It’s that time of year again. Members of the House and Senate are working, right now, to develop and submit their policy priorities for the annual spending bills that will fund the NIH, CDC, FDA, and other key federal agencies for Fiscal Year 2021.
Over the years, requests made through these letters have been indispensable in driving forward much of the progress that has occurred in recent years. This includes:
- Driving forward the development and issuance of Duchenne care standards and expanding Duchenne surveillance.
- Advancing Duchenne research at the National Institutes of Health.
- Supporting the continued implementation of patient engagement policies to ensure the Duchenne patient and caregiver voices are being heard and their input considered by the FDA.
This year’s letter seeks to build on this foundation and will ask for the following:
- Increase funding (by $2 million) to $8 million for the Muscular Dystrophy program at the CDC.
- An evaluation of the Duchenne Muscular Dystrophy Care Considerations, a consideration of the possible relationship between patient outcomes and the presence of a Certified Duchenne Care Center (CDCC), and an assessment of whether we have reduced the delay to diagnosis since establishment of the National Task Force for Early Identification of Childhood Neuromuscular Disorders.
- For NIH to convene a multi-stakeholder workshop to evaluate pre-clinical animal models used frequently in Duchenne treatment research and to consider whether alternative models or strategies may improve therapy development outcomes.
- For the FDA to include in the benefit-risk framework a description of how patient experience data was considered.
- Support level funding of $10 million for the Duchenne Muscular Dystrophy Research Program (DMDRP) within the Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP).
The House letter is once again being led by Representatives Peter King and Doris Matsui in the House and by Senators Roger Wicker and Debbie Stabenow in the Senate. We are grateful to our Congressional Champions for their incredible support of our community!
The deadline for the House letter is March 11 and the deadline for the Senate letter is March 20.
Please reach out today to ensure your members hear from you directly about the importance of supporting the request.Take Action