
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
4K televisions for Extreme Film Watching Experience - 2
New subclade K flu strain raises concerns: What families should know - 3
6 Methods for further developing Rest Quality - 4
Solar storms can trigger auroras on Earth. This star’s explosion could destroy a planet’s atmosphere - 5
6 Popular Ladies' Aromas On the planet
Help Your Business with Master Web based Promoting Arrangements
Share your number one city visit transport that leaves a mark on the world wake up!
Visiting This Japanese City Just Got A Little More Expensive (Here's What Travelers Should Know)
The most effective method to Examine a Cellular breakdown in the lungs Finding with Family
Herzog, German Chancellor Merz discuss final Gaza hostage, Arrow 3 exchange in Jerusalem
Washington resident is infected with a different type of bird flu
James Webb Space telescope spots 'big red dot' in the ancient universe: A ravenous supermassive black hole named 'BiRD'
Flourishing in a Cutthroat Work Market: Vocation Methodologies
Picking Your Next SUV: 4 Brands Offering Execution, Solace, and Wellbeing













