
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
Figure out How to Amplify the Resale Worth of Your Kona SUV - 2
Damaged Shenzhou-20 spacecraft to return to Earth uncrewed for inspection - 3
Figure out How to Keep up with and Clean Your Brilliant Bed for Ideal Execution - 4
Flu illness count nears 5 million, with New York City among the hardest hit - 5
6 Shades Brands For Seniors
Public Parks in the USA
From White Elephant to Favorite Things parties, here are all the rules you need to know every kind of gift exchange
Finding the Force of Mentorship: Self-awareness Through Direction
Hostile to Maturing Skincare Items to Rejuvenate Your Skin
6 Robot Vacuum Cleaners for Easy Home Cleaning
Support Your Investment funds with These Individual accounting Thoughts
When preventable infections turn deadly behind bars | The Excerpt
Wellness Bits of knowledge Readily available: A Survey of \Following Wellbeing and Progress\ Wellness Wearables
Top Pastry: What's Your Sweet Treat of Decision?












