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Accelerated Approval: US FDA Defends Size Of Premarket Safety Databases, Confirmatory Endpoints

 
• By Sue Sutter

Taking on critics who say it is too quick to award accelerated approval based on surrogate endpoints and limited safety data, agency's 25-year review shows cancer drugs receiving accelerated approval generally have twice the amount of safety data as efficacy, and more than half have confirmed clinical benefit after approval.

Medical book with Stethoscope

Safety databases for new molecular entity oncology and hematology drug indications approved under accelerated approval are, on average, twice the size of the efficacy databases, US FDA staff say in a review article published online in JAMA Oncology March 1.

The review, which takes a data-driven look at the agency's use of the accelerated approval mechanism for cancer treatments over...

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More from Approval Standards

Post-Approval, Real-Time Monitoring Will Help Approval Standards, US FDA’s Makary Says

 
• By Sue Sutter

The FDA will use electronic health records to “get eyes” on drugs immediately after approval, Commissioner Martin Makary told the DIA 2025 Global Annual Meeting. He also wants to reduce development time with better communication and new approval pathways.

Generic Drug Office’s Policy Staff Returning To US FDA

 
• By Derrick Gingery

The group had been laid off as part of the 1 April reduction-in-force, which led to missed guidance publication deadlines.

UK MHRA: Bacteriophage Developers Wanted ‘More Clarity’ On UK Framework

 
• By Eliza Slawther

Julian Beach, interim executive director of healthcare quality and access at the UK drug regulator, the MHRA, tells the Pink Sheet how the agency is responding to increased efforts to develop bacteriophage therapies.

LDT Final Act? US FDA Will Not Appeal District Court Decision Vacating Final Rule

 
• By Brian Bossetta

The US FDA ended efforts to regulate lab-developed tests as medical devices for now when it did not appeal a decision from the Eastern District of Texas that tossed out the agency's final rule.

More from Pathways & Standards

Elevidys Helps Sarepta To First US FDA Platform Technology Designation

 
• By Bridget Silverman

Sarepta's rAAVrh74 vector, used in the marketed Duchenne muscular dystrophy gene therapy Elevidys and across the company's limb girdle muscular dystrophy pipeline, earned a platform designation as the lead LGMD candidate prepares for BLA filing.

US FDA Could Miss User Fee Spending Triggers With House FY 2026 Funding

 
• By Sarah Karlin-Smith and Derrick Gingery

FDA employees are concerned that while the budget authority total proposed for fiscal year 2026 meets the user fee appropriations' triggers, the FDA could have trouble meeting allocation triggers without cuts to non-review work.

US FDA’s Rising First-Cycle Complete Response Rate Draws Congress’ Attention

 
• By Sue Sutter

The House Appropriations Committee wants an analysis of how issues that led to CRLs could have been resolved within the first review cycle and seeks an agency crackdown on counterfeit GLP-1 agonists.