On Friday, PPMD submitted additional comments to the latest version of PDUFA VI.  At this point, there are few members of our Duchenne community who haven’t heard the acronyms ‘PDUFA’ or ‘FDASIA’. But, it’s possible that there are many among us who aren’t aware just how important those acronyms are.


This year our Duchenne community’s Congressional champions have urged the FDA to apply the ‘tools within FDASIA’ to ongoing product reviews. We have circled ‘PDUFA dates’ on our calendars – and learned that thorough product reviews can sometimes extend beyond said PDUFA deadline 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. But what is PDUFA – and why has PPMD spent so much time engaging on this policy area?


Understanding PDUFA


The Prescription Drug User Fee Act (PDUFA) was a law that was first enacted in 1992 that allowed the FDA to fund 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 wit the establishment of this 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 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.




In 2012, PDUFA V was passed and broke new ground by inserting patient perspectives as meaningful and critical through the Patient Focused Drug Development (PFDD) provisions. PPMD immediately became a early adopter of PFDD and issued a white paper entitled Putting Patients First which viewed the PDUFA V agreement through the lens of the Duchenne community. Our leadership continued throughour work in patient preference studies, the Duchenne policy forum in 2013, the community-led Duchenne guidance, and our community’s leadership on the Patient Focused Impact Assessment Act S. 1597 (a bill which passed in the Senate this year and aimed at ensuring that PFDD tools are applied to product reviews).


So, five years have passed since PFDD was innovated and PDUFA V passed. Our landscape has shifted significantly. We have created an extraordinary infrastructure, our community has proven bold innovators, and our industry partners are more committed than ever before. But we still do not have one approved therapy. The urgency within our community is greater than ever before. So, when the opportunity to assess the policy and regulatory framework upon which we rely for all drug development exists – PPMD responds.


Ensuring PDUFA VI Works for Duchenne


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 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.


Last month, FDA shared the proposed provisions for PDUFA VI and invited all stakeholder to respond to the draft provisions through comment to the Federal Register and through participation at a public meeting at the FDA on August 15th. In addition, the FDA invited select stakeholders to participate in discussion panels at the August 15th PDUFA VI Stakeholder meeting and I was among those invited to participate.


To read PPMD’s submitted comment, along with our testimony and media coverage from last week’s meeting, see below.


But here’s the short version, 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. PFDD transformed the drug development landscape. And we must continue to ensure that the framework that the landscape is built upon can support the speed at which it is evolving.


That said, since our engagement around PDUFA VI began last July and we submitted our first comments around PDUFA VI, much has happened in our Duchenne community. We have a robust therapeutic pipeline, nearly 40 companies in our space, sophisticated PFDD tools developed – and arguably the most empowered patient community in the world.


In the last 12 months, our Duchenne community has powerfully engaged in two FDA Advisory Committee meetings and has seen how and whether the PFDD tools are being applied. We have had one product denied approval, one product receive a Refusal To File, one product which met its primary endpoint in the clinical trial denied an accelerated approval pathway, and another product still awaiting a decision more than 80 days after the PDUFA deadline date.


So, while we were thrilled to see the proposed enhancements in the FDA commitment letter and draft provisions, our recent experiences provided us with additional insights and opportunities to further enhance the regulatory process. The comments we submitted today and that I spoke about briefly at Monday’s FDA meeting, reflect this evolving ‘science of patient input.’


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