Patient Perspectives from the FDA Public Meeting: the Impact of Rare Diseases

I recently had the opportunity to attend this public meeting at the FDA to discuss the Patient Perspectives on the Impact of Rare Diseases. This meeting was held as opportunity to bring the voices of patients and caregivers to the table of the Agency

and drug developers so they could further understand the impact of rare disease and bring the much-needed advancement of new treatments. I was also given the privilege to speak on behalf of patients with Amyloidosis and the obstacles patients and caregivers face daily. Similar stories were shared by the overwhelming number of attendees, all highlighting the many common symptoms and struggles; fatigue, pain, sleep and mobility issues, cost and insurance obstacles. It was not lost on this community that developing a treatment for a rare disease can present its unique challenges, given the relatively small number of affected people. This meeting allowed common issues within rare disease to be recognized which in turn may help to enhance drug development, through the creation of better endpoints and more effective clinical trials design. The impactful comments shared are small reminders that the patient voice is the driver to change and meetings like this are progress.

Robyn Himick

Clinical Trial Manager, ARC