Ongoing Legislative & Public Policy Initiatives
Federal Funding for Duchenne Research & Programs
Every year PPMD advocates head to Washington to ensure the agencies that matter most to Duchenne – the Center for Disease Control and Prevention (CDC), the Centers for Medicare Medicaid Services (CMS), the National Institutes of Health (NIH), the Food and Drug Administration (FDA) , and the Social Security Administration (SSA) – are funded at appropriate levels and include specific report language guiding these agencies when they make decisions about Duchenne programs and priorities. To date, that funding has yielded $500 million specifically for Duchenne research.
The issue of healthcare is obviously central for our community. Throughout the current Administration’s debates and discussions, we have wanted to ensure our voice was heard above all the noise in Washington. Our core message was to urge Congress not repeal the Patient Protection and Affordable Care Act until there is a guarantee that communities like ours will continue to have healthcare access that is to equal or better coverage than we do now. This has never been a partisan issue for PPMD. We view all legislation and politicies through the lens of Duchenne community priorities.
Our Advocates have repeatedly reached out to Members of Congress with the following key messages, reflecting the shared healthcare priorities of those within our Duchenne community:
- Ensure meaningful and affordable access
- Ensure affordable and predictable out-of-pocket costs
- Maintain ability for dependents to remain on guardian’s coverage through age 26
- Ensure coverage for pre-existing conditions
- Elimination of annual and lifetime benefit caps
Both the House and Senate proposed versions of healthcare legislation that posed direct threats to most of the above-mentioned community priorities. As benefits and benefit eligibility for our Duchenne community were increasingly in jeopardy, PPMD engaged in discussions with lawmakers and committee staff in Washington – and activated our powerful grassroots networks to ensure that our Duchenne community fully understood the implications of each proposal as it was released.
PPMD will continue to remain engaged in these discussions and ensure that our Duchenne community has a seat at the table as our nation’s leaders work together to craft healthcare policies that reflect the needs of all Americans – including those with Duchenne.
Duchenne Newborn Screening
Since 2014, PPMD has convened leading experts in Duchenne and newborn screening (NBS) to build a national Duchenne NBS infrastructure in the U.S., as well as the evidence to support a nomination for Duchenne to be added to the federal NBS screening panel. The Duchenne NBS effort has established the partnerships required to research, pilot, and implement nationwide NBS for Duchenne. Through these efforts we have begun to create information technology tools to support the development of screening and diagnosis technologies, as well as to enable longitudinal studies to understand the health outcomes of newborns diagnosed and treated early.
Once developed and implemented, the tools will be available for population-based NBS and state NBS program implementation. An essential component of nationwide Duchenne NBS is the nomination to and acceptance by the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) for the Secretary’s Recommended Unified Screening Panel (RUSP). We have also been active in legislative efforts around the reauthorization of the Newborn Screening Save Lives Act and federal funding for U.S. NBS.
PPMD has funded the Duchenne NBS Infrastructure and multiple associated efforts. This PPMD effort is focused on ensuring that all families and clinicians have access to uniform educational and training materials, and those diagnosed and treated are followed long-term. We believe that the use of a centralized and established infrastructure for NBS pilots will accelerate this generation of evidence, the submission of a RUSP nomination packet, the review and recommendation for RUSP status, and ultimately nationwide NBS.
PPMD will continue to remain committed to supporting infrastructure and leading policy efforts around Duchenne NBS.
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, Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) introduced the Better Empowerment Now to Enhance Framework and Improve Treatments or the BENEFIT Act (S. 1052) which passed in the Senate in August of 2017.
This legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, 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 will send an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency’s review process and will encourage such entities to develop scientifically rigorous and meaningful tools and data.
The BENEFIT Act will also enhance an important transparency and accountability provision included in the 21st Century Cures Act by requiring the FDA to say how such patient experience and PFDD data was considered within the benefit-risk assessment for any approved therapies. This will provide additional learnings to all stakeholders, particularly patients, and help further refine and develop such tools going forward.
We look to now move the BENEFIT Act forward in the House of Representatives.
Access & Coverage Advocacy
With the first two FDA approvals of Duchenne therapies, the Duchenne community has entered a new environment for access and reimbursement. With this new and complex environment at play, PPMD has been leading the effort to educate and guide the community through these unchartered waters.Learn about PPMD’s Access & Coverage Advocacy