Parent Project Muscular Dystrophy (PPMD) is a patient advocacy foundation focused on advancing research that will lead to treatments for Duchenne and Becker muscular dystrophy (DMD and BMD). PPMD has released Putting Patients First, a white paper outlining recommendations to speed responsible access to new treatments for DMD and other, rare, serious, and life-threatening neurological disorders. This white paper was developed in collaboration with an expert Advisory Committee on Policies to Promote Responsible Access to New Therapies, made up of leading authorities from the nonprofit, academic, medical, legal, and pharmaceutical sectors. Putting Patients First urges the U.S. Food and Drug Administration (FDA) to work with PPMD and the rare disease community to take advantage of opportunities created by the 2012 passage of the Food and Drug Administration Safety and Innovation Act to enhance patient access to new therapies for serious and life-threatening disorders. In implementing the law, PPMD and the Advisory Committee encourage the FDA to “strike a more appropriate balance between clinical certainty and patient access to potentially life-saving treatments.”
