Advocacy efforts have been moving at such a pace this fall that every draft of this newsletter was quickly outdated with more and more exciting news. So, as we close out 2022 and begin 2023, here's a recap on all the incredible accomplishments from the last few months. |
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United Against Ataxia Hill Day |
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Our last newsletter was sent just before the fourth United Against Ataxia Hill Day which occurred on September 21, 2022. 78 Congressional meetings were held with 88 FARA and NAF advocates participating from 31 states and the District of Columbia. Additional advocates, who could not attend the meetings but wanted to lend their support, reached out to their Members directly during the Hill Day, as well as, the days and weeks after. The result was just amazing! Here were our “asks” and the outstanding results! |
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ASK #1: Broaden the definition on The Congressionally Directed Medical Research Programs (CDMRP) to “Hereditary Ataxia” – last year our advocates were successful in getting FA added to the Peer Review Medical Research Program (PRMRP), one of the grant programs within the CDMRP. Advocates set out to broaden that term to include all hereditary ataxias.
RESULT: President Biden signed the Fiscal Year (FY) 23 Congressional budget on December 29 which listed “Hereditary Ataxia” under the PRMRP! This is huge and will be discussed more below.
IMPACT: Increased funding for ataxia research.
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ASK #2: Pass the Prescription Drug User Fee Act (PDUFA VII)
RESULT: Nine days later, on September 30, PDUFA VII passed!
IMPACT: Increased funding for the FDA. This law authorized the FDA to charge user fees for the next five years (FY23-27), which will provide the necessary resources and funds to expedite new drug applications.
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ASK #3: Secure bipartisan support for a Senate Resolution recognizing September 25 as National Ataxia Awareness Day.
RESULT: Senators Cindy Hyde-Smith (R-MS), Debbie Stabenow (D-MI), and Shelly Moore-Capito (R-WV) introduced Senate Resolution 850 and it was passed by Unanimous Consent.
IMPACT: The Resolution helps to raise awareness of ataxia and educate Congressional Members on the needs of the community.
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ASK #4 Robust and timely funding for federal
research agencies.
RESULTS: Funding for all relevant federal research agencies increased when President Biden signed the FY23 budget on December 29. More details on FY23 budget under Capitol Hill Updates.
IMPACT: Increased funding allows the federal agencies to have the resources necessary to support grant application (NIH), have timely and thoughtful review of drug development (FDA), and invest in innovative new therapies (ARPA-H).
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The day ended with a United Against Ataxia Hill Day Celebration! We hope that you were able to join the celebration or view the recording after, but if you missed it, click below to enjoy. It is short, about 22 minutes, but full of exciting moments including the presentation of this year’s Ataxia Research Champion Awards to Congresswomen Rosa DeLauro and Chrissy Houlahan.
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The momentum around our Hill Day has continued to grow over the last few years and the results speak for themselves. It is through your commitment to learning the issues and your dedication to building Congressional relationships that we are seeing legislative action that directly impacts our ultimate goal – treatments for FA! Your hard work is paying off!
The new year will bring additional opportunities to continue this important work including the celebration of the 40th Anniversary of the Orphan Drug Act. FARA is excited to build on these incredible legislative accomplishments in 2023. The FA community may be small in numbers, but we are powerful in advocacy!
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Fiscal Year 2023 Budget Passes |
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On December 29, President Biden signed, the Consolidated Appropriations Act of 2023 which Congress passed days earlier. This law will fund the federal government for FY23, which ends on September 30, 2023. This budget is a win, win, win for FA research! Relevant sections include:
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“Hereditary Ataxia” is listed in the PRMRP, within the CDMRP – this designation makes FA researchers eligible for the $370 million appropriated to the PRMRP. Although FA is not guaranteed a specific amount, it is not limited to one either. The amount awarded will depend on the success of the grant applications submitted.
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Federal healthcare agencies saw increased funding – HHS received $120.7 billion (up nearly $10 billion), the CDC will get $9.2 billion (up $760 million), the FDA received $6.6 billion (up $226 million), the NIH received $47.5 billion (up $2.5 billion), and ARPA-H will get $1.5 billion to fuel innovative approaches to research.
