Researchers who develop medicines for rare diseases rely on input from patients and families who live with these conditions. In fact, patients and advocates are becoming more empowered than ever to engage in drug development in meaningful ways. After all, who understands the impact of rare diseases better than people who live with them every day? Specific opportunities include participating in patient registries, providing input during meetings with regulators and giving input into the design of clinical trials.

Learn more about how you can get involved. Visit the Global Genes toolkit “How Patients Can Share Their Voices Throughout the Drug Development Process,” the Rare University course “Understanding Drug Development,” and/or Clinical Trials Transformation Initiative’s “Patient Engagement Across the Clinical Trial Continuum.”

Nicole Boice
Founder and CEO
Global Genes