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Major win for the Duchenne community

PPMD applauds the President for signing into law the Prescription Drug User Fee Act also known as The Food and Drug Administration Safety and Innovation Act. Most recently, PPMD worked with the Everylife Foundation for Rare Diseases and Genetic Alliance to organize a letter to Congress signed by nearly 120 patient advocacy organizations urging the inclusion of specific provisions within the final legislation. PPMD is grateful for the many actions advocates have taken to achieve this success. We also appreciate our board’s clear and compelling position on FDA engagement issues that produced a board policy and guided our advocacy efforts.

 

This is a major win for the Duchenne community as well as the rare disease community as a whole.

The legislation includes:

  • A provision from the Senate bill that would deepen patient engagement in the review of medical products.
  • 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.
  • 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.

 

These changes have the potential to speed up the delivery of clinical candidates for Duchenne. Along with the MD-CARE Act, this is among the most important pieces of legislation to the Duchenne community.

We are grateful to everyone who helped by contacting your members of Congress to educate them on the importance of the provisions we championed. Your voice made all the difference!


Ryan Fischer, Director of Outreach & Advocacy
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