A Win in Washington Today – For Every Single Person Living with Duchenne

Our Duchenne community is on our way to achieving ANOTHER milestone.


MD-CARE Act passed 2001

MD-CARE Act reauthorized 2008

MD-CARE Act Amended 2014

Heavy Engagement within PDUFA V; FDASIA reauthorization 2012

Today, March 9, 2016 on our way to another banner day with the passage of our Patient Focused Impact Assessment Act (S. 1597) in the Senate HELP Committee!


MD-CARE, FDASIA, PDUFA, ODA and now the PFIA…A veritable ‘alphabet soup’!*


But much more than mere acronyms, these landmark bills have served as the blueprint – designed by our Congressional champions and heralded by our community – that have laid the groundwork for our clinical, research, drug development, and regulatory infrastructure. And each time our community has recognized a resource gap, we have again turned to our congressional champions to design a ‘new plan’, push for new laws and policies, and pave a new path forward.


Today is one more example of such an achievement. Today, our Senate champions Senator’s Roger Wicker (R-MS) and Amy Klobuchar (D-MN) led a heroic effort within the Senate HELP Committee to ensure that the tools our community and so many others have created within the era of patient focused drug development have the regulatory impact they were intended to. The PFIA aims to build on the Food and Drug Administration Safety and Innovation Act (FDASIA) and its many provisions intending to strengthen the voice of the patient throughout the drug and larger medical product development process. FDASIA has catalyzed the movement toward patient-focused drug development (PFDD) and has motivated stakeholders to move toward developing PFDD tools informed through patient engagement.


Put simply, our community has worked tirelessly to fund research, build clinical research infrastructure, certify clinical centers, develop care standards, and most recently, develop tools so that as potential therapies move through the FDA for review, the FDA reviewers would have a lens by which to review them that would reflect what our community considers to be meaningful.




But what we still don’t know, is whether those tools are actually being used by reviewers. Tools such as the benefit/risk data, draft or final guidances, patient experience data, patient preference data, validated patient reported outcome measures, and more.

That’s what we pushed for. A law that would create a feedback loop and transparency – across FDA Divisions – as to whether these tools are being utilized. So that our community, industry, and other interested stakeholder can continue to work to accelerate the drug development and regulatory process – and understand whether our efforts are impactful. In addition to the assessment, the legislation directs FDA to issue guidance to further clarify and inform development of PFDD tools created by patient-industry collaborations.


One Voice


Our community pushed hard for PFIA. Hundreds of Duchenne community members came to Washington – not just once, but TWICE. Enduring the June heat and our frigid February. Thousands of community members reached out to your Senators – time and again. And our Duchenne community was joined by many others! The EveryLife Foundation brought more than 200 advocates to the Hill last week who helped to champion PFIA and more than 60 other organizations have signed on in support of this bill. Proving once again that our rare disease community is mighty.


But, we’re not done yet.


Next Steps


While PFIA has passed the Senate HELP committee, it now becomes a part of the Medical Innovation package. This package of bills serves as the Senate complement to the House 21st Century Cures Act (HR 6). We must now urge the full Senate to pass the Senate Innovation package – or none of the bills contained within this package will become law.


In addition to PFIA, among the other bills of particular interest to our Duchenne community within the Senate Innovation package is the Advancing Targeted Therapies for Rare Diseases Act of 2015 (S.2030). The Targeted Therapies provision (S.2030) clarifies FDA’s existing authority to leverage data previously used in the approval of a targeted drug when approving a new therapy that “incorporates or utilizes the same or similar genetically targeted technology, or the same variant protein targeted technology.” This authority is critically important to accelerating the development of innovations within follow-on exon therapies.


Take Action


Thus, between PFIA, the Targeted Therapies provision, and other elements of the Innovations package, this Senate effort represents real hope for our Duchenne community in moving the needle for rare disease drug development. Please continue to help us ensure success by reaching out to your Senators today and urging them to PASS the Senate Medical Innovation package.


Today was a win for every single one living with Duchenne. Help us turn today’s win into federal law.




*MD-CARE Act –Muscular Dystrophy Community Assistance, Research, and Education Act

FDASIA – Food and Drug Administration Safety and Innovation Act

PDUFA – Prescription Drug User Fee Act

ODA – Orphan Drug Act

PFIA – Patient-Focused Impact Assessment


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