- 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
Discussion on deployment of foreign troops ongoing, two sources tell 'Post' - 2
'Stranger Things' character guide: The nerds, the newcomers and the rest of the Season 5 cast - 3
Qatar, Ireland accuse Israel of using chemical weapons on Palestinians, demand watchdog probe use - 4
How to watch the 2025 Macy's Thanksgiving Day Parade for free - 5
Young Muslims in Germany feel left out of Mideast debate, experts say
Vote In favor of Your #1 Method for lessening Pressure
Experience Arranging: Planning for Epic Excursions
Figure out How to Consolidate Cutting edge innovations in Senior's SUVs
More people are addicted to marijuana, but fewer of them are seeking help, experts say
Vote in favor of the Top Vegetable for Senior
RFK Jr. says he's following 'gold standard' science. Here's what to know
All that You Want to Be familiar with Dental Inserts Centers
Discovering a sense of harmony: Individual Accounts of Reflection and Care
Getting through a Lifelong Change: Individual Examples of overcoming adversity












