At Long Last, PDUFA VI Hearings Have Begun – But There’s a Twist

Wow...what a week in Washington! President Trump released his budget proposal, the American Health Care Act moves to the floor of the House for a vote today, the U.S. Supreme Court ruled on a landmark case around the rights of students with disabilities within the public school system, and at long last after months of our Duchenne community’s engagement, testimony, Hill visits, and discussions with Congressional Champions – the User Fee Hearings have begun. But like so much else these days, the User Fee negotiations took an unexpected twist. This ‘plot twist’ has made our community’s engagement more important than we ever could have anticipated.


Understanding PDUFA

At this point, there are few members of our Duchenne community who haven’t heard the acronyms ‘PDUFA’ or ‘FDASIA’. This year our Duchenne community’s Congressional champions have urged the FDA to apply the ‘tools within FDASIA’ to ongoing product reviews and we have come to measure the passage of time by ‘PDUFA dates’. And we have heard time and again that the Duchenne community is recognized as leaders and innovators in the patient focused drug development (PFDD) space; a space which was created through PDUFA V.


The Prescription Drug User Fee Act (PDUFA) was a law that was first enacted in 1992 that allowed the FDA to supplement the public health funding provided through annual congressional appropriations by funding the new drug approval process and its infrastructure through fees collected from industry (user fees) at designated time points in the drug development cycle. Along with the establishment of this fee structure, Congress was able to insert legislative provisions that 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. Important to emphasize however is that the User Fee structure yielded from the fact that the FDA did not have sufficient resources to conduct the activities needed at current funding levels – thus User Fees were to be layered on top of federal funding.



PDUFA provisions are updated every five years through a process of negotiations between industry representatives and the FDA, and includes formal opportunities for stakeholder engagement by way of public comment through the Federal Register (FR) and participation in meetings conducted by FDA. PPMD has been among the most actively engaged stakeholder groups in the PDUFA VI reauthorization process since negotiations began in July of 2015, including active participation and testimony at all public stakeholder meetings conducted by the FDA, submission of public comments to the FR 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.


In August of 2016, when the FDA commitment letter and proposed provisions were released, we were very pleased. The draft strongly reflected the engagement and input we had previously had and we feel will certainly build upon the firm foundation laid by PDUFA V. In particular, goals within the PDUFA VI Reauthorization commitment letter of particular importance to our community include: 

  • Enhancing the incorporation of the patient’s voice in drug development and decision-making by facilitating the advancement and use of systematic approaches to collect and utilize robust and meaningful patient and caregiver input that can more consistently inform drug development and, as appropriate, regulatory decision making;
  • Enhancing benefit-risk assessment in regulatory decision-making by including the incorporation of the patient’s voice in drug development and decision-making;
  • Advancing development of drugs for rare disease through building on the success of the Rare Disease Program (RDP) in CDER and CBER by continuing to advance and facilitate the development and timely approval of drugs and biologics for rare diseases, including rare diseases in children;
  • Enhancing drug development tools qualification pathway for biomarkers through enhancing staff capacity, holding public meetings and publishing guidances, developing/revising relevant MAPPS and SOPPS on the qualification process, and publically posting biomarker qualification submissions;
  • Enhancing the use of real world evidence for use in regulatory decision-making through public workshops, pilot studies, and draft guidance;
  • Promoting transparency and communication between the FDA review team and applicant; and
  • Enhancing both the evaluation and communication of post-marketing safety findings related to new drugs.


When our community came to Washington this February we prioritized the PDUFA VI legislation as one of the items that we leant our voice to. These goals within the PDUFA VI legislation are integral to the Duchenne community and we came together to urge Congress to ensure these provisions continue to be included as discussions took place around this bill.


Our Hill Ask in February:

We urge you to support passage of PDUFA VI
We especially support the patient focused drug development aspects of the legislation.

In addition, this year we are working to build on the foundation of Patient Focused Drug Development (PFDD) to enhance the integration of patient experience data within the regulatory review framework.

We need Congress to continue insisting that FDA formally incorporate the patient voice into product review.


PDUFA Talks Begin

Well, this week those formal discussions began in the form of two separate Congressional User Fee Hearings; conducted by the Senate Health, Education, Labor & Pensions (HELP) Committee and the House Energy & Commerce Health Sub-Committee. This is the beginning of a process that will extend throughout the spring, with a passage deadline of late August.


Here’s the plot twist.


In addition to the fact that the climate in Washington is tense these days and the exact same legislators who are charged with passing the User Fee package are also those responsible for our nation’s healthcare legislation, the President’s budget proposed significant cuts to the FDA funding levels and further proposed that the funding instead come from User Fees. To be specific, the budget proposal includes $2 billion in industry fees in FY 18 – more than double the fees that were paid in 2017 and more than were negotiated in the proposals that are currently being considered by Congress. User Fees were not intended to supplant tax-payer support of a federal agency’s commitment to safety and efficacy – but to resource innovative programs and accelerate development.


PPMD has been actively working with our Congressional champions to ensure that the priorities of the Duchenne community are understood. PFDD transformed the drug development landscape for Duchenne and we must continue to ensure that the framework that our landscape is built upon can support the speed at which it is evolving. PDUFA VI is must-pass legislation.


Please help us to continue to support our Congressional champions as they review the PDUFA VI Reauthorization and ensure that they understand how important this issue is to our Duchenne community’s fight to End Duchenne. Stay tuned for Action Alerts from PPMD and if you’re not signed up, sign up today!

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