Legislative Advocacy Victories

In the early 90s, a family entering the Duchenne community would have been faced with the fact that almost no one had ever heard of Duchenne, there was very little federal funding into research, no pharmaceutical companies were interested in Duchenne, no formalized data collection or registries to join, and not one human clinical trial was underway.

PPMD changed the Duchenne landscape.

By galvanizing the Duchenne-patient community and bringing our unified voice to Washington, D.C., we fostered relationships with elected officials who became unwavering Duchenne champions on Capitol Hill, have passed banner legislation, and have established collaborations with every federal agency that touches Duchenne. As therapies have moved through development, we’ve told our personal stories to regulators and transformed those stories into quantifiable data. We have catalyzed research funding, and built a regulatory infrastructure for rare disease products that has reshaped the therapeutic, care, and trial environment.

PPMD – through the active engagement of our Duchenne community and strong collaborations with our partners – has placed Duchenne on the global map.

The critical, strategic inflection points in PPMD’s impact on legislation and policy to date have been:

  • Muscular Dystrophy Community Assistance Research & Education Act (MD-CARE Act)
    • MD-CARE Act 2001
    • Paul D Wellstone MD-CARE Act Amendments of 2008
    • Paul D. Wellstone MD-CARE Amendments of 2014
  • Prescription Drug User Fee Act (PDUFA)
    • PDUFA V (2012)
    • PDUFA VI (2017)
  • Patient Focused Impact Assessment (PFIA) Act (2016)
  • 21st Century Cures (December 2016)
  • Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act (Passed Senate August 2017)

Muscular Dystrophy Community Assistance Research & Education Act (MD-CARE Act)

In 2001, PPMD achieved another stirring victory when the MD-CARE Act was signed into law. It is also the only condition-specific legislation to become law in recent years. This bill has dramatically increased the National Institutes of Health’s (NIH’s) investment into muscular dystrophy research and helped to strategically coordinate the federal agencies that impact Duchenne. The bill was later re-authorized in 2008 and updated in 2014.

Since the MD-CARE Act in 2001, over $500 million has been invested in Duchenne by the federal government.

Our collective efforts helped to establish the Care Considerations, or standards of care, in Duchenne for the first time in history.

MD-CARE Act 2001


  • MD Centers of Excellence – to research MD
  • MD STARnet tracking and surveillance – to understand the incidence, prevalence of Duchenne and Becker muscular dystrophy
  • Muscular Dystrophy Coordinating Committee (MDCC) – an interagency federal committee comprised of federal agencies that impact Duchenne, as well as designated advocacy organizations (including PPMD) that work together to identify national priorities for muscular dystrophy
  • The Action Plan for Muscular Dystrophies – established by a working group of the MDCC

MD-CARE Act Reauthorization 2008

  • Added the National Heart, Lung, and Blood Institute (NHLBI) to the MDCC
  • Enhanced clinical research congressional directed by the MD-CARE Act
  • Expanded MD STARnet to collect data on teens and young adults with Duchenne and Becker muscular dystrophy
  • Duchenne Care Considerations – develop and disseminate

MD-CARE Act Amendments 2014

  • Expanded research to focus on endocrine, pulmonary, cardiac, and transitions
  • Additional federal agencies were added as members of the MDCC to reflect the adult Duchenne community (Social Security Administration, Agency for Community Living)
  • Expansion of CDC Duchenne Becker Care Considerations to include adults with Duchenne and reflect advances in care since first published in 2010
  • Update of MD Action Plan by MDCC

Prescription Drug User Fee Act (PDUFA)

The Prescription Drug User Fee Act (PDUFA) was a law that was first enacted in 1992 that provided the FDA with additional funding support for a critical new drug approval process and infrastructure through fees collected by FDA from industry (user fees) at designated time points in the drug development cycle. Through the establishment of these user fees, along with the establishment of a fee structure, Congress was able to insert legislative provisions focused on accelerating therapy development and enhancing public safety. Thus was born PDUFA – a law that is reauthorized every 5 years and that has established much of the innovative product development infrastructure and initiatives that have revolutionized drug development for rare diseases like Duchenne.

Noteworthy programs that have come directly from PDUFA and that have changed the drug development landscape include the Initiative.

The 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 was engaged in those negotiations and, once passed, immediately became a early adopter of PFDD and issued a white paper entitled Putting Patients First (PDF) which viewed the PDUFA V agreement through the lens of the Duchenne community.

Our leadership continued through our 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).


As the negotiations began around the next PDUFA agreement (PDUFA VI) in late 2014, our landscape had shifted significantly.

We had created an extraordinary infrastructure, our community had proven bold innovators, and our industry partners were more committed than ever before. But we still did not have one single approved therapy for Duchenne. The urgency within our community was greater than ever before. So, when the opportunity to assess the policy and regulatory framework upon which we relied for all drug development came – PPMD responded.

PPMD was among the most actively engaged stakeholder groups in the PDUFA VI reauthorization process. Negotiations began in July of 2015 and included opportunities for 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.

When the FDA commitment letter and proposed provisions were released – and PDUFA VI was ultimately passed into law – we were very pleased with the provisions that were included. They strongly reflected the engagement and input we and others had throughout the discussions and will build upon the firm foundation laid by PDUFA V. PFDD transformed the drug development landscape, and the provisions within PDUFA VI are focused on ensuring that framework serves as a foundation that can be built upon and withstand the speed at which it is evolving.

