In an article dated September 19, 2016, Muscular Dystrophy patients and advocates, with the help of select members of Congress, celebrated the FDA’s approval of Eteplirsen, a drug manufactured by Sarepta Therapeutics for treatment of a type of muscular dystrophy that predominantly affects boys in their childhood. Advocates fought heavily for the approval even though there are limited clinical trials to confirm the drug’s effectiveness. While the drug may help up to 12,000 Americans affected by the disease, critics worry that the FDA has set a “dangerous precedent” by its decision.
From NYTimes.com, “F.D.A. Approves Muscular Dystrophy Drug That Patients Lobbied For”
Excerpt from article:
[I]t was taken as a deeply troubling sign among drug policy experts who believe the F.D.A. has been far too influenced by patient advocates and drug companies, and has allowed the delicate balance in drug approvals to tilt toward speedy decisions based on preliminary data and away from more conclusive evidence of effectiveness and safety. . .
To read full article: F.D.A. Approves Muscular Dystrophy Drug That Patients Lobbied For