
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
Interstellar comet 3I/ATLAS reveals weird wobbling jets in rare sun-facing tail - 2
From Specialist to Proficient Picture taker: Individual Triumphs - 3
Wellness Bits of knowledge Readily available: A Survey of \Following Wellbeing and Progress\ Wellness Wearables - 4
Quandoo to shut restaurant booking platform by end of 2026 - 5
What are the health benefits of whole milk for kids?
UK to hold fresh pork, other affected Spanish products at border amid African swine fever outbreak
Cygnus XL brings cargo to the ISS for 1st time | Space photo of the day for Dec. 1, 2025
Allow Innovative Progressions To have a Massive Effect
Step by step instructions to Figure out the Natural Effect of 5G Pinnacles
Robert Pattinson claims he's a pathological liar. What 'The Drama' star has said about his 'shtick'
From record warming to rusting rivers, 2025 Arctic Report Card shows a region transforming faster than expected
Ukrainian Army Converts E38 BMW 7-Series Into Multiple Rocket Launch Platform
How did Ariana Grande get her Glinda voice? I’m the man behind the magic.
As reefs vanish, assisted coral fertilization offers hope in the Dominican Republic













