There are lots of ways to collaborate, but it all comes down to one thing: the patient organization will help you understand what it’s like to live with the disease. This helps the company understand what needs to be done to develop a therapy and get it to the patients who need it.
How many people live with a given rare disease? How many more remain undiagnosed? Can we develop a formula to predict improvement in diagnosis rates across diseases? A recent article by the International Society for Pharmacoeconomics and Outcomes Research attempts to answer these riddles.
On May 19, the Wall Street Journal revisited industry and patient relationships leading up to FDA approval of Sarepta’s eteplirsen. Chris Smith weighs in.
As hundreds of gene therapies advance through clinical trials, a genomic revolution is taking place before our eyes. Are we ready?