Latest News

STAT Plus: Debate erupts over whether Sarepta’s rare disease drug has enough clinical data to determine cost-effectiveness

A new controversy is emerging over a costly treatment for Duchenne muscular dystrophy, a rare disease that can send boys to an early death, thanks to a new analysis suggesting that clinical data is so limited that it is impossible to determine whether the medication is worth a price tag of $300,000 or more.

The medicine, which is called Exondys 51 and marketed by Sarepta Therapeutics (SRPT), caused a heated public debate over regulatory approval standards and satisfying unmet medical needs during the run-up to its endorsement nearly three years ago by the Food and Drug Administration.

Continue to STAT Plus to read the full story…

Source link

Related posts

AI pathology diagnostic tool developed using deep learning technology from Olympus


Photoacoustic Imaging Measures Temperature Deep Within Body


What Is Giant Hogweed? Plant Leaves Teenager With Third-Degree Burns


This website uses cookies to improve your experience. We'll assume you're ok with this, but you can opt-out if you wish. Accept Read More

Privacy & Cookies Policy