by: Parent Project Muscular Dystrophy
As a result of 123 meetings with Members of Congress in Washington DC, and over 2,500 action alert emails sent by this community, we are pleased to report a very successful outcome for the Duchenne Fiscal Year 2021 (FY21) funding letters signatures.
91 members of Congress in total (68 in the House and 23 in the Senate) agreed to support our request sending a strong message of support behind Duchenne research, public health, drug development, and patient support initiatives. Both letters have bi-partisan support, which is really important for these efforts. And this is an increase in signatures from last year!
PPMD is grateful to the Foundation to Eradicate Duchenne for their partnership and collaboration on the Advocacy Conference.
WHAT WAS IN THE REQUEST TO CONGRESS?
Centers for Disease Control
- Asks Congress to increase funding for CDC’s Muscular Dystrophy Program to $8 million.
- With that funding, evaluate the impact of the Care Considerations on patient outcomes, particularly in rural and underserved areas.
- Evaluate the impact of the presence of a Certified Duchenne Care Center on patient outcomes.
- Assess diagnostic odyssey and provider resource needs before and after implementation of recommendations made by the National Task Force for Early Identification of Childhood.
National Institutes of Health
- Requests the NIH convene a multi-stakeholder workshop to evaluate pre-clinical models us to consider whether alternative models or strategies may improve therapy development outcomes.
- Encourages the National Institute of Neurological Disorders and Stroke (NINDS) to develop a centralized resource to store and access de-identified data for further research.
Food and Drug Administration
- Encourages the FDA expand on the implementation of 21st Century Cures to include in the benefit-risk framework a description of how patient experience data was considered.
Department of Defense
- 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).
WHAT HAPPENS NEXT?
The timing for when the funding bill (known as appropriations) is finalized can often be unpredictable, particularly with so much happening with COVID-19, and can be delayed for many reasons – rest assured, they eventually get done. The good news is you have done the hard part already, ensuring the best chance for Duchenne related report language and funding are included for agencies that impact Duchenne in the coming year. We will keep you posted when the bills pass through and are signed into law.
THE BENEFIT ACT: A WORK IN PROGRESS
While on the Hill, advocates were also pushing for passage of the BENEFIT Act. The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act was re-introduced by Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) as S.3385 and was crafted in partnership with PPMD. The BENEFIT Act amends food and drug law to ensure that invaluable patient perspectives – such as input from patient preference studies, patient reported outcomes, and other types of patient experience data – are included as part of the FDA risk-benefit review of candidate new drugs. PPMD will send out additional alerts regarding this bill once Congress re-focuses its attention following COVID-19.
THANK YOU!
We appreciate that your time is precious, and we are grateful to everyone who made the trip to DC and to all those who advocated from home. This community is incredible and continues to lead the way in rare disease advocacy. We are so proud of the work you did and continue to do! We will be in touch with future action alerts related to BENEFIT.
Senate Signatures
| Member | State |
|---|---|
| Kyrsten Sinema | AZ |
| Diane Feinstein | CA |
| Kamala Harris | CA |
| Michael Bennet | CO |
| Richard Blumenthal | CT |
| Chris Coons | DE |
| Dick Durbin | IL |
| Ed Markey | MA |
| Elizabeth Warren | MA |
| Chris Van Hollen | MD |
| Angus King | ME |
| Gary Peters | MI |
| Debbie Stabenow | MI |
| Amy Klobuchar | MN |
| Roger Wicker | MS |
| Kevin Cramer | ND |
| Jeanne Shaheen | NH |
| Cory Booker | NJ |
| Jacky Rosen | NV |
| Catherine Masto | NV |
| Kirsten Gillibrand | NY |
| Sherrod Brown | OH |
| Tammy Baldwin | WI |
House Signatures
| Name | State |
|---|---|
| Terri Sewell | AL07 |
| Doris Matsui | CA |
| Mark DeSaulnier | CA11 |
| Jimmy Panetta | CA20 |
| Ted Lieu | CA33 |
| Mark Takano | CA41 |
| Joe Neguse | CO02 |
| John B. Larson | CT01 |
| Jim Himes | CT04 |
| Jahana Hayes | CT05 |
| Lisa Blunt Rochester | DE01 |
| Ted Yoho | FL03 |
| Stephanie Murphy | FL07 |
| Bill Posey | FL08 |
| Darren Soto | FL09 |
| Brian Mast | FL18 |
| Ted Deutch | FL22 |
| Lucy McBath | GA06 |
| Danny K. Davis | IL07 |
| Raja Krishnamoorthi | IL08 |
| Jan Schakowsky | IL09 |
| André Carson | IN07 |
| John Yarmuth | KY03 |
| Cedric L. Richmond | LA02 |
| Jim McGovern | MA02 |
| Seth Moulton | MA06 |
| Ayanna Pressley | MA07 |
| Stephen F. Lynch | MA08 |
| William R. Keating | MA09 |
| Jamie Raskin | MD08 |
| Chellie Pingree | ME01 |
| Jared Golden | ME02 |
| Elissa Slotkin | MI08 |
| Haley Stevens | MI11 |
| Collin C. Peterson | MN07 |
| Wm. Lacy Clay | MO01 |
| Donald Norcross | NJ01 |
| Andy Kim | NJ03 |
| Josh Gottheimer | NJ05 |
| Tom Malinowski | NJ07 |
| Albio Sires | NJ08 |
| Susie Lee | NV03 |
| Lee Zeldin | NY01 |
| Peter King | NY02 |
| Kathleen Rice | NY04 |
| Jerold Nadler | NY10 |
| Max Rose | NY11 |
| Carolyn B. Maloney | NY12 |
| Antonio R. Delgado | NY19 |
| Anthony Brindisi | NY22 |
| John Katko | NY24 |
| Brian Higgins | NY26 |
| Peter A. DeFazio | OR04 |
| Brian Fitzpatrick | PA01 |
| Dwight Evans | PA03 |
| Mary Gay Scanlon | PA05 |
| Susan Wild | PA07 |
| Glenn “GT” Thompson | PA15 |
| Connor Lamb | PA17 |
| John Rose | TN06 |
| David Kustoff | TN08 |
| Steve Cohen | TN09 |
| Eddie Bernice Johnson | TX30 |
| Colin Allred | TX32 |
| Ben McAdams | UT04 |
| Abigail D. Spanberger | VA07 |
| Peter Welch | VT01 |
| Adam Smith | WA09 |
