SPRING | 2022
Hello Advocates!

The FY 2023 federal budget cycle has begun. President Biden has released his budget recommendations to Congress which includes several health initiatives. The release of the President’s budget kicks off the federal budget cycle. A few priorities in the President’s budget and of our advocacy partners includes funding support for the FDA, NIH, ARPHA-H, and Cures 2.0. NAF supports the requests for funding these and other ataxia related initiatives. Now congress is holding committee hearings about the bills and funding requests to include in the FY 23 budget. In this newsletter you will find a link to one of those congressional hearings that was recently held, along with information about NAF’s and FARA’s joint CDMRP funding request.

Be sure to check out the Calls for Action section in this newsletter where you will find several opportunities to support relevant legislation. Also, mark your calendar for the events that you will find in the Advocacy Events section.

Lori Shogren
Community Program and Services Director
Advocacy News
New Funding Opportunities for Friedreich's Ataxia Research from the DoD
The Department of Defense’s (DoD) Congressionally Directed Medical Research Programs (CDMRP) is a global funding organization that fosters novel approaches to congressionally targeted biomedical research areas. CDMRP receives annual appropriations that are disease or condition-specific and for the FY22 budget has added Friedreich's Ataxia to the list of topic areas eligible for $370M in funding within the Peer-Reviewed Medical Research Programs (PRMRP). This new funding opportunity is the result of the joint advocacy efforts of the NAF and FARA members.

This year our goal is to secure continued funding and expand the language to include all hereditary ataxias. Stay tuned for more information on how to support this initiative!
House Energy & Commerce Committee Holds Health Hearing
On March 17th, the House Energy & Commerce Subcommittee conducted a hearing on The Future of Medicine: Legislation to Encourage Innovation and Improve Oversight. A number of bills relevant to the Ataxia community were discussed, including the Cures 2.0, the HEART Act, the BENEFIT Act, and ARPA-H .You can view a recording of the hearing below.
NEW Advanced Research Projects Agency for Health (ARPA-H)
This new agency would strive to apply a high-risk/high-reward research model to biomedical research that would authorize an important conduit for bold new health innovations. There is an abundance of support for this new agency in Congress. $1 Billion was allocated in the FY22 budget to establish this new agency as its own office within the U.S. Dept. of Health and Human Services.

Fiscal Year 2022 Budget Highlights
In March Congress finally passed the FY 22 Omnibus Appropriations bill. Here are a few highlights in the FY 22 federal budget reported in recent FARA, Alliance for a Stronger FDA, RDLA, NORD and MDA newsletters.

  • Friedreich’s Ataxia added as an eligible condition under the Peer Review Medical Research Program (PRMRP), a category of the CDMRP, which will allow FA researchers access to a new funding opportunity. The PRMRP has $370 million available to support accepted research.

  • $5 million in funding for the ACT for ALS research grants program, which gives the FDA funding to infuse into rare neurodegenerative disease clinical research. 

  • $2.25 billion increase in medical research funding at the NIH.

  • $27 million increase in funding for important programs at the National Center for Advancing Translational Science (NCATS).

  • $102 million increase for programs at the FDA. 

  • $1 billion start for ARPA-H.  

  • Inclusion of several major telehealth provisions, which will continue several COVID-era telehealth policies for about five months after the public health emergency (PHE) ends. These provisions provide an opportunity for the telehealth community to continue advocating for permanent telehealth reform.
Advocacy featured in Brain & Life Magazine
We all can support a cause we believe in—and action can take many forms. Anyone Can Become a Patient Advocate. Read the articles below from the latest issue of Brain & Life and become an Ataxia Advocate!

Rare Disease Contest Winner
Congratulations to Shundra Wooten from Decatur, GA who was one of three winners of the EveryLife Foundation Rare Disease Story Contest! A $1,500 donation was made by the EveryLife Foundation in Shunda Wooten’s name to the National Ataxia Foundation.
You can read Shunda’s story and learn more about the Everylife Foundation “What’s Your Story?”
Rare Disease Week on Capitol Hill Scavenger Hunt Winners
18 NAF members joined over 950 other Rare Disease Advocates during the Rare Disease Week Lobby Days. 370 meetings with Congress members were held over the 2 lobby days.

Support Group Leaders, Greg Rooks and Lisa Cole were among the top-50 point earners of the Rare Disease Week Scavenger Hunt. Because of this, they were selected to receive a $1,000 donation to NAF from the EveryLife Foundation in their names.
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.

