Giving Voice to Patients at the FDA

Nearly a year ago, more than 400 members of spinal muscular atrophy community along with industry, clinicians, and researchers convened a patient-focused drug development meeting with the U.S. Food and Drug Administration to give voice to patient views on the impact the rare condition has on their lives and their priorities for treatments. CureSMA, which organized the event, recently issued its Voice of the Patient report based on the meeting. We spoke to Rosángel Cruz, associate research director of clinical affairs for CureSMA about the report, how it is being used, and what other patient groups could learn from CureSMA’s experience.