New Voices in Medical Advocacy Often Are Patients

In the world of advocacy for children with disabilities, life-threatening conditions and chronic disease, a new generation of advocates is emerging: the patients themselves.

Christopher “Buddy” Cassidy Jr. was in a difficult spot last year as the sole patient on a Food and Drug Administration panel advising regulators who were considering whether to approve an experimental drug for a rare disease. He spent hours before the hearing reading company and FDA documents, then listened carefully to parents’ testimony that the drug was helping their children and slowing down the progression of the disease, Duchenne muscular dystrophy, which affects primarily males and leads to progressive weakening of muscles and premature death.

But despite the compelling personal testimony, Mr. Cassidy, a 26-year old from Annandale, Va., who has Duchenne, says he worried about the drug’s safety profile. Finding himself on a different side of the issue than many of the parents at the hearing was “wrenching,” he says.

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