  • The Helping Experts Accelerate Rare Treatments (HEART) Act’s H.R. 6888 campaign page helps you share your story with your representatives and shows them that you support this bill. You can help by visiting the "Take Action" campaign pages HERE.

  • 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.

  • Haystack Project encourages you to share your Telehealth Tale! Complete their SURVEY HERE to represent your rare and ultra-rare communities telehealth needs and experiences.

  • 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.

  • Rare Disease Congressional Caucus. Click HERE to invite your legislators to join the bipartisan Caucus

  • 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).
New Letters of Support
NAF has signed on to the following letters:

  • IA, MD, MS RUSP Alignment Letters: This legislation implements a timeline in which newborn screening must begin for new conditions added to the federal Recommended Uniform Screening Panel (RUSP). On March 16th, the Mississippi Governor signed HB 927 into law. In Iowa, SF 2345was signed into law by the Iowa Governor on April 21st. In Maryland, HB 109 and SB 242 has passed out of the Maryland General Assembly. Read the letter HERE.

  • Conrad State 30 and Physician Access Reauthorization Act Letter: The bill would extend and expand the Conrad State 30 program, which allows qualified foreign-trained physicians to provide care in underserved communities. Read the letter HERE.

  • NBPP HHS Comment Letter: This sign-on letter urges Health and Human Services (HHS) to require insurers and pharmacy benefit managers to count patient copay assistances toward the beneficiary’s annual deductible of out-of-pocket costs in the proposed 2023 Notice of Benefit and Payment Parameters (NBPP) rule. Read the letter HERE.

  • NBSSL FY22 Omnibus Letter: Letter to congressional leadership to include Newborn Screening Saves Lives Reauthorization Act with a FY 22 omnibus appropriations measure. Unfortunately, NBSSL Reauthorization was not included in the FY 22 omnibus package. Read the letter HERE.

  • Step Therapy Letters: This legislation is important in ensuring patients can access the therapies their health care provider prescribes, in a timely and appropriate manner by providing clear exemptions and approval timelines when step therapy is required. Read the letters: TN, CA, Safe Step Coalition.

  • Non-Medical Switching Letter: This legislation would protect patients against non-medical switching practices. Read the letter HERE.

  • Copay Accumulator Letters: These bills will protect patients by ensuring that they can continue to access the medication they need without facing surprise out-of-pocket costs. Read the letters: NE, WI, DC, PA.

  • BENEFIT Act Support Letter: This legislation would elevate the patient voice in the FDA drug development process. Read the letter HERE.

  • NIH Funding Recommendation Letter: This letter is to support the recommendation of at least $49 billion for the NIH’s base. Read the letter HERE
Advocacy Events
Save the Date for Ataxia Hill Day - September 21, 2022
United Against Ataxia Hill Day

NAF & FARA will be jointly holding the fourth annual United Against Ataxia Hill Day will be held virtually on Wednesday, September 21, 2022. Because of our advocacy efforts last year, we were able to get Friedreich’s Ataxia added to Congressionally Directed Medical Research Program! We will continue to advocate on this year’s Hill Day to get all hereditary ataxias added to the program. Registration this year will be a two-step process. Look for announcements on the first step which is registering for the required training.
Rare Across America - August 8 -19, 2022

Make an impact in your district. Share your rare disease story. Meet other rare disease advocates. Registration is now open.

RARE Patient Advocacy Summit - September 12 - 15, 2022

Coming Soon: 2022 registration information, agenda, speaker details, and more from Global Genes.

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 naf@ataxia.org.
State Level Advocacy Opportunities
  • The RareAction Network® (RAN) is the nation’s leading advocacy network working to improve the lives of the 30 million Americans living with a rare disease at the state level. RAN serves as a broad spectrum of stakeholders ranging from patients, to their families, caregivers, and friends; from researchers to industry; to physicians and academia. While working predominantly at the state level, the network will filter information up to NORD’s national federal policy team to help address issues of national concern. Join the RareAction Network - https://rarediseases.org/advocate/take-action-locally/join-rare-action-network/

  • A Rare Disease Advisory Council (RDAC) provides an important platform that elevates the voice of the rare community in state government, and NORD is working to get an RDAC established in all 50 states! Right now, NORD is leading active RDAC coalitions in Connecticut, California, Colorado, Georgia, and Maryland. Do you want to learn more or get involved? Contact Annissa Read with NORD’s Policy team at areed@rarediseases.org.
More Legislative issues can be found on the Advocacy webpage
Find Your Local Representatives:
763-553-0020 | naf@ataxia.org