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Parent Project Muscular Dystrophy and Expert Advisory Committee Releases Putting Patients First

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 adaptive approval for serious and life-threatening disorders with significant unmet medical need, using existing authority under current law.
  • Give greater weight to the demonstrated benefit/risk preferences of patients, as well as caregivers in the case of pediatric illness, when making risk benefit determinations.

Pat Furlong , Founding President and CEO of PPMD, hopes that Putting Patients First will illustrate the need for the FDA to accelerate approval of potentially life-saving drugs intended to treat rare disorders, like Duchenne.

Says Furlong, "We have been working with the FDA and other regulatory agencies for the last decade to educate them on Duchenne muscular dystrophy and the catastrophic affect this disorder has on both patients and families. As our relationship continues to grow and build, and with the implementation of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA), we are hopeful that the FDA will work with PPMD and the rare disease community to take advantage of the new opportunities created by this landmark legislation."

PPMD is disseminating Putting Patients First throughout the various regulatory agencies, Congressional offices, and to stakeholders in the Duchenne community.

If you would like to read Putting Patients First and to learn more about PPMD's advocacy efforts, please visit

About the Advisory Committee on Policies to Promote Responsible Access to New Therapies
The Advisory Committee includes a diverse and distinguished group of leading authorities from the nonprofit, academic, medical, legal, and pharmaceutical sectors. Members include Richard Finkel , MD, Chief, Division of Neurology, Nemours Children's Hospital; Marlene E. Haffner , MD, MPH, CEO of Haffner Associates, LLC; Emil Kakkis, MD, PhD, President, EveryLife Foundation for Rare Diseases; Laurie Letvak , MD, Senior Vice President, Novartis Pharmaceuticals Corporation; H. Lee Sweeney , PhD, Chairman of Physiology, University of Pennsylvania; and Susan L. Weiner , PhD, Founder and Board President, Children's Cause for Cancer Advocacy.

About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our  mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to for more information or to learn how you can support our efforts and help families affected by Duchenne.

SOURCE Parent Project Muscular Dystrophy
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