The Food and Drug Administration Monday unveiled the details of a new policy designed to make it easier and quicker for patients with very rare diseases to get cutting-edge treatments.
The new guidance would enable the agency to approve new treatments for rare diseases based on evidence for a "plausible mechanism" for how the treatment would work. The policy aims to speed the use of state-of-the-art technologies like gene-editing to create treatments tailored to individual patients suffering from diseases that are so rare that it would be difficult if not impossible to conduct a traditional study first.
"For decades families heard the same thing: There are not enough patients. The approval will take too long. You just have to wait for the science to catch up with your child, " Health and Human Services Secretary Robert F. Kennedy Jr. said at a briefing announcing the proposed new policy. "That ends today. Individualized medicine is no longer theoretical."
A Pennsylvania court on Monday said that the state’s constitution guarantees a right to abortion while...
Donald Trump has selected Erica Schwartz to lead the Centers for Disease Control and Prevention (CDC),...
When Bug got home from school one winter afternoon in late 2024, his mother was on...
More than 3.1 million bottles of eye drops sold at major retailers, including Walgreens and CVS,...