That said, much has happened in our Duchenne community in the last two years. We have a robust therapeutic pipeline, more than 40 companies in our space, sophisticated PFDD tools developed – and arguably the most empowered patient community in the world. So, while we are pleased with the provisions within PDUFA VI, our recent experiences provided us with additional insights and opportunities to further enhance the regulatory process and we will continue to work in an anticipatory capacity – with FDA and other stakeholders – to ensure that PPMD is evolving the ‘science of patient input’, even as the PDUFA VI provisions are being implemented.

21st Century Cures

The 21st Century Cures Act codifies PFDD as a part of FDA’s Mission.”
Janet Woodcock, speaking to patient advocacy community leaders at a Listening Session with FDA Commissioner Califf on December 13, 2016

It was an effort three years in the making, launched by bipartisan leaders, Representatives Fred Upton (R-MI) and Diana DeGette (D-CO). This bold initiative, called 21st Century Cures, set in motion months and months of proposals for sweeping changes to research infrastructure and academic systems that includes stakeholders from all segments of the research discovery and healthcare ecosystem.

And at the beginning, middle, and end of the effort sat the patient community. From Pat Furlong’s participation in one of the first Congressional Hearings held on Capitol Hill; to Duchenne families and clinicians participating at 21st Century Roundtables throughout the nation; to PPMD convening Congressional briefings, engaging with BIO, PhRMA, and the FDA; and drafting legislative language that eventually became a provision of the final bill – PPMD and our Duchenne community were at the heart of 21st Century Cures.

Our PPMD team spent three years on the Hill sharing the language that we put forward to help take the patient focused drug development (PFDD) effort to the next level. We worked closely with all of our coalition partners from other rare disease groups, to coalition organizations, to industry partners.

And ultimately, as a community we rallied powerfully in front of our Congressional champions to show them that we all believe that therapy development begins and ends with patients. But more, that patient-experience IS data. That patient needs and perspectives are quantifiable and meaningful and powerful.

 “I believe that patient engagement is the ‘blockbuster drug of the century’ – helping patients live longer, richer, more meaningful lives.”

Pat Furlong, PPMD 21st Century Cures Congressional Hill Briefing

Patient Focused Impact Assessment (PFIA) Act

The FDA Safety & Innovation Act (FDASIA) and PDUFA contained a number of provisions intended to strengthen the meaningful engagement of the patient and other critical voices within the medical product review process. These provisions included the strengthening of existing tools and the creation of new ones needed to fill gaps, policies to allow for greater engagement of external experts in the review process, and a requirement for FDA to engage in multiple stakeholder meetings on the important issue of benefit-risk assessments. While patient stakeholders strongly supported these reforms, concerns exist as to the impact these policies are having and how these new tools are being used by the FDA.

To address these concerns and ensure that the FDA uses existing and future patient-focused drug development tools and authorities to the greatest extent possible, Congress worked with PPMD and partner communities to enact an additional reform focused on achieving this goal. The Patient-Focused Impact Assessment Act (PFIA) required the development of a patient engagement assessment tool whose results would be included within the publicly disclosed safety and effectiveness data package of any approved drug. The assessment will be a simple and straightforward survey that asks reviewers how a number of PFDD tools and authorities informed their decision on a new drug application. Topics would include benefit/risk data for the indicated populations, draft or final guidances, patient-preference data, patient-reported outcomes data, and the views of patients and other external experts on the application. The assessment would shed light into how PFDD tools and authorities are or are not being used and would help identify gaps or shortcomings in the space. The FDA would also compile a report summarizing the agency’s use of PFDD tools and authorities within applications reviewed during the preceding year.

PFIA passed in the Senate and became a part of the PFDD section of the 21st Century Cures Act (provisions S. 3001, 3004, and 3005) in 2016.

PFIA has now been fully implemented within the FDA’s product review as a new ‘Patient Experience Data’ section was added to the FDA’s product review in January of 2018. The new review section includes a table outlining the patient experience data relevant to the application, including check boxes for the kinds of information (such as patient-reported outcomes, qualitative studies, or natural history studies) submitted by the applicant, and where that information is discussed in the agency’s review documents. The table also identifies other patient experience data that was considered in the review but not submitted by the applicant.

Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act

While much progress has been made though previous legislative efforts, some significant gaps remain. One such gap is the lack of any requirement in law today that the FDA include as part of its benefit-risk framework any patient experience or patient-focused drug development (PFDD) data. This means that the agency’s signature tool for evaluating benefit-risk does not have to include data from the patient perspective that could be critical to informing the agency’s evaluation and, ultimately, decision on whether or not to approve a product.

To address this gap, Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) and Representatives Doris Matsui (D-CA) and Brad Wentrup (R-OH) have introduced the Better Empowerment Now to Enhance Framework and Improve Treatments or the BENEFIT Act (S.373 and H.R. 4472).

The legislation would amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, patient-focused drug development (PFDD), and related data – including information developed by a product sponsor or a third party such as a patient advocacy organization or academic institution – be considered as part of the benefit-risk assessment. This action would send an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency’s review process and would encourage such entities to develop scientifically rigorous and meaningful tools and data.