Transitions are hard. That’s the truth whether you’re talking about a new school year, a new job, or even a new Congress. And, in particular, new Presidential Administrations are always complex political climates in Washington.
But this year, our Duchenne community came together once again and embraced the challenge of this latest transition. With so many new Members of Congress and Federal Agency leadership in flux, we knew that our 2017 February Advocacy Conference and local outreach was more important that ever before.
And once again we came together around concise messaging to ensure that existing Congressional Champions continued to view policies through the lens of Duchenne and that newly elected Members began to understand our community’s priorities. Messaging around accelerating therapy development, ensuring that patient experience data is incorporated into regulatory review processes, access to appropriate health care, and access to emerging therapies. In addition, PPMD and our advocates worked hard to ensure that Duchenne-specific language reflecting our priorities would be included in the annual Appropriations bill (a bill that directs federal spending and activities of health & human services agencies).
Earlier this week, the House Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies passed the FY 18 Labor HHS bill. Included were the following elements of the Report language we proposed (MOST of it!):
Duchenne Muscular Dystrophy.—The Committee is encouraged by FDA’s recent approvals of therapies to treat Duchenne Muscular Dystrophy and is aware of the authorities within the 21st Century Cures Act that clarify the agency’s ability to allow sponsors of targeted rare disease therapies to use data from previously approved applications. The Committee is aware of the relevance of this policy to Duchenne and other rare disease therapy development and of the agency’s work to implement the law. The Committee requests an update on these efforts within 180 days of enactment of this Act, including a description of any challenges or impediments faced by the agency in implementing these new authorities.
(Includes $6 million for the muscular dystrophy program at the CDC)
Duchenne Muscular Dystrophy.—The Committee expects NCBDDD to continue its work to update, evaluate, and disseminate the revised Duchenne care standards; to expand surveillance of Duchenne via the MD-STARnet, to develop a Duchenne newborn screening program; and to support refinement of an International Classification of Disease (ICD 10) code for Duchenne and Becker Muscular Dystrophy.
Duchenne Muscular Dystrophy.—The Committee is aware of stakeholder efforts to achieve validated and qualified biomarkers for Duchenne Muscular Dystrophy. The Committee supports these activities and urges NINDS to work with other Institutes and with the Food and Drug Administration to provide the necessary guidance and to assemble a workshop of all stakeholders to advance this work. The Committee is also concerned about a lack of access to Federally-funded data, such as imaging and biomarker data, which could support qualification of an MRI imaging biomarker and directs NIH to ensure all Federally-funded Duchenne investigators are in full compliance with the data sharing requirements included in the 21st Century Cures Act. The Committee also remains optimistic about the potential for additional Duchenne treatments using exon skipping splicing and encourages NIH to work with other Federal agencies to convene a workshop on exon skipping in Duchenne and other neuromuscular diseases.
Also under the FDA language, the following was included which was influenced by work of our community and applies to Duchenne:
Expedited Programs.—The Committee directs the FDA to exercise its current law authorities, as provided under the FDA Safety and Innovation Act (FDASIA) and the 21st Century Cures Act, when reviewing new drug applications for patients with 100 percent fatal and debilitating diseases. FDASIA expanded FDA’s authorities and strengthened the agency’s focus on accelerating the approval of drugs that treat unmet medical needs, prioritizing the patient perspective in evaluating new drugs and treatments and providing reviewers with flexibility when evaluating drugs for a life-threatening illness.
What does all of this mean?
Well, first it means that our Duchenne community is stronger than ever before and that coming to Washington to advocate matters. THANK YOU!
Second, it means that our community’s Congressional Champions are listening. The time you spend calling, emailing, sitting with them is impacting the way they shape policies and budgets. Thank them. Keep in touch with them. Invite them to local events, .1Ks, Certified Duchenne Care Center Openings, golf tournaments, or to join you in a Run For Our Sons race. Talk to them about Duchenne Action Month and see if they can help you get a Proclamation acknowledging World Duchenne Awareness Day on September 7!
We wanted to be sure that you knew that there IS progress being made in Washington, DC – and we ARE continuing to move our Duchenne community’s priorities forward alongside our Congressional Champions.
THANK YOU to everyone who participated in this year’s outreach whether by coming to DC with PPMD in February or urging your Congressional Members to sign on to our Appropriations Letter Dear Colleagues. The relationships you foster matter.