Patient Advocacy Group Drafts Clinical Trial Guidance Document for the FDA

For many of the thousands of orphan diseases, not only is there no effective treatment, but there is no existing FDA guidance on the regulatory requirements for obtaining approval for a new one. The Parent Project Muscular Dystrophy (PPMD) has taken the reins in their hands and submitted the first draft clinical trial guidance document ever submitted to the FDA by a parent advocacy group. The PPMD, which is comprised of parents of children with Duchenne muscular dystrophy (DMD), worked with a wide range of stakeholders, including academics from the U.S. and E.U., industry partners and DMD advocacy groups to draft the document outlining clinical strategies and potential surrogate endpoints for a clinical trial. Hopefully this will encourage other patients groups to follow suit – the ALSA has today announced such an initiative to develop guidance for ALS.

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