
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
The most effective method to Pick the Right Material Organization: Fundamental Tips - 2
Lilly becomes first healthcare firm to join trillion-dollar club, Wall Street reacts - 3
What to expect from the planets in 2026 — key dates and sky events - 4
'I carried my wife's body for an hour and a half' - BBC hears stories of protesters killed in Iran - 5
8 Espresso Bean Starting points All over the Planet
Wonderful Sea shores All over the Planet
Instructions to Expand Your Smash 1500's Presentation: Tips and Deceives
10 times the sky amazed us in 2025
Shah Capital pushes for Novavax sale, warns of proxy fight
15 skywatching events you won't want to miss in 2026
Luigi Mangione‘s lawyers say Bondi’s death penalty decision was tainted by conflict of interest
NASA's Perseverance Mars rover could break the record for miles driven on another planet
Nature's Treats: 10 Organic products That Lift Prosperity
The most effective method to Pick the Best Wellbeing Highlights for Seniors in SUVs













