated Approval Path Changes Explained

FDA’s accelerated approval pathway, which is more than three decades old, has undergone major changes since 2024 following mounting evidence of systemic weaknesses, including delayed confirmatory trials, questionable validity of surrogate endpoints, and inconsistent enforcement. A Bioprocess Online guest column says the changes started with a 2024 comprehensive draft guidance issued by FDA to implement mandates in the FDA Omnibus Reform Act and directly address findings from academic research on pathway performance.

The draft clarified that drugs must meet each of these criteria

address serious or life-threatening conditions with no available therapies or limited effective alternatives;
provide evidence of effectiveness through surrogate or intermediate clinical endpoints reasonably likely to predict clinical benefit; and
demonstrate meaningful advantages over existing therapies when available.

FDA now has new authority to require that confirmatory trials be underway before or shortly after accelerated direct response to data showing that preapproval trial initiation correlates with timely completion, the column says. The legislation also gave the agency expedited withdrawal authority that lengthy formal processes had previously constrained. Under the new process, if a confirmatory trial fails to demonstrate clinical benefit on the pre-agreed primary endpoint, FDA may withdraw approval more rapidly, provided that manufacturers retain appropriate due process rights. “This balances patient protection with regulatory efficiency,” the author writes.

The column uses case studies to highlight and explain some of the changes that have been implemented. It concludes that the agency’s “heightened accountability for accelerated approval confirmatory trials responds directly to documented regulatory failures while preserving the pathway’s essential functioning, thereby enabling rapid access to breakthrough therapies for severe conditions with unmet needs.”

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome