Parent Project Muscular Dystrophy Applauds FDA Proposals to Encourage Therapies for Rare Diseases

Sep 07, 2011, 12:32 ET from Parent Project Muscular Dystrophy

Largest National Duchenne Organization Responds to FDA's "PDUFA Reauthorization Performance Goals and Procedures, Fiscal Years 2013 Through 2017"

HACKENSACK, N.J., Sept. 7, 2011 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), applauded the FDA's "PDUFA Reauthorization Performance Goals and Procedures, Fiscal Years 2013 Through 2017" (PDUFA V).  In addition to setting out the anticipated fees the agency will collect from industry applications, the proposed PDUFA V agreement includes performance goals, dispute resolution procedures, drug safety enhancements, and regulatory science improvements that FDA and industry agree to pursue for the duration of the agreement.


PPMD is very pleased that the FDA's proposed agreement seeks to address the unique challenges associated with developing therapies for Duchenne and other rare diseases, in particular the inclusion of a proposal for advancing development of drugs for rare diseases.  Specifically:

  • To increase the size of FDA's rare disease programs, including targeted staff training.
  • To increase ongoing outreach to industry and to patient representatives and organizations regarding development of therapies for rare diseases (including public meetings with stakeholder and expert feedback).
  • To develop FDA staff capacity and expertise in reviewing submissions that contain complex issues involving pharmacogenomics and biomarkers.
  • To create a framework for more efficiently and effectively responding to submissions that involve patient-reported outcomes (PROs) and other endpoint assessment tools.

Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, commented on the recent proposal: "PPMD has led the Duchenne community in its advocacy efforts since 2000 with the passage of the MD-CARE Act.  We have looked to Members of Congress and the FDA as friends of this community and have come to count on them to see the urgency in this disorder and to do what they can to help us end Duchenne. This proposal indicates the FDA's ongoing promise to support the rare disease community, allowing for expedited and appropriate reviews and the assurance that the patient's voice will be heard."

Ms Furlong went on to say that, "The FDA sees the need to support clinical trials in rare disease therapies that are happening now.  They know that minutes matter in the fight to end Duchenne and I believe these new measures will make the approval process even more efficient and provide earlier access to breakthrough therapies.  We look forward to working with Members of Congress to advance and fund these critical updates from the FDA."

For more information on Parent Project Muscular Dystrophy's advocacy program, including a history of Duchenne in Washington, please visit

About Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy.  Our mission is to end Duchenne. We accelerate research, raise our voices in Washington, demand optimal care for all young men, and educate the global community. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey.  For more information, visit

SOURCE Parent Project Muscular Dystrophy