RARECast: How Rare Disease Patients Can Get their Voices Heard in Drug Discovery and Development

There is increasing recognition of the important role patients can play by providing their insights into the drug discovery and development process. The Penn Medicine Orphan Disease Center and Global Genes will be hosting the second annual Rare Patient Advocacy Symposium in Philadelphia May 19, at the Sheraton University City Hotel, a day-long exploration of how rare disease patients can better get their voices heard in this process. We spoke to David Fajgenbaum, associate director of patient impact for the Penn Medicine Orphan Disease Center and research assistant professor of Medicine at the Perelman School of Medicine, about the changing view of rare disease patients, their emerging role in the drug development process, and the value they can provide to researchers, regulators, and drug developers.

RARECast is a weekly series by Daniel S. Levine. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013, which produces The Bio Report and RARECast podcasts. Read his full bio here.