
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
Go on A Careful spending plan: Modest Objections for Your List of must-dos - 2
Charli xcx recorded original songs for 'Wuthering Heights' — what to know about the new album for Margot Robbie's film - 3
Canada Awards C$1.5 Billion Defense Contracts to L3Harris, Airbus - 4
Satellite observations offer insight into a tsunami's early stages - 5
Kennedy approves adding two rare disorders to newborn screenings
Farewell, comet 3I/ATLAS! Interstellar visitor heads for the outer solar system after its closest approach to Earth
Miley Cyrus flashes a diamond ring on the red carpet, sparking engagement rumors with Maxx Morando: A timeline of their four-year relationship
Geminid meteor shower 2025 peaks next week. Here's what you need to know about this year's best meteor shower
Electric Vehicles for Eco-Accommodating Driving
Extravagance SUVs for Seniors: Solace, Innovation, and Security
Proficient Cultivating Devices for a Lovely and Useful Nursery in 2024
One killed, several injured in Iran missile barrage on southern, central Israel
Becoming amazing at Systems administration: Individual and Expert Tips
Israel strikes Iranian nuclear development facilities, Tehran vows retaliation










