
By Michael Erman
NEW YORK (Reuters) -The U.S. Food and Drug Administration can approve new personalized treatments for rare and deadly genetic diseases based on data from a handful of patients, two of the agency's top officials said on Wednesday.
FDA Commissioner Marty Makary and Chief Medical and Scientific Officer Vinay Prasad said in an essay published in the New England Journal of Medicine that for certain conditions, companies could rely on appropriately designed studies with small sample sizes rather than randomized trials. They will rely on biological plausibility and clinical improvements in those early patients.
"Current regulations are onerous and unnecessarily demanding," Makary and Prasad wrote. "For patients and families, there is no time to wait."
The new "plausible-mechanism" pathway would allow the agency to grant marketing authorization after manufacturers demonstrate success with several consecutive patients.
Companies that receive these approvals will be required to collect real-world evidence to confirm efficacy continues and to look for safety issues that might arise.
The new approach will prioritize treatments for rare diseases that are fatal or cause severe childhood disability. Common diseases with unmet medical needs may also qualify.
While makers of cell and gene therapies are likely to be significant beneficiaries of the new approval process, Makary and Prasad said that other types of treatments could also receive licensure this way.
"The FDA will work as a partner and guide in ushering these therapies to market," the officials wrote.
(Reporting by Michael ErmanEditing by Bill Berkrot)
LATEST POSTS
- 1
Moon rush: These private spacecraft will attempt lunar landings in 2026 - 2
Falcon 9 rocket launches Starlink satellites before making 550th SpaceX landing (video) - 3
Hamas propaganda expert explains Israel's internal conflicts influenced Hamas's Oct. 7 assault - 4
The most effective method to Pick A Trade-in vehicle Stage - 5
Horses really can smell our fear, new study finds
2026 Golden Globes: How to watch, start time, TV channel, full nominee list and more
The Most Enrapturing Authentic Milestones to Visit
19 Peculiar Films You Shouldn't Watch With Your Mum
Russia Creates New Military Branch Dedicated To Drone Warfare
Some super-smart dogs can pick up new words just by eavesdropping
We may be witnessing the messy death of a star in real time
Bother Control Administrations for 2024: Decide for Your Home
Woman shocked to welcome baby after experiencing stomach pain on Christmas
4 Jeep Models: Dominating Execution and Flexibility for Each Experience













