On Tuesday September 15th, several members of the Patient Worthy team were attending the Annual Rare Disease Scientific Workshop hosted by the EveryLife Foundation for Rare Diseases. The topic this year was “Incorporating the patient Perspective in Rare Disease Drug Development” With the mission to “Create a usable, scientifically rigorous, framework for industry to incorporate patient input in rare disease drug development in a quantitative and objective way that is acceptable to the FDA.”
One of the main discussions within this, surfacing throughout the conference, was simply how important the patient voice is, and how it shouldn’t be undervalued. Many executives continually emphasized that there is some knowledge that only patients can supply and that can’t be found anywhere else.
“Patients are experts in what it’s like to live with their disease”- Sara Eggers, Ph.D. , CDER, FDA
Patients bring “knowledge of what these diseases are actually like…they provide a reality check” –Philip J. Brooks, Ph.D , NCATS, NIH
“An organized patient voice is necessary and important for regulatory change” – Pat Furlong President and CEO, Parent Project Muscular Dystrophy
You probably already KNOW you’re an expert.
Rare disease patients often become amazing researchers: they know the best physicians to see, the medications that work best for them, and lifestyle changes that help them cope. They become, unfortunately, accustom to having to explain to most people, even general practitioners, the nature of their disease.
When they get in touch with other patients? They’re unstoppable. Patients have created patient registries, bio banks of samples, and patient surveys. Patients support each other, raise funds for research, and at times even create their own biotech companies.
You can probably give far better advice to someone who has just gotten their diagnosis regarding the reality of living with your disease than top medical researchers could. Your knowledge is priceless and indispensable.
It’s nice to know that leaders in the pharmaceutical industry, the FDA and the NIH are recognizing what you’ve already known, that you’re experts as well. Big leaps in health care and pharmaceuticals couldn’t happen without you.
Read more about the conference here or check out some of our live tweets!
