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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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
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Letter to FDA & Reata Pharmaceuticals regard Access to Omaveloxolone
A Call to Action was held from January 5, 2021 – January 20, 2021 where the entire FA community (individuals with FA, family, friends, caregivers, supporters, other rare disease advocates) to sign-on a letter to the Food and Drug Administration (FDA) and Reata Pharmaceuticals. This letter requests Reata to submit a New Drug Application (NDA) on an urgent basis and the FDA to exercise the flexibility granted by law and contained in the FDA guidance in considering approval of an NDA for Omaveloxolone in FA based on the existing evidence from clinical trials.
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FDA & Reata Pharmaceuticals: Allow Individuals with Friedreich Ataxia Access to Omaveloxolone
74,070 individuals signed on to the FA Community Response letter requesting Reata Pharmaceuticals submit a New Drug Application (NDA) on an urgent basis and FDA consider approval of an NDA for omaveloxolone in FA based on the existing evidence from clinical trials.
FARA issued a Call to Action in the form of the FA Community Response letter asking individuals to sign-on in support of a request to FDA and Reata that they work together to approve omav for FA as quickly as possible. FARA shared the FA Community Response letter via our website, email lists and social media channels. Sign-on opportunity was open from January 5-20, 2021.
May 19, 2021 - Reata Announces that The FDA Has Asked The Company to Request a Pre-NDA Meeting for Omaveloxolone for the Treatment of Friedreich’s Ataxia
Click the buttons below to read the full petition and cover letter that were submitted to FDA and Reata Pharmaceuticals.
Community Petition Cover Letter
View the Submitted Petition
51 FA clinical experts and the investigators who participated in the MOXIe studies have provided a separate letter that discusses the clinical significance of the MOXIe trial and omav results.
Healthcare Provider Letter of Support
At the links below, you will find a copy of the FA Community Response Letter and 13 letters of support from FA and Ataxia organizations, writing on behalf of the FA patients they represent and joining FARA in making the request of FDA and Reata to urgently submit a marketing application, approve and provide access to omaveloxolone.
FA Community Response Letter
Letters from Advocacy Organizations
Click a heading below to read more on that topic:
Voice of the Patient
Omaveloxolone targets a specific cellular dysfunction in FA and has been demonstrated in clinical trials to improve disease specific biomarkers and meaningful neurological clinical outcomes and activities of daily living. Omav has also been demonstrated to be safe and well-tolerated.
Given the clinical trial results, FA patient families and clinicians strongly encourage Reata and the FDA to work together promptly to give people with FA, who currently have no other choice for treatment, access to omav as soon as possible. FDA has emphasized the importance of the patient voice, especially for rare conditions without FDA-approved treatment options. As summarized in the report on The Voice of the Patient: Friedreich Ataxia, people with FA experience severely compromised quality of life, loss of independence and early mortality due to symptoms of FA. The majority of patients reported that balance/walking, upper limb function and fatigue have the highest impact on quality of life and treating even one of these individual symptoms would be meaningful. Ninety-five percent (95%) of individuals indicated that slowing or stopping disease progression would be extremely meaningful to them when considering a drug therapy. The patients and clinicians of the FA community are fully aware of the clinical trial results evaluating the use of omav in FA and are convinced that the results demonstrate meaningful benefit and low risk. More than 95 percent of eligible individuals who participated in the clinical trials elected to enter the open-label extension study to continue their access to omav while awaiting formal analysis of the trial data and regulatory review. We ask Reata to submit a New Drug Application (NDA) on an urgent basis and FDA to exercise the flexibility granted by law and contained in FDA guidance in considering approval of an NDA for omav in FA based on the existing evidence from clinical trials.
Community Home Health and Telehealth Policies
In the early weeks and months of the COVID-19 public health emergency, Congress and the Administration took important steps to ensure patients have access to essential care while the nation grappled with controlling the spread of the virus. Specific time-limited regulatory flexibilities have removed significant barriers to care and improved access for the 30 million Americans living with a rare disease or condition including Ataxia. However, these flexibilities are at risk of going away when the public health emergency ends. We urged policymakers to recognize how these flexibilities have benefited members of the rare disease community and consider which policies should be kept in place after the public health emergency ends.
Creating Hope Reauthorization Act
This bill will permanently authorize the Pediatric Priority Review Voucher (PRV) program which will further the opportunity to spur innovation in rare and neglected diseases that disproportionately impact children.
(Photo credit: Duke University - The Fuqua School of Business)
Contacting Elected Officials
The most effective voice in making change is yours. The first step is to determine who are your elected officials. You can search on both the official Senate and House of Representative websites for your federal officials. However, by using one of the below resources, you can also see the district map, see all your federal officials in one place and even learn about some state representatives.
Once you have your list of officials, you can email or call them to discuss concerning matter or request a meeting. Contact information will be listed on their official website or, for federal officials, you can call the US Capitol Switchboard at (202) 224-3121. Both federal and state officials will have local office(s) and one in the capitol where they serve. You can also engage your representatives through social media such as Facebook and Twitter.
Preparing for Meetings
Meeting with your representative can be one of the most effective ways to share your message. A little preparation before will not only strengthen your position but make you feel more comfortable. Here are some helpful hints to get ready.
