For the first time, patient advocates played a key role in giving advice on how to create new treatments for a debilitating rare disease that targets children.
The Food and Drug Administration released a draft regulatory guidance Tuesday for Duchenne muscular dystrophy, which does not have any known cure. The guidance explains how a drug company can develop drugs for the disease and related ailments.
The disease primarily targets children between the ages of 3 and 5, and causes muscle degeneration and weakness. It is one of nine types of muscular dystrophy, according to the Muscular Dystrophy Association.
Normally the agency writes up guidance, which contains key advice for meeting agency regulations on drug approvals.
But this time, the agency collaborated with a patient advocacy group called the Parent Project Muscular Dystrophy to create the guidance.
The group actually created its own guidance that it submitted to the agency for review. The FDA said Tuesday that it considered the advocates’ thoughts when developing its own guidance.
The disease was at the center of a social media campaign called Be Reasonable that started last month. The campaign sought to get the FDA to accelerate approval for treatments that treat specific mutations of Duchenne.