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Telehealth Flexibilities Extended – provisions enacted during the COVID-19 public emergency which allowed expansive use of telehealth services for Medicaid and Medicare beneficiaries was set to expire. Instead, these provisions will be extended through December 31, 2024.
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Reauthorization of the Orphan Products Grants Program – FA researchers are eligible for this FDA grant program, which includes $5 million under the ACT for ALS.
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Support to improve the current newborn screening program – funding included for a National Academy of Medicine study to review and propose enhancements to the current system.
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Changes to the Accelerated Approval process – which would strengthen this accelerated FDA approval pathway.
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New FDA studies to understand how external expertise is incorporated into their review of new drug applications
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HOT OFF THE PRESSES!
ABLE Age Adjustment Act Just Passed!
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ABLE accounts, which were created in 2014, allow individuals who acquire a qualifying disability by age 26 to save money for specific, disability-related expenses not covered by insurance without jeopardizing their eligibility for Medicaid or other benefits like SSI. The ABLE Age Adjustment Act increased the age by which individuals must have a qualifying disability from 26 to 46, which will expand the ability of people who get diagnosed with FA after age 26 to use an ABLE account to save for disability-related expenses. |
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President Biden selects Inaugural Director for ARPA-H
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Dr Wegrzyn, who holds a Ph.D. in applied biology, was working in the private sector on applied synthetic biology to outpace infectious diseases – including COVID-19. Among her prior roles, she was a program director at the Defense Advanced Research Projects Agency (DARPA) and worked with the federal Intelligence Advanced Research Projects Activity (IARPA). FARA will keep you posted on the development, and hopefully successes, of this historic program.
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After a multiyear campaign, advocates were successful in getting FA added to the Peer Review Medical Research Program (PRMRP), one of the grant programs within the CDMRP, for FY22 (which was passed late on March 15, 2022). Adding FA was a huge accomplishment! The PRMRP was appropriated $370 million dollars in FY22 and, once FA was listed, our researchers became eligible to apply for that money.
Many FA researchers took advantage of this opportunity and applied to the program last spring. In October, the first round of grants was announced and three were for FA research, totaling $839,000! Join us in congratulating:
• Joriene de Nooij, Columbia University Medical Center
• Erin Seifert, Thomas Jefferson University
• Katia Aquilano, Tor Vergata University of Rome
There are still many FA grant applications outstanding, so stay tuned for further announcements.
Conditions are selected for the CDMRP each year when Congress passes the budget. For FY23, which just passed on Dec 23, the terminology was broadened to “Hereditary Ataxia.” FA researchers can still apply under that category, but now other types of hereditary ataxias are also eligible. Grant applications for FY23 will be due in the spring.
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FARA Meets with the FDA Office of Tissues
and Advanced Therapies (OTAT)
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This past fall, FARA had two opportunities to meet with the FDA about gene therapy in Friedreich's ataxia. FARA hosted a first of its kind meeting between the FDA Office of Tissues and Advanced Therapies (OTAT) and multiple industry sponsors developing gene and gene editing therapeutics for FA. FARA coordinated with the industry partners in advance of the meeting regarding the issues of concern to them and covered topics suggested by OTAT. Dr. Wilson Bryan, Director of OTAT, attended along with 22 FDA colleagues.
FARA then had an opportunity to take part in a virtual patient-focused drug development listening meeting hosted by the FDA OTAT. The meeting was designed to hear patient perspectives on gene therapy across different conditions. Several members of the FA community, including a patient, parents of children with FA, as well as the CEO of FARA, were able to speak at the session. The listening session can be viewed HERE. (FA Community members speak at the following timepoints in the recording: Shandra Trantham- 49:52; Randy and Maureen Juip- 2:50:23; and Jen Farmer- 3:35:38.)
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FDA and NIH Announce New Partnership for
Rare Neurodegenerative Diseases
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On September 14, 2022, the NIH and FDA announced their plans to launch Critical Path for Rare Neurodegenerative Diseases (CP-RND). CP-RND is a joint public-private partnership which aims to increase understanding of rare neurodegenerative diseases and develop treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases. To learn more about the Critical Path for Rare Neurodegenerative Diseases, visit the CP-RND webpage.
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