
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
Israel faces tough choices over haredi draft exemptions, legal expert warns - 2
Visiting This Japanese City Just Got A Little More Expensive (Here's What Travelers Should Know) - 3
The most effective method to Pick the Right Volvo XC40 Trim for Your Way of life - 4
A NASA spacecraft orbiting Mars may be dead - 5
Instructions to Pick the Ideal Pre-assembled Home for Your Necessities
US FDA grants market authorization to six on! PLUS nicotine pouch products
The biggest black hole breakthroughs of 2025
The Solution to Individual budget: Dominating Cash The board
Which Film Has the Incomparable Melodic Score?
Manual for Tracking down One of a kind Store Inns
Putin critic gets six years in penal colony, vows hunger strike
SpaceX launches Starlink satellites from California on 160th Falcon 9 flight of the year (video)
Sean 'Diddy' Combs faces new sexual assault allegations, currently under investigation by Los Angeles Sheriff's Department
How does Spotify Wrapped calculate your listening age? What your number says about you.











