
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
6 Methods for further developing Rest Quality - 2
Figure out How to Explore the Infotainment Framework in the Slam 1500. - 3
This Canadian crater looks like marbled meat | Space photo of the day for Jan. 6, 2026 - 4
Undeniably popular Historical centers: Where Craftsmanship and History Meet - 5
Vote in favor of your favored spot to peruse
2025 Was Another Exceptionally Hot Year
Instructions to Really Oversee Unsold SUVs in the Auto Business
Guns N' Roses 2026 Tour: How to get tickets, presale times, prices and more
The most effective method to Really Adjust Hypothesis and Practice in Your Brain science Studies
An Extended period of Voyaging Carefully: the World with Reason
6 Famous kind of practice on the planet
Opening Potential: Self-awareness and Long lasting Learning
The Way to Recuperation: Defeating Dependence
An Extended time of Careful Nurturing: Individual Bits of knowledge on Bringing up Kids













