As the U.S. Senate continues debate on legislation that seeks to accelerate delivery of safe and effective therapies to patients and to increase the patient voice as part of the therapy review process, PPMD is pleased to offer our enthusiastic endorsement of the legislation.
Over the past several months, PPMD has fought to include several key provisions included in the Senate version of the Food and Drug Administration Safety and Innovation Act, commonly referred to as the Prescription Drug User Fee Act or PDUFA. A number of similar provisions are also included in a House bill expected to be voted on by that chamber next week.
The provisions included in the Senate user fee bill will help expand the patient voice when key decisions are being made, an issue of grave importance to our community and to the larger rare disease community. The provisions will also provide the FDA with additional tools to more quickly review potential therapies for life-threatening conditions, including Duchenne.
PPMD has also supported expanded external expert engagement on key issues such as benefit/risk and trial design, and has urged the FDA to accelerate the timing of various rare disease-focused activities.
As Congress works to finalize the user fee legislation, PPMD calls upon the U.S. House of Representatives to amend their version of the user fee bill to mirror the patient representative provisions contained in the Senate legislation.
The legislative provisions that strengthen the patient voice throughout the FDA review process parallel the FDA Policy position statement adopted by the PPMD Board of Directors in February, and align with the Congressional request made by the PPMD community during the 2012 Advocacy Conference in Washington, DC.
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