
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
Your big brain makes you human – count your neurons when you count your blessings - 2
‘The White Lotus’ sparked online interest in risky anxiety pills, study says - 3
Believe Should Unwind? Look at These Scaled down Games - 4
How a niche Catholic approach to infertility treatment became a new talking point for MAHA conservatives - 5
The most effective method to Perceive the Early Side effects of Cellular breakdown in the lungs
Italy now recognizes the crime of femicide and punishes it with life in prison
5 VIPs That Changed Style
The most effective method to Pick the Ideal Shrewd Bed for Your Special Rest Needs
Flu illness count nears 5 million, with New York City among the hardest hit
Washington resident contracts bird flu, first human case in U.S. since February
‘Wicked: For Good’ streaming release — How to watch the sequel starring Ariana Grande and Cynthia Erivo
UN torture cm'tee report flags Israel for allegedly mistreating journalists, detainees, ex-MAG
Innovative Versatility: Examples of overcoming adversity from Entrepreneurs
6 Asian Urban areas to Visit













