
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
Thousands of genomes reveal the wild wolf genes in most dogs’ DNA - 2
Figure out How to Keep up with Oral Wellbeing During Pregnancy - 3
The Best 15 Applications for Efficiency and Association - 4
German men need approval for stays abroad under military service law - 5
Virtual reality opens doors for older people to build closer connections in real life
An Extended time of Self-Reflection: Self-awareness through Journaling
A mom's viral post is raising the question: Do kids need snacks? Dietitians have answers.
German official report: Teen social media ban faces legal hurdles
Coalition led by Iraqi PM al-Sudani wins parliamentary elections
3 back-to-back storms forecast to bring snow and surges of cold air across the Midwest to the Northeast
Vote in favor of the bloom plan that adds a bit of excellence to your life!
Delta flight bound for Atlanta makes emergency landing after engine issue
A Couple of Modest Guitars for 2024
What's Your #1 Pizza Beating Mix?













