If you’re in the biotech business, you’re likely closely following the Sarepta saga, or, at the very least, you’re hearing about it in passing because everyone around you is talking about it.
Sarepta Therapeutics, a Cambridge, Massachusetts-based company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases, currently has its lead Duchenne muscular dystrophy (DMD) product candidate, eteplirsen, under FDA review.
While the FDA should have already made a decision, on May 25, 2016, Sarepta announced the FDA would not complete its review of the new drug application (NDA) by the Prescription Drug User Fee Act (PDUFA) goal date of May 26, 2016. In Sarepta’s case, the delay is positive news, considering its tense history with the agency and the FDA’s overall track record with previous DMD drug candidates.
What makes this case unusual is the pressure the FDA is receiving from the DMD patient community, not to mention some members of congress, to approve eteplirsen. The FDA and the medical community at-large believe the clinical trial results are not sufficient. While the drug has been deemed safe, the data leaves doubt that the drug is actually effective. But if you ask the patients or their families, you’ll hear touching stories of the drug enabling them or their children to walk when they would have otherwise been in wheelchairs.
How did these DMD patients find each other and get organized? The internet, of course.
In today’s digital era, online patient and medical communities are more valuable than ever. Patients can discuss their experiences with others who take the same drug (approved and unapproved), receive advice from physicians, or even interact with the drug companies directly.
For the DMD community, digital outlets and initiatives, such as those run by CureDuchenne, Parent Project Muscular Dystrophy, and Muscular Dystrophy Association (MDA), play a vital role. They give patients, families, and advocates the ability to support each other, promote clinical research, push for drug approvals, and ultimately create visibility into a type of rare disease that historically would have only been heard of through MDA’s infamous “Fill the Boot” campaign or the now defunct Jerry Lewis MDA Labor Day Telethon. In other words, our digital world helps give children with DMD a chance to live better and longer lives.
The Sarepta/eteplirsen story is a great example of how drug companies should embrace the fact that they now have direct access to patients, caregivers, advocates, and even potential patients via digital engagement. Relationships with these kinds of stakeholders should be prioritized and nurtured through an ongoing process of learning about their needs and wants, and providing them with the kind of digital experience that will keep them loyal for life.
Photo credit: statnews.com
