
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
Two reportedly killed as Israel attacks Hezbollah targets in Lebanon - 2
When darkness shines: How dark stars could illuminate the early universe - 3
Strengthening through Wellness: Individual Preparation Achievement - 4
Exploring the Difficulties of Beginning a Family: Individual Experiences - 5
The Delight of Perusing: Book Proposals for Each Class
80 km. on foot: Sharren Haskel’s three-day march in protest of haredi draft bill
Malaysia To Revive Search for Missing Flight MH370
5 Wellbeing Applications Assist You With remaining Fit
Astonishing Deserts All over The Planet You Really want To Visit
The Main 15 Applications for Efficiency and Association
The most effective method to Look at Medical caretaker Compensations Across Various Clinics
Manual for Financial plan Agreeable PC
Family Matters: Tips and Guidance for Effective Nurturing and Everyday Life
Internet Bookkeeping Programming for Consultants













