
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
Must-Sit in front of the Programs from Europe and the US - 2
Man who grabbed Ariana Grande at 'Wicked: For Good' premiere also rushed Katy Perry onstage this year. Who is he and why is he doing this? - 3
Figure out how to Team up with Your Auto Crash Legal advisor for Best Outcomes - 4
Viable Correspondence: Building Solid Connections - 5
Tasting America: An Excursion Through Darling Cheap Food Brands
Vote in favor of your Number one method for praising a birthday
Different Film Classification: What's Your Go-To for Amusement
Figure out How to Take part in Open Conversations Around 5G Pinnacles
Grasping Wrongdoings and Crimes: A Correlation
5 Fundamental Ways to employ a Criminal Legal counselor
The Force of Care: Living with Goal
Fundamental Home Machines: An Easy to understand Determination Guide
4 Electric Vehicle Brands: Execution, Unwavering quality, and Development
The Leonid meteor shower is peaking early this week. Here’s what to know












