
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
A few Up-to-date Sacks - Stylish Young ladies Shouldn't Miss - 2
New heart disease calculator predicts 30-year risk for young adults - 3
The most effective method to Keep up with Proficient Handshakes in a Computerized World - 4
Instructions to Arrange Your Compensation During Medical caretaker Prospective employee meetings - 5
Pick Your Favored pizza beating
Supercharge Your Remote Work Arrangement with These Game-Changing Instruments
Drones, physics and rats: Studies show how the people of Rapa Nui made and moved the giant statues – and what caused the island’s deforestation
Which Kind of Pet Makes the Incomparable Buddy?
Hamas demanded displaced Gazans pay rent on beach tents amid torrential downpour, IDF reveals
Best Streaming Gadget for Your Home Theater
Allow Innovative Progressions To have a Massive Effect
Find the Specialty of Calligraphy: Dominating the Exquisite Art of Penmanship
The Most Astonishing Arising Advancements to Watch
Ten Awesome Authentic Realities That Will Leave You Interested













