
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
From School Dropout to Example of overcoming adversity: My Excursion - 2
Which Diet Prompts the Incomparable Wellbeing Results? - 3
Gym tied to outbreak of obscure disease that spreads through mist - 4
Help Your Efficiency: 10 Authoritative Apparatuses to Attempt - 5
The most effective method to Integrate Compact disc Rates into Your Retirement Arranging
Sophie Kinsella, 'Confessions of a Shopaholic' author, dies at 55 after battle with cancer
Lift Your Style: Famous Hairdos for Ladies
Medical team successfully delivers baby and removes massive tumor
Rocket shines under the northern lights | Space photo of the day for March 25, 2026
How to see the Ursids, the final meteor shower of 2025
'Supergirl' drops 1st teaser trailer: Watch Milly Alcock as Kara Zor-El and the return of Krypto the Superdog
Figure out How to Augment Your Rooftop Substitution Speculation
UK, Canada, Germany, others condemn Israel's West Bank settlement plan
Israel reports first missile fire from Yemen since start of Iran war












