
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
French and Malaysian authorities are investigating Grok for generating sexualized deepfakes - 2
Father and son spending Christmas together after health scares - 3
Email Promoting Instruments for Compelling Efforts - 4
Dental Embed Innovation: An Achievement in Helpful Dentistry - 5
Opening Innovativeness: Moving Thoughts and Tasks
Yoshi mania, Happy Meals and not-so-great reviews: A small talk guide to 'The Super Mario Galaxy Movie'
Germany and trade unions kick off tough public-sector wage talks
Eurovision Song Contest changes voting rules after controversial allegations against Israel
These Cities Led Global Jet-Setting In 2025, According To New Data
Vote in favor of your Number one method for praising a birthday
Cheetos and Doritos to launch new versions without artificial dyes
The most effective method to Perceive the Early Side effects of Cellular breakdown in the lungs
Examination In progress into Abuse of Japanese Government-Supported Advance
How did I get my own unique set of fingerprints?













