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Doing It Themselves for Duchenne

Parent Project Muscular Dystrophy, an advocacy group for patients with Duchenne muscular dystrophy, has put together a draft guidance for pharmaceutical companies in the hopes of spurring drug development for the fatal condition, the Wall Street Journal reports.

A committee of more than 80 people, including parents, clinician, researchers, and drug company executives, wrote the draft guidance and submitted it to the US Food and Drug Administration in late June. The agency is "reading it intently, intensively,'' according to Robert Temple, the deputy director for clinical science at the Center for Drug Evaluation and Research there.

FDA, the Journal notes, typically develops such guidances, but after a series of setbacks in Duchenne drug development, the members of the Parent Project Muscular Dystrophy decided to tackle the issue themselves. In particular, they argue that how the success of a drug is measured needs to be altered and that, as there are many gene mutations that can lead to Duchenne, that more patients need to be eligible for trials.