- Be prepared. Learn about current issues. Check out FARA's 2021 Initiatives for resource information. Read relevant material and/or listen to any webinars offered. Gather materials that support your position and bring them to the meeting. It is up to you to educate the Member on the particular issue or piece of legislation you are addressing. There is no way that they would know or understand what the implications of their legislative actions would be on FA specifically, or rare disease more generally, unless you tell them!
- Develop your “elevator speech.” How would you explain ataxia to someone who has never heard of it? Include a brief explanation of your diagnostic odyssey if pertinent. Think about details of how ataxia impacts your life daily. Tie your experiences to the issue(s) currently being considered.
- Learn about your elected official. Check out his/her website, read his background, see what areas he/she is most interested in. You may find a personal connection (town, school, activity, sports team) that you both share and this could open a conversation. Advocacy is about building relationships and sometimes that happens over the most unusual items in common.
- Be prompt, be concise, be direct. Elected officials, and their staff, have busy schedules but meeting constituents is an important part of the job. Be considerate of their time but confident in why you are there. Tell them your “ask” or what you want them to do.
- Don’t be nervous. They are folks just like you, and in fact, they work on your behalf!
- You will likely meet with a staffer. Do not be disappointed – they are the ones that learn the issues and advise the official. They will also most likely be the person who follows up with you. Good connections with staff will translate to good communications with your representative.
- Make a connection to constituency. Members of Congress want to represent the best interests of their district or state. Who else would be impacted by the action? Would revenue or jobs be impacted?
- Thank them, leave material behind, follow up. Thank them for their time and, if appropriate, offer assistance. Make sure to leave behind relevant material including the FA Fact Sheet. Send a follow up email that includes points covered during the meeting, next steps, and any additional information requested. Follow up periodically on the status of your issue. Each communication helps to develop a relationship with your representative's office.
Coalition Letters FARA joined
Research!America letter to Congress requesting timely passage of the FY23 appropriations, September 2022
Alliance for Connected Care letter urging the Senate to extend key Medicare COVID-19 pandemic telehealth flexibilities for two years, September 2022
March of Dimes letter to the Senate HELP Committee to pass S.350, the "Newborn Screening Saves Lives Reauthorization Act of 2021", September 2022
NORD letter encouraging quick and decisive action to pass updated User Fee legislation, August, 2022
Research!America's letter requesting robust funding allocation for the Labor-HHS-Education Subcommittee, May, 2022
EveryLife Foundation letter supporting Iowa RUSP Alignment Legislation, Feb 2022
NAF and FARA welcomes the 117th Congress, Feb 2022
AAAS letter requesting Congressional leaders to finalize FY22 appropriations, Jan 2022
EveryLife Foundation letter supporting Mississippi RUSP Alignment Legislation, Jan 2022
EveryLife Foundation letter supporting Maryland RUSP Alignment Legislation, Jan 2022
PPMD letter to Congressional leadership supporting the BENEFIT Act (S.373/H.R.4472), Jan 2022
EveryLife Foundation letter in support of Dr Robert Califf as FDA Commissioner, Dec, 2021
Friends of Cancer Research letter in support of Dr Robert Califf as FDA Commissioner, Dec 2021
MDA letter calling for passage of the Disability Employment Incentive Act (S.630), Oct 2021
NORD's letter to Governors to continue telehealth access, Oct, 2021
Research!America's letter addressing how a Continuing Resolution (CR) hinders research, Oct 2021
NORD, MDA & FARA's letter to Senate Leadership in support of NCATS funding, Sep 2021
Genetic Alliance letter in support of Ending the Diagnostic Odyssey Act of 2021, Aug 2021
Neuromuscular Disease Community Calls for Passage of ACT for ALS (H.R. 3537), Jul 2021
ALD Alliance letter supporting funding for RUSP alignment, May 2021
EveryLife Foundation letter supporting North Carolina RUSP Alignment Legislation, May 2021
EveryLife Foundation letter supporting Ohio RUSP Alignment Legislation, Apr 2021
Research!America letter asking President Biden to support the RISE Act, Mar 2021
EveryLife Foundation letter supporting Georgia RUSP Alignment Legislation, Feb, 2021
NAF and FARA welcome the 117th Congress, Feb 2021
Letter to President Biden Commending the Appointment of Dr Janet Woodcock as Acting Commissioner of the FDA, Jan 2021
Neuromuscular Advocacy Collaborative Sign-on Letter: COVID-19 Vaccine Distribution to NMD Patients, Dec 2020
Research!America letter asking Congress to fund research, development, and vital public health agencies, Dec 2020
NORD letter on Telehealth Access During the COVID-19 Public Health Emergency, Nov 2020
Scientific Society letter Supporting World Health Organization, Jul 2020
Michael J Fox letter to Congress to provide bridge funding to stalled research, May 2020
EveryLife letter to CMS in Anticipation of Nation Reopening, May 2020
EveryLife letter to Department of HHS regarding access during COVID-19, Apr 2020
Research!America DeGette-Upton Dear Colleague letter in support of COVID-19 research related relief, Apr 2020
EveryLife letter highlighting unique access barriers created by COVID-19, Mar 2020
National Health Council letter for aid to patient organizations, Mar 2020
I am Essential Letter to Congress addressing medication access in Third COVID-19 package, Mar 2020
Global FA Patient Organizations
Friedreich's Ataxia Research Association