
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
5 Instructive Toy Brands for Youngsters - 2
Pedal Power: Divulging Well known Bike Brands for Each Cyclist - 3
Figure out how to Separate Among Fledgling and Master Fender bender Legal counselors - 4
Figure out How to Explore Land Close to 5G Pinnacles - 5
Watch SpaceX launch NASA's Pandora exoplanet-studying satellite on Jan. 11
Grass Care Administrations for a Wonderful, Sound Yard
Former GLP-1 users regain lost weight after about 18 months, study says
Bruno Mars tour 2026: How to get tickets for 'The Romantic Tour,' presale times, prices and more
When will the Epstein files be released — and will they reveal anything new?
Vote In favor of Your Number one Cell phones
Multi-million-euro win in Spanish lottery in doubt due to oversight
Trump signs bill allowing whole milk to return to school lunches
From Lounge chair to Money: Online Positions That Will Change Your Profession
EU chief urges Iran to free imprisoned protesters, lift internet ban












