THE PATIENT VOICE

Welcome to Issue 5!

Billy Ellsworth just graduated from high school, he even walked across the stage and got handed his diploma, something most of us would take for granted. But if you knew Billy, you’d know that was pretty incredible, because Billy has a rare disease called Duchenne Muscular Dystrophy, or “DMD”. There was a time not long ago when people with DMD would not have lived long enough to graduate high school. 

Part of what has made Billy’s relative health possible is a drug called eteplirsen, or Exondys 51. In September 2016, the Food and Drug Administration granted accelerated approval for eteplirsen because the evidence was positive and DMD patients had so few options for treatment. 

Doctors, patients, even the FDA came to the conclusion that eteplirsen had unmistakable value for Duchenne patients. But there are outside forces, like ICER (the Institute for Clinical and Economic Review) who use algorithms like QALY (“quality-adjusted life-year”) to assign the unthinkable - a monetary value to sick people. ICER routinely undervalues drugs for rare diseases because they are ‘too expensive to justify’, even though drugs like eteplirsen have had significant positive impact on people like Billy. 

That’s why Billy and his mom are fierce advocates for access to treatment, because without that access, who knows where Billy would be right now. Like Patients Rising Now, you too can help the Ellsworths tell ICER to back off and let medical decisions stay between doctor and patient.

-- Terry