Parent Project Muscular Dystrophy Co-Leads Letter Urging Strongest Patient Engagement & Rare Disease Provisions in …

Letter Signed by Nearly 120 Organizations, Latest PDUFA-Oriented Action

HACKENSACK, N.J., June 13, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), the nation's largest Duchenne muscular dystrophy (Duchenne) advocacy organization co-led a letter to key lawmakers that was signed by 119 organizations and that urges Congress to include the strongest patient engagement and rare disease provisions within the final use fee legislation.

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Developed in partnership with the EveryLife Foundation for Rare Diseases and Genetic Alliance, the letter represents the latest of several actions PPMD has engaged in for months as Congress has developed the user fee package. Other groups signing onto the letter include the National Down Syndrome Society, the Muscular Dystrophy Association, the American Association on Health and Disability, the National Tourette Syndrome Association, and Special Olympics.

The House and Senate both passed user fee bills that included provisions aimed at accelerating Food and Drug Administration (FDA) review of therapies to treat rare diseases and to enable FDA to more aggressively engage external experts in reviewing candidate therapies.

"The House and Senate should both be commended for including a number of provisions intended to accelerate the development of therapies for patients suffering from rare and life-threatening disorders like Duchenne. We are urging that as they negotiate the final bill, they include the strongest possible provisions, particularly those intended to increase the patient voice and to provide the clearest possible guidance on the review of potential therapies for rare conditions," PPMD President Pat Furlong said.

Specifically, the letter urges lawmakers to include a provision from the Senate bill that would deepen patient engagement in the review of medical products, and to include content from the House bill that would provide explicit direction to FDA in developing Fast Track and Accelerated Review guidance that recognizes the small patient populations and other issues. The letter also urges lawmakers to include a provision from the House bill that would incent industry to develop treatments for pediatric rare diseases by providing a voucher the sponsor could use to speed up FDA review of another candidate treatment.

"The fact that more than 110 rare disease organizations have come together to support these provisions is testament to the degree to which they are supported by the community. I hope Congress heeds this message and includes these components in the final user fee bill," Furlong said.

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