- CONTACT US
- AFS
- Business
- Bussiness
- Car
- Career
- Celebrity
- Digital Products
- Education
- Entertainment
- Fashion
- Film
- Food
- Fun
- Games
- General Health
- Health
- Health Awareness
- Healthy
- Healthy Lifestyle
- History Facts
- Household Appliances
- Internet
- Investment
- Law
- Lifestyle
- Loans&Mortgages
- Luxury Life Style
- movie
- Music
- Nature
- News
- Opinion
- Pet
- Plant
- Politics
- Recommends
- Science
- Self-care
- services
- Smart Phone
- Sports
- Style
- Technology
- tire
- Travel
- US
- World

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
How to watch 'A Charlie Brown Christmas' for less with this Apple TV Black Friday deal - 2
This Underrated Italian City Boasts Indulgent Food & Captivating Views For A Romantic Escape - 3
Tens of thousands protest as far-right AfD forms new youth group - 4
Far-right German youth group delegates seek deportations, remigration - 5
Israeli president concerned over proposed renaming of park
Sound Propensities: 20 Methods for helping Your Insusceptible Framework
Manual for Vegetarian Protein Powder
Global measles cases drop 71% in 24 years as vaccination coverage improves, WHO says
The most effective method to Pick the Right Teeth Substitution Choice for You
The most effective method to Examine a Cellular breakdown in the lungs Finding with Family
What to know about new CDC deputy director who has been critical of COVID vaccines
Holiday season sees uptick in norovirus cases, according to CDC
Insurance warning signs in doctors’ offices might discourage patients from speaking openly about their health
A Manual for Pick Viable Psychological well-being Backing Administrations In 2024













