What are we asking Congress to do?
This year’s request to Congress is broken into two parts. We are asking members of the House and Senate to:
- Sign the FY23 Duchenne Muscular Dystrophy Letter
- Cosponsor the BENEFIT Act; support its inclusion in the next “PDUFA”
Helpful Resources for Advocates
ASK #1 – Sign Duchenne Funding Letter
Each year, we ask our members of Congress to sign a letter to the Appropriations Committee supporting funding for Duchenne research and programs next year. The Appropriations Committee is the committee that oversees funding the federal government.
This year’s funding letter is led by Sens. Wicker (R-MS) and Stabenow (D-MI) in the Senate and Reps. Matsui (D-CA) and Balderson (R-OH) in the House.
The funding letter requests the following:
- $8 million in funding for Muscular Dystrophy related programs at the Centers for Disease Control and Prevention (CDC).
- $12 million in funding for the Department of Defense (DOD) Congressionally Directed Medical Research Program (CDMRP) for Duchenne research.
- Request that Congress submit Duchenne report language to federal agencies that touch Duchenne. Report language provides guidance to the federal agencies, including CDC, NIH, and FDA, about how to spend funding (your tax dollars) related to Duchenne.
ASK #2 – Cosponsor BENEFIT
We ask that Senators and Members of Congress support the BENEFIT Act, S. 373 led by Sens. Wicker (R-MS) and Klobuchar (D-MN) in the Senate and H.R. 4427 led by Representatives Matsui (D-CA) and Wenstrup (R-OH) in the House.
What does the legislation do?
- This legislation builds on prior laws and will make sure patient engagement information is fully considered as part of the FDA benefit-risk framework, by requiring FDA to disclose how they use or incorporate patient experience data in the review of new therapies.
- Patient Experience Data is data that is collected from patients and families with the intention to provide information about patients’ experiences with a disease or condition, including the impact of the disease or condition or related therapy or, and patient preferences for treatments.