Current Initiatives

United Against Ataxia Hill Day

National Ataxia Awareness Day Resolution

NIH & FDA Appropriations

Congressionally Directed Medical Research Program (CDMRP)

The BENEFIT Act (S.526/H.R.1092)

Rare Disease Congressional Caucus

Rare Disease Advisory Councils

Past Initiatives

United Against Ataxia Hill Day

United Against Ataxia Hill Day 2024 will take place September 25, 2024.

Since September 2019, FARA and the National Ataxia Foundation (NAF) have hosted an annual United Against Ataxia Hill Day allowing individuals to meet with their Members of Congress and advocate for action that would positively affect the ataxia community. The impact of this initiative has been monumental, resulting in increasing awareness, appropriation and legislation to facilitate drug development.

United Against Ataxia Hill Day, September 25, 2024

National Ataxia Awareness Day Resolution

As a companion to International Ataxia Awareness Day, FARA and NAF have successfully worked with Senators to recognize September 25 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) (2020-23); Senator Chris Murphy (D-CT) (2021, 2023); Senator Debbi Stabenow (D-MI) (2022-2023); Senator Shelley Moore Capito (R-WV) (2022-2023); Senator Elizabeth Warren (D-MA) (2020).

NIH & FDA Appropriations

Ensuring robust funding with sustained and predictable increases is essential to ensuring that good science moves forward. One of 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.

Congressionally Directed Medical Research Program (CDMRP)

Each year, Congress directs the Department of Defense to support research in designated conditions through the CDMRP program. The CDMRP focuses on funding high impact, high risk, and high gain research projects. There are over 45,000 veterans who suffer from ataxia and that number multiplies when you add family members who have ataxia, all of which face a future without a cure. The CDMRP has provided millions of dollars of ataxia research funding to date, further advancing our understanding of FA and propelling the treatment landscape forward.

The BENEFIT Act (S.526/H.R.1092)

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, Patient-Focused Drug Development (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.

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.  You can find out if your Representatives are members of the Rare Disease Caucus by clicking the link below. 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.

Past Initiatives

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.

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 for 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.

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.