A Wake Up Call For Millions With A Rare Disease

  Posted on July 12, 2017

A Wake Up Call For Millions With a Rare Disease

Just a year ago, there was not a single FDA-approved treatment option available to patients with Duchenne muscular dystrophy. However, thanks to the FDA’s Accelerated Approval Program, a 25-year old FDA process that Congress expanded in 2012, treatments can now get to market faster than the traditional approval process. This expansion potentially shaves off years of the time it takes to get a drug approved in the United States. While this is a major step forward, the rare disease community stands to lose a great deal if insurers continue to deny coverage of FDA-approved treatments. While many point to high prices, the reality is that they treat a fraction of each patient population and will never be a financial burden to insurance companies that make billions of dollars in profits every year.

Approximately 30 Million Americans Have a Rare Disease

If insurers are drawing a line in the sand now, what does this mean for other medicines that come to market in the next few years? According to the National Institutes of Health, it is estimated that 30 million Americans (10 percent of the U.S. patient population) have a rare disease. By restricting and/or denying access, insurance companies and state Medicaid Boards are setting a dangerous precedent that could prevent innovation and medical progress for diseases like DMD, Lou Gehrig’s disease, and rare forms of cancer that have yet to see an FDA-approved treatment. Everyone with a rare condition could eventually be impacted.

Kelly Maynard
President, Little Hercules Foundation
Mom to Jackson