
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 single shot of HPV vaccine may be enough to fight cervical cancer, study finds - 2
Benedict Cumberbatch takes on something even Sherlock can’t solve: male grief - 3
Worldwide Objections Ideal For A Golf Outing - 4
Instructions to Upgrade the Mechanical Highlights of Your Shrewd Bed for a Superior Night's Rest - 5
Former 'Bachelorette' welcomes 1st baby via emergency c-section
An Extended period of Voyaging Carefully: the World with Reason
Spain and Catholic Church agree to compensate sex abuse victims
Agios Pharma shares jump as US FDA expands approval for its blood disorder drug
The Craft of Computerized Detox: Individual Trials
This Tiny Bright Yellow Frog Is One of the Most Toxic Animals on Earth
When a sperm whale gives birth, the mother gets help from her friends
Picking the Right Pot for Your Plants: An Aide for Plant Devotees
Germany paves the way for tighter EU asylum rules
Europe could get 42 more days of summer by the year 2100 due to climate change













