PPMD Leads Critical Letter for Rare Disease Community

Parent Project Muscular Dystrophy (PPMD) has teamed up with EveryLife Foundation for Rare Diseases and Genetic Alliance to galvanize the rare disease community and send a strong message to Congress to ensure the most important provisions for the rare disease community were included in the final Prescription Drug User Fee Act (PDUFA) legislation. The provisions are 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.

With Congress hoping to vote on the final measure before the July 4th recess, 119 rare disease organizations united together in an urgent letter to the key committees tasked with reconciling the legislation in the House and Senate. 

The letter requests lawmakers to include:

• 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.

The Prescription Drug User Fee Act (PDUFA) allows for the FDA to collects fees from drug manufacturers to, in turn, improve and expedite the approval process for drugs. The fees collected are used by the FDA to increase efficiency and timeliness with review of license application and increase their medical and scientific staff capability. We are currently in a reauthorization year of the bill, meaning this is the time to make needed changes in the legislation. These proposed changes have the potential to speed up the delivery of clinical candidates for Duchenne. Along with the MD-CARE Act, this bill is among the most important pieces of legislation to the Duchenne community.

PPMD will continue to keep the community updated as we move toward the final vote of the bill. 

Read the press release.

For a better understanding of what this legislation means for your child, click here.


Ryan Fischer, Director of Outreach & Advocacy

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