Thanks to your advocacy, this week we learned that all of the Duchenne priorities we advocated for this year with Members of Congress are one step closer to being included in next year’s budget.
Whether you traveled to DC for face-to-face meetings with members, or advocated from home through emails, phone calls, and social media – your actions this year have yielded incredible results for Duchenne related funding at the federal level!
So what does this mean?
Every year, starting in February, we work with our champions in Congress to craft legislative language that reflects the specific needs of our Duchenne community – messages you consistently communicated to congress from February-April through meetings, phone calls, and emails.
This annual effort is how we keep the MD-CARE Amendments (MDCA) functioning.
As you know, every five years we need to update the MD-CARE Act (2014 was the last update). The MDCA is not a funding bill; rather, it lays out the framework for what we need to get done for those five years.
So every year we go to Congress and discuss the funding needed for the upcoming fiscal year to match up with the MDCA provisions (pieces of the bill).
With that funding request we also request specific “report language” to accompany to funding bills. When a federal agency is looking at spending money on Duchenne they refer to this language for guidance and direction.
Those agencies that matter to Duchenne include:
- U.S. Food and Drug Administration (FDA)
- Center for Disease Control (CDC)
- National Institutes of Health (NIH)
- Department of Defense (DOD)
And with the passage of the 2014 MDCA Amendments, the:
- U.S. Social Security Administration (SSA)
- Administration for Community Living (ACL)
- Centers for Medicare & Medicaid Services (CMS)
This is why advocacy is a marathon and not a sprint.
And thanks to YOU – our AMAZING community – once again we have achieved SUCCESS! The following language was included in the Senate FY17 bill.
The report language includes the following highlights for agencies that matter to our community.
Center for Disease Control (CDC)
- $6M for Muscular Dystrophy - The Committee acknowledges the efforts by the CDC to collect data about our population through the Muscular Dystrophy Surveillance, Tracking, and Research Network [MDSTARnet] and supports the enhancement of the quality and quantity of that data.
- The Committee also requests a plan for the dissemination of the newly updated care standards including for adults with Duchenne, requesting a report on this within 180 days.
- Finally, The Committee supports the CDC’s engagement on Duchenne newborn screening.
National Institutes of Health (NIH)
- The Committee recommends NIH consider strategies that could lead to the development of combinatorial therapies for Duchenne Muscular Dystrophy.
- The Committee supports facilitating clinical trial readiness and establishing research infrastructure needed to conduct high-quality, efficient trials in Duchenne alongside efforts to improve performance of clinical trial endpoints and develop and validate biomarkers of the disease.
- The Committee also recognizes that there have been significant scientific advances in exon skipping technology since the 2010 NIH/FDA meeting and urges the NIH, in coordination with the FDA and other agencies, to conduct a Duchenne follow-on meeting to examine the current state of the science of exon skipping and targeted therapeutics.
Office of the Director
- The Committee is aware of the updated MD Action Plan released in the winter of 2015, and supports the Plan's recommendation to expand Duchenne research into bone health, endocrine functioning, and cardio-pulmonary functioning.
- Further, the Committee is encouraged that NIH support earlier stage research in Duchenne muscular dystrophy has successfully resulted in a substantial pipeline of new potential therapeutics.
- The Committee urges NIH to increase support for late stage preclinical and early stage clinical projects; and to consider partnering with outside entities, including patient organizations and industry, to facilitate research focused on the rapid identification and qualification of new biomarkers for Duchenne.
Administration for Community Living (ACL)
- The Committee is aware that the ACL is included in the Muscular Dystrophy Coordinating Committee under the Muscular Dystrophy CARE Act Amendments enacted in September 2014.
- The Committee supports programs and initiatives focused on the transitions of persons with Duchenne into adulthood.
- The Committee requests a report within 180 days of enactment of this act on the administration's plans to conduct comprehensive studies focused on demonstrating the cost-effectiveness of independent living programs and supports for persons living with various forms of muscular dystrophy.
U.S. Social Security Administration (SSA)
- The Committee is aware that the Social Security Administration is included in the Muscular Dystrophy Coordinating Committee under the Muscular Dystrophy CARE Act Amendments enacted in September 2014.
- The Committee expects the agency to make data available on the rate at which persons with Duchenne and Becker Muscular Dystrophy utilize SSA pro-grams, particularly those focused on promoting employment and community independence such as the Ticket to Work Program.
Thank you for supporting PPMD's legislative and federal advocacy efforts!
Our community's two decades of hard work has created the infrastructure through which our Duchenne drug development pipeline now flows, and our FY 17 report language reflects the tremendous evolution of our community's priorities.
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