Patients as Partners—the Future of Orphan Drug Development
If you ask the patient advocates, and according to most we aren’t asking them enough, the growing power of patient communities is going to directly affect the future of rare disease research and orphan drug development. If the patients, families, and advocates we met at the Global Genes 3rd Annual RARE Patient Advocacy Summit are any indication, we would tend to agree.
Connexion Healthcare’s Rare Disease Center of Excellence attended this year’s Pa
tient Advocacy conference on September 11 and 12 in Huntingdon Beach, California, as we do each year as a Global Genes Corporate Alliance member, and came away more confident than ever in the concept of “patients as partners.” You’ve probably heard the phrase before, but have you spoken directly to the driving force behind this idea?
There is no doubt that the number of patients with rare diseases connecting with each other online—referred to as the “e-patient revolution”—is empowering patients and their advocacy groups. Finding someone 10,000 miles away who can relate to your seemingly unique journey as a parent struggling to find the right diagnosis doctor after doctor, year after year, for your sick child suddenly gives you hope. You can seek advice on specialists who can help with diagnosis, find out about relevant advocacy groups, learn about current treatment options, and find information about clinical trials that are underway, detailed information.
We asked a seasoned patient advocate about this detailed information that patients share online. Specifically, we asked about the randomized controlled trials in which they are involved, side effects they are experiencing, etc. “The only people blinded are the study sponsors,” was the answer. Not surprisingly, it seems that patients and their families want to talk openly with others about what is the most important thing in their lives at that moment, and to be as directly involved as possible in finding new treatments for their rare diseases. To many, this means that the future of randomized, controlled trials for orphan drugs is questionable at best.
The feeling among most patient advocacy leaders is that clinical trial designs will need to evolve, with smaller experimental designs. Their goal is simple–to involve themselves in the process now while trial designs are evolving as a result of their online communities, and convince study sponsors that partnering with the patients and their advocacy groups to collaboratively design trials is the future of orphan drug development.
For assistance on increasing awareness of your clinical trials and partnering with patient advocacy groups, please contact our Rare Disease Center of Excellence today (firstname.lastname@example.org).