
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: Your Definitive Aide - 2
British Columbia's Secret Lakeside Town With Hot Springs Is 'An Oasis Of Arts, Culture And Relaxation' - 3
From ‘Project Hail Mary’ to Artemis II, spaceflight captures audiences when it centers on people because human space travel is hazardous - 4
Weeks-Long Australian LNG Outage Will Further Tighten Supply - 5
America's Confided in Cooler in 2024
Sahel coups push Africa to top of global democratic declines, report finds
4 Electric Vehicle Brands: Execution, Unwavering quality, and Development
Idris Elba is the king of the stress-watch
Instructions to Shield Your Gold Speculation from Possible Dangers: Fundamental Protections
Wedding trip Objections in the US
Evidence of lost baptismal rite stage uncovered in Byzantine era cathedral near Sea of Galilee
Astonishing Deserts All over The Planet You Really want To Visit
ISS astronauts spy airglow and dwarf galaxy | Space photo of the day for Jan. 13, 2026
HR exec caught on Coldplay 'kiss cam' with boss finally breaks her silence: 'I made a bad decision and had a couple of High Noons'












