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

tive 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 ParentProjectMD.org/puttingpatientsfirst.

    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 pharmaceu
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    SOURCE Parent Project Muscular Dystrophy
    Copyright©2012 PR Newswire.
    All rights reserved

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