Advocacy

National Ataxia Awareness Day Resolution
International Ataxia Awareness Day is celebrated on September 25. NAF and FARA have successfully advocated for a Senate Resolution also recognizing that date as "National Ataxia Awareness Day." Designating a National Ataxia Awareness Day highlights the impact of ataxia on individuals and their families within the United States and helps accelerate funding, development, and access to effective treatments. This designation occurs yearly. Prior co-sponsors for the resolution: Senator Cindy Hyde-Smith (R-MS) (2021, 2020); Senator Chris Murphy (D-CT) (2021); Senator Elizabeth Warren (D-MA) (2020).

Congressionally Directed Medical Research Program (CDMRP)
FARA and NAF has been working to get “hereditary ataxia” added to the CDMRP, which is run by the Department of Defense. There are over 45,000 veterans who suffer from ataxia and that number multiplies when you add family members who have ataxia. The CDMRP focuses on funding high impact, high risk, and high gain research projects. Since its origin in 1992, the CDMRP has funded $17.8 billion dollars for research. If added, this would provide a new source of funding for FA researchers.
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Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813)
On May 25, 2021, the bipartisan, bicameral Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813) was introduced. FARA actively supported this bill because it had several provisions that would help expedite ataxia research: 1) it established a Health and Human Services (HHS) Public-Private Partnership for Rare Neurodegenerative Diseases between the NIH, the FDA, and eligible stakeholders with a connection to the patient population(s) to advance the understanding of rare neurodegenerative diseases and foster the effective development and evaluation of treatments; 2) it commissions the publication of an FDA Action Plan on rare neurodegenerative diseases; and 3) it created a FDA grant program to fund research and therapy development for life-threatening or severely debilitating rare neurodegenerative diseases. The ACT for ALS was passed by the House and Senate in December and signed into law by President Biden on December 23, 2021 (Public Law No: 118-79). FARA will monitor the implementation of relevant provisions this year.

The BENEFIT Act (S.373/H.R. 4472)
The Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT Act) will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD and related data – including information developed by a product sponsor or a third party such as a patient advocacy organization or academic institution – be considered as part of the risk-benefit assessment. This action will send an important signal to all stakeholders that patient experience and PFDD data will be fully incorporated into the agency’s review process and will encourage such entities to develop scientifically rigorous and meaningful tools and data.
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Increased Federal Funding for Research
Ensuring robust funding with sustained and predictable increases is essential to ensuring that good science moves forward. FARA's top priority is to help secure continued support for our federal partners at the National Institutes of Health (NIH) and the Food & Drug Agency (FDA) so they have the resources necessary to advance promising research and promptly review new drug applications.
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Increased federal funding for research

• Research relies on our federal partners at the National Institutes of Health (NIH) and the Food & Drug Agency (FDA). The NIH is the steward of medical and behavioral research for the National. The FDA is the regulatory authority over new drug applications. The NIH & FDA are complementary in their roles and functions — NIH supports and conducts biomedical and behavioral research and FDA ensures the safety and effectiveness of medical and other products.
• Elected officials will be working on appropriations for the NIH and FDA in April. These agencies are vital to the ataxia drug development programs and they need the resources to appropriately manage the demands of COVID in addition to all the other important research programs they review.

COVID-19 Effects on Research
FARA will continue to address issues arising from COVID-19 that directly impact FA research. Current issues include supplemental funding, access to vaccinations, telemedicine, and application of lessons learned from the pandemic that could expedite drug development.
Info on COVID-19 and Friedreich's Ataxia letter

PDUFA VII: Fiscal Years 2023-2027
The timely review of the safety and effectiveness of new drug applications (NDAs) and biologics license applications (BLAs) is central to FDA's mission to protect and promote the public health – and PDUFA is essential to these efforts. Before PDUFA's enactment in 1992, Americans’ access to innovative, new medicines lagged behind other countries. FDA's premarket review process was understaffed, unpredictable, and slow. The Agency lacked sufficient staff to perform timely reviews or develop procedures and standards to assure a more rigorous, consistent, and predictable process. To tackle these challenges, Congress passed PDUFA, which authorized FDA to collect industry user fees to hire additional staff and upgrade its information technology systems. In return, it committed the Agency to speed the application review process for new drugs without compromising its high standards for new drug safety, efficacy, and quality.
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Newborn Screening Saves Lives Reauthorization Act (S. 350/H.R. 482)
The Newborn Screening Saves Lives Reauthorization Act, H.R. 482/S. 350 was introduced in the House on January 25, 2021 by Representative Lucille Roybal-Allard (CA) and in the Senate on February 19, 2021 by Senators Maggie Hassan (NH) and Roger Wicker (MS). This Act would continue critical federal programs that aid states to improve and expand their newborn screening programs, support parent and provider education, and ensure laboratory quality and surveillance for newborn screening. Authorizations for these programs expired in 2019.
Click here to contact your Members about this bill.

Rare Disease Congressional Caucus
The Rare Disease Congressional Caucus helps bring public and Congressional awareness to the unique needs of the rare disease community (including patients, physicians, scientists, and industry), and creates opportunities to address roadblocks to the development of and access to crucial treatments. The Caucus gives a permanent voice to the rare disease community on Capitol Hill. Find Out if your Representatives are members of the Rare Disease Caucus. Thank your Representatives that are members and Invite those that are not to join.

Rare Disease Advisory Councils
FARA supports all efforts at a state level to include the patient community in decision making. A Rare Disease Advisory Council serves to inform the legislative body and give the rare disease community a voice in programs and policies that would directly impact them.
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Photo credit: National Organization for Rare Disorders

DOES YOUR STATE HAVE A RARE DISEASE ADVISORY COUNCIL?

In 2015, the first rare disease advisory council was created in North Carolina as a result of the rare disease community coming together and demanding a stronger voice in government. With over 7000 rare diseases, it would be impossible for state legislators to understand the challenges rare disease patients face every day around diagnosis, treatment, and access. Additionally, state policies are often determined without consulting the individual disease community that will be directly affected. A state rare disease advisory council provides an avenue for all stakeholders including patients, caregivers, doctors, insurers, drug manufacturers, and researchers to offer insight and recommendations to state elected officials and leaders as those decisions are being considered.

North Carolina sparked similar legislation to be enacted throughout the country. Today, with the support of the National Organization of Rare Diseases (NORD) Project RDAC and other patient organizations like FARA, twenty states have rare disease advisory councils with others currently considering creating one. Kudos to the states who have acted!

A comparison state program would be a state legislative caucus. A caucus is a group of legislators coming together around a common interest, such as rare disease. To learn more, check out these resources from the EveryLife Foundation.

Creating a Rare Disease State Legislative Caucus

Comparing State Caucuses and Advisory Councils

Past Initiatives




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