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PDUFA VI Passes the Senate – Now Moves to President’s Desk to be Signed Into Law

The PDUFA VI bill – known as FDA Reauthorization Act in the House (FDARA) – passed the House of Representatives early in July with strong bi-partisan support. It then moved to the Senate but consideration was delayed until after the Senate healthcare reform deliberations were completed. Today, though, we are excited that a companion bill (mirroring the House version) passed in the Senate! It will now move to the President’s desk where it is expected to be signed into Law.

The Prescription Drug User Fee Act (PDUFA) was first enacted in 1992 to supplement FDA’s federal funding through fees collected from industry (user fees) at designated time points in the drug development cycle for critical programs such as the new drug approval process and its infrastructure. Along with the establishment of the user fee structure, Congress was able to insert legislative provisions which focus on accelerating therapy development and enhancing public safety. Noteworthy programs that have come directly from previous versions of PDUFA and changed the drug development landscape include the Accelerated Approval Pathway, the Breakthrough Therapy designation, and Patient Focused Drug Development.

 

PDUFA must be renewed every five years and includes a formal process of negotiations between industry representatives and the FDA. Numerous opportunities for stakeholder engagement have also been critical to the current reauthorization.

 

PDUFA VI through the Lens of the Duchenne Community

 

PPMD has been among the most actively engaged stakeholder groups in the PDUFA VI reauthorization process since negotiations began in July of 2015, including participation and testimony at all public stakeholder meetings conducted by the FDA, submission of public comment to the Federal Register on enhancements that would benefit the regulatory review process from the Duchenne community perspective, and active engagement with both FDA and the industry representatives leading negotiations. PPMD advocates have taken these messages to Congress throughout the year to ensure provisions that impact the Duchenne community and rare disease were taken into account and included in the bill.

 

We are pleased to see many of the provisions we advocated for were included in both the House and Senate versions:

  • Collect and utilize meaningful patient and caregiver input that can inform drug development and, ultimately FDA decision making;
  • Enhance how the FDA incorporates benefit and risk preferences of our community in regulatory decision making
  • Improve the pathway for qualifying biomarkers
  • Enhance the use of real world evidence for use in regulatory decision making;

It Takes A Village – An Engaged, Empowered Village – Thank YOU!

 

The passage of PDUFA VI signifies years of discussions and negotiations. Almost two years of stakeholders coming together to work toward legislation that would support infrastructure and programs aimed delivering newly approved medicines to U.S. patients as swiftly as possible. This was the first time in history that a PDUFA bill was negotiated in one Administration – and then passed under a new Administration.

 

We are incredibly grateful to our advocates who reached out to your elected officials all throughout the negotiations to ensure they understood how critical this bill is to us.

We are grateful to our advocates who joined us here in Washington in February and spent time talking with Members of Congress about the importance of PDUFA.

And we are grateful to our Congressional champions – on both sides of the aisle, in both the House and Senate – who ensured that reauthorization of PDUFA was a priority prior to the August Recess.

 

PDUFA is about innovation for patients – innovation for our Duchenne community. PDUFA VI passed! 

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