
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
First Greenland, now Iceland? Annexation joke by Trump ally gets frosty response in the Arctic nation. - 2
An 'explosion' of solo-agers are struggling with rising costs and little support: 'I'm flying without a net' - 3
The Fate of Gaming: 5 Energizing Advancements Not too far off - 4
A mom's viral post is raising the question: Do kids need snacks? Dietitians have answers. - 5
Novo Nordisk cuts Wegovy price as CEO pledges to go 'all in' on weight loss pill
A Manual for the Right SUV for Seniors
5 things for parents to know about changes to kids vaccine schedule
Equality requires universal draft, participation in economy and workforce, MK Liberman says
Astronauts welcome arrival of new crewmates | On the International Space Station this week Nov. 24-28, 2025
'Euphoria' releases Season 3 trailer, premiere date: Watch Rue and Laurie finally face off
Early Thanksgiving week forecast: Where Americans can expect cold, rain and snow for the holiday
Watch China's Shenzhou 22 rescue ship arrive at Tiangong space station (video)
Get away from the Tedious Drudgery: Go into Business Today!
Solar storms have influenced our history – an environmental historian explains how they could also threaten our future












