
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
No respite for German economy as experts slash forecast over Iran war - 2
Step by step instructions to Prepare with Senior Protection for Inward feeling of harmony. - 3
Sea level doesn’t rise at the same rate everywhere – we mapped where Antarctica’s ice melt would have the biggest impact - 4
What to know about voluntary chocolate recall - 5
'The best gift ever': Baby is born after the rarest of pregnancies, defying all odds
A photographer finds thousands of dinosaur footprints near Italian Winter Olympic venue
Hanwha Ocean secures orders worth $866m for five vessels
Starfront Observatories: A haven for distant stargazers
Vote in favor of Your #1 sort of pie
DEA seizes 1.7 million counterfeit fentanyl pills in Colorado storage unit
My Excursion to a Better Way of life: Health Experiences
Everything to know about NASA's moon mission launching this week
Canada's Friendly Sunshine Coast City Is An Outdoor Playground Perfect For Hiking And Paddling
Figure out How to Store Your Gold Ventures: A Thorough Aide safely













