Tis the Season, for FDA legislation called PDUFA

The Senate and House are moving closer to taking action on critical legislation focused on the Food and Drug Administration called PDUFA. Every five years Congress must pass a package of policies, known as user fees, that dictate the terms for the pharmaceutical and biotechnology and related industries to help fund the FDA and its review of candidate therapies. The bills often are opportunities to pursue other various reforms of FDA, such as ways to streamline the review process so safe and effective therapies can be provided to patients as quickly as possible.

Most recently, PPMD has offered our thoughts to both House and Senate offices engaged in the process. As you will see in the attached letter to the Senate Health, Education, Labor and Pensions (HELP) Committee, we suggested ideas to help maximize the likelihood that the patient voice will be heard when key decisions on topics like benefit/risk, trial design and trial outcomes are made; and we noted the importance of paying attention to patient-reported outcomes. Both of these points are consistent with the larger policy statement our board approved earlier this year and are ways to achieve our goal of ensuring FDA is ready to hit the ground running when it receives applications for Duchenne therapies.

Also on the FDA front, PPMD recently signed onto multi-organization letters to both the House and Senate authors of the EXPERRT Act applauding their efforts to improve the process of review potential drugs. EXPERRT directs the FDA to ensure that rare disease experts, academic researchers, health professional organizations, patient advocates and care providers have a greater opportunity to offer input and advice to the FDA on rare diseases.

How You can Help - Latest Action Alerts
PPMD has officially endorsed HR. 4132, the Faster Access to Specialized Treatments (FAST) Act. Visit our Take Action page to send a message to your members of Congress today asking them to endorse this important piece of legislation.

PPMD will continue to aggressively advocate throughout the PDUFA process on behalf of specific measures to advance our goals, and will alert all advocates to specific advocacy needs or opportunities.

Stay Informed

Please be sure to sign up for our action alerts to be kept up to date on all advocacy matters. 


Ryan Fischer, Director of Outreach & Advocacy
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