SUMMER | 2022
Hello Advocates!

Registration for this year’s United Against Ataxia Hill Day is open! Your voices have raised awareness among lawmakers and unlocked new funding opportunities for researchers. Sharing your journey with Ataxia is the most impactful way to build relationships with your representatives and influence legislation. Join us to keep this momentum going to increase research funding and expedite treatments.

The fourth United Against Ataxia Hill Day is on September 21, 2022. All meetings with Congressional Members will be virtual and run approximately 15-30 minutes in duration. You will be assigned 2-5 meetings between 9 am and 5 pm.

Registration requires two steps.

  • Sign up for Hill Day HERE! - Registration will close on August 31, 2022

  • Sign up for a one hour required training on August 10 at 7 pm ET HERE!

Important! Your Congressional meetings will not be scheduled until you complete the training.
If you cannot attend the training on August 10, or have any other questions, please contact me at
Lori Shogren
Community Program and Services Director
Advocacy News
16 year-old with FA wins NIH's Brain Initiative Challenge
Jake Juip, a 16-year-old who has Friedreich's Ataxia (FA), was a Tier 1 Winner of NIH's Brain Initiative Challenge! His video shows his journey of living with FA. He advocates for FA research and brings the patient experience to clinical research. Congrats Jake! 
Reasons to Invest in NIH
The United for Medical Research released the latest in their "Why Invest in NIH?" series. "Enabling Patient Stories" is a fact sheet that profiles five individuals who were a part of biomedical breakthroughs. The breakthroughs, which have transformed lives, were made possible by NIH-funded research.

Read and share "Why Invest in NIH?" series to remind policymakers and the public why funding NIH is essential to helping sustain and advance lifesaving biomedical research and innovation. Tag your social media posts with #keepNIHstrong.
We Can All Play an Important Role in Rare Disease Advocacy
Samuel Kirton, NAF President, talks about the importance of advocacy work in rare disease communities and opportunities to get involved. Kirton says, “Advocacy efforts do make a difference. For instance, the National Ataxia Foundation and Friedreich’s Ataxia Research Alliance worked together through two federal budget cycles to add Friedreich’s ataxia to the Peer Reviewed Medical Research Program (PRMRP) for fiscal year 22.”

You can read his full article in the latest edition of Pulmonary Fibrosis News.
FY 23 House Appropriations Update

The House Appropriations Committee approved its Fiscal Year 2023 Labor, Health and Human Services, Education, and Related Agencies Appropriations bill. The House committee would provide a total of $47.5 billion for the National Institutes of Health (NIH), an increase of $2.5 billion over the FY 2022 enacted level. The committee bill also proposes $620 million for the BRAIN Initiative, equal to the FY 2022 level of funding. Under the committee’s bill, the Advanced Research Projects Agency for Health (ARPA-H), funded separately from NIH, would receive $2.75 billion in FY23. More detail on these House appropriations bills and their associated reports is available here.

The House Adopts FY23 Appropriations Minibus Including Ag/FDA Funding. Under the bill, BA (budget authority) funding for FDA would reach $3.645 billion, an increase of $341 million (10%). More detail on these bills is available here.
House Passage of PDUFA Amendments (H.R. 7667)
The House passed H.R. 7667 in a 392-28 vote. H.R. 7667 is a bipartisan bill that is an important step forward in reauthorizing the FDA’s medical product user fee programs (PDUFA) and helps strengthen the accelerated approval pathway that protects the intent of the Orphan Drug Act. This passage of amendments is vital for the passage of PDUFA to happen by its expiration. It is now up to the Senate to pass the proposed amendments for PDUFA in H.R. 7667.
FDA Launches Accelerating Rare Disease Cures (ARC) Program to Speed Up Rare Disease Drug Development
The FDA's Center for Drug Evaluation and Research rolled out a new program to expedite and increase the development of new therapies for rare diseases. The Accelerating Rare Disease Cures (ARC) Program will leverage regulatory and scientific engagement to promote the development and approval of these treatments. For further detail, read a recent review here by Rachel Sher, formerly NORD’s VP for Public Policy and Regulatory Affairs. is Seeking Patient and Advocate Feedback
The National Library of Medicine is recruiting participants for feedback as part of the modernization effort.
If you are a patient, patient advocate, family member, or health care provider, the modernization team wants to speak with you! Participants will be asked to perform tasks on the Beta website, complete a survey or card sorting activity, or to participate in a virtual interview to help improve the experience for users. The duration may range from 15 to 60 minutes.
Please email to learn more.
FDA is Searching for Patient Representatives to Serve on Advisory Committees
The US Food and Drug Administration (FDA) is requesting your recommendations for patient representatives to serve on its advisory committees or panels. The FDA relies on its many advisory committees to help them make sound decisions based on the best science available.
Patient organizations interested in participating in the selection of an appropriate voting or nonvoting member to represent patient interests on an FDA advisory committee or panel may send a letter or email to stating that interest by Monday, August 15, 2022.

