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Parent Project Muscular Dystrophy and Expert Advisory Committee Releases Putting Patients First
Date:4/23/2013

HACKENSACK, N.J., April 23, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) has released Putting Patients First, a white paper outlining recommendations to speed responsible  access to new therapies for Duchenne muscular dystrophy (Duchenne) and other, rare, serious, and life-threatening neurological disorders. PPMD developed this white paper 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." The white paper makes four recommendations:

  • Expand the use of accelerated approval for therapies intended to treat rare diseases, including Duchenne muscular dystrophy.
  • Issue clear guidance outlining the level of evidence required for the use of surrogate endpoints in order to expand the scope of acceptable endpoints, including novel surrogate and intermediate clinical endpoints, used to approve drugs for serious or life-threatening diseases with unmet medical need.
  • Pilot the use of adap
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SOURCE Parent Project Muscular Dystrophy
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