The U.S. Food and Drug Administration (FDA) announced a new process called “Rare Disease Evidence Principles” (RDEP), under which eligible drugs and biologics for ultra-rare diseases caused by known ...

The FDA already authorizes the use of experimental drugs under what’s called “compassionate use,” for people with no other ...

“Designing treatments unique to individual patients has always been the promised goal of personalized medicine. After 25 years the FDA has, for the first time, outlined a framework to facilitate these ...

FDA approval describes when the Food and Drug Administration (FDA) deems a medical product safe and effective for its intended use based on scientific data and testing. Before people use a medical ...

After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...

In a major shift, the US Food and Drug Administration is relaxing a long-standing drug approval requirement for common diseases.

The FDA proposed new guidelines for approving experimental therapies for rare diseases without large group studies.

Scientific, political pressures colliding at FDA, delaying rare disease treatment for patients who have few or no options, experts said at CNBC Cures Summit.

In a highly unusual move for a normally buttoned-up regulatory agency, a senior official with the US Food and Drug Administration held a call with reporters Thursday to defend the agency’s recent ...

Pharmaceutical Executive: Can you discuss FDA’s history with complex plants? Joel Stanley: In 2004, FDA released its first botanical drug guidance to the industry. This was the first time that complex ...

Once the active ingredient is defined, an Applicant must select which patent, or patents, to put forward for extension. The statute permits only one PTE per regulatory review period, so the choice is ...