Patient representatives may be self-nominated or may be nominated by a patient organization. Patient representative nominations should be submitted electronically through the FDA Advisory Committee Membership Nomination Portal by August 15, 2022.
Calls for Action
  • Contact your Representatives in support of the newly introduced Cures 2.0 bill, H.R 6000. This bill is designed to revolutionize how the U.S provides care to patients, including provisions aimed at speeding up the delivery of groundbreaking cures, treatments, and innovations for rare disease. Click HERE to send a letter to your Members now.

  • Contact your Members of Congress in support of the BENEFIT Act, H.R 4502/S. 373. A bill to strengthen the use of patient-experience data within the benefit-risk framework for approval of new drugs. Click HERE to send a letter to your Representative and Senators now.

  • Support the Access to Rare Indications Act. The lead co-sponsors of the Access to Rare Indications Act have circulated this LETTER to their fellow Representatives asking for support of H.R. 6160. Please share this letter to help encourage your elected official to co-sponsor the bill. This is legislation to support patients with rare and ultra-rare diseases in getting evidence-based, medically necessary care covered by their health insurance. Take action HERE.

  • The ABLE Age Adjustment Act S.331 will expand access to ABLE accounts by increasing the age by which an individual must have established a qualifying disability from 26 to 46. Now the bill moves to the Senate floor. Urge your Senators to support this crucial bill today!

  • Newborn Screening Saves Lives Reauthorization Act, S. 350/H.R 482: The Newborn Screening Saves Lives Reauthorization Act, will continue critical federal programs that provide assistance to 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. You can find more information on the bill HERE and invite your Member of Congress to cosponsor the bill HERE.

  • Join us in advocating for increased federal funding to support brain health. Click HERE to ask your Legislator to support the BRAIN Initiative.

  • Join us in supporting access to home-based care. Click HERE to learn more about Direct Care Workforce and Family Caregivers Act (S.2344).

  • Rare Disease Congressional Caucus. Click HERE to invite your legislators to join the bipartisan Caucus
New Letters of Support
NAF has signed on to the following letters:

  • Comment Letter regarding Pharmacy Benefit Managers’ Impact on Affordable and Accessible Care for Movement Disorders Patients.

  • Support Letter to incorporate S. 4185, the Retaining Access and Restoring Exclusivity (RARE) Act, into the FDA Safety and Landmark Advancements Act (FDASLA). Good news! Both the Senate’s FDASLA (S. 4348), passed out of the HELP Committee last month, and the House’s Food and Drug Amendments of 2022 (H.R. 7667), which passed out of the House with a bipartisan vote of 392-28, include provisions to maintain the original intent of the Orphan Drug Act, clarifying that orphan drug exclusivity is tied to the approved indication, while ensuring proper incentives remain in place to foster robust rare disease drug development. 

  • Comment Letter to include proposals to strengthen the patient voice in PDUFA VII.
Advocacy Events
Rally for Medical Research on Capitol Hill - September 13-14, 2022

Attendees will meet with their Members of Congress in-person in Washington, DC on September 14th. A reception to celebrate medical research will be held on September 13th for all partners and participants.

Global Genes RARE Patient Advocacy Summit - September 12 - 15, 2022

Registration is open. Summit sessions provide attendees with insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies to take home. This year, the Summit will be a hybrid event so all can attend – in person at the Town & Country Hotel in San Diego, California and live streamed online in their virtual event platform.

International Ataxia Awareness Day (IAAD) - September 25, 2022

International Ataxia Awareness Day (IAAD) is on September 25th of each year. It is a coordinated effort from individuals and Ataxia organizations around the world to help shed light on this rare disease. We encourage individuals and disease organizations to join us by planning events and/or awareness campaigns for IAAD.

Advocacy Training Resources
These resources by the Rare Disease Legislative Advocates (RDLA) are designed to help rare disease patients, parents, caregivers and others to make their voices heard. If you would like more information about a topic or have tools to share, please contact us at
State Level Advocacy Opportunities
You can obtain a Proclamation or Resolution to recognize September 25 as International Ataxia Awareness Day (IAAD) in you state or community. Check out this GUIDE for information on how to obtain a Proclamation or Resolution and samples.
Young Adult Advocacy Opportunities
YARR Leadership Academy Applications Now Open: Applications for the 2022 YARR Leadership Academy are open between Tuesday, July 12th and Friday, August 5th. Now in its second year, the YARR Leadership Academy is open to young adults between 18 and 29 years old who are deeply committed to the rare disease community and ready for a leadership role. Through an eight-week series of online classes with experts and peers, students will learn about opportunities for patient representation in policy making, drug development, and the regulatory process and prepare for the steps it takes to enter those roles. The YARR Leadership Academy application will be available here.
More Legislative issues can be found on the Advocacy webpage
Find Your Local Representatives:
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