Join NAF and FARA in raising awareness about Ataxia and supporting legislation that directly impacts our communities.

The third United Against Ataxia Hill Day will be held on Wednesday, September 29, 2021. It will be a virtual event so every U.S. resident, regardless of where you are, can participate; there is no cost or need to travel. Instead, you can speak to your Congressional Members right from the comforts of your home!
What Happens at Hill Day?
A group of advocates will meet with Congressional Members to discuss issues important to the Ataxia community. This year’s topics include:

1. National Ataxia Awareness Day Resolution
Senators Christopher Murphy (D-CT) and Cindy Hyde-Smith (R-MI) are co-sponsoring a Resolution declaring September 25, 2021, National Ataxia Awareness Day. (S.Res. 339)

2. 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.

The request was submitted to the Defense Appropriation Sub-Committee for consideration for FY22 but it did not make it out of Committee. We are starting early in building a coalition of support for FY23 and already have several Members’ support.

3. 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. Although this bill is directed primarily towards ALS, there are very important parts that help all rare neuromuscular diseases like FA. The three initiatives in this legislation that would help FA research are:

· Establish 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;
· Commission the publication of an FDA Action Plan on rare neurodegenerative diseases;
· Implement an FDA grant program to fund research and therapy development for life-threatening or severely debilitating rare neurodegenerative diseases. You can check if your Member is one of the 315 who have signed on in the House or 15 that have signed on in the Senate. If they did, you will be able to thank them! If not, you can ask them to support ACT for ALS (H.R. 3537/S. 1813).

How can I prepare for my meetings?

NAF and FARA will be running a Hill Day Advocacy training webinar on Wednesday, September 15 at 7 pm. Access information will be sent to all registrants. The training will also be recorded and available for those who cannot participate that evening.
Registration for this event will close on September 20, 2021. Don't miss the chance to join us, register today! Your voice is needed to pass these important initiatives!
United Against Ataxia Celebration
After your legislative meetings, please join NAF and FARA for a short virtual Hill Day celebration. Tune in at 7 pm to see Senators Chris Murphy (CT) and Cindy Hyde-Smith (MS) be recognized as our first Ataxia Research Champions! Stay tuned for access information coming to your email soon.
Rare Disease Week Review
Rare Disease Week was a success!
600 RARE DISEASE ADVOCATES
367 MEETINGS WITH CONGRESS
250 PATIENT ORGANIZATIONS
The 10th Rare Disease week continued to be an empowering and inspiring week of action. For those that missed it, a list of events and videos are available here.
 
FARA Ambassadors Shandra Trantham and Mary Nadon Scott were not only the voice(s) of the FA community during Rare Disease week, but through their incredibly dedicated commitment to the week’s programs, they each earned a grant for FARA! Thank you Shandra and Mary!
 
Please mark your calendar for in-person Rare Disease Week on Capitol Hill 2022 taking place February 28th through March 2nd.
A YARR Leadership Academy Graduate!

FARA Ambassador Shandra Trantham graduated from the Everylife Foundation YARR (Young Adult Representatives of the Rare Disease Legislative Advocates) Leadership Academy during Rare Disease Week. The YARR Leadership Academy is a series of six on-line courses on the roles and opportunities for patient representation in policy making, drug development and the regulatory process. Each course is three hours so Shanda received 18 hours of advocacy training, culminating in a Capstone Project! Out of 45 applications received, Shandra was one of thirteen selected to participate in this program. Congratulations Shandra!
The Rare Artist Program was established in 2010 to exhibit the unique gifts of individuals affected by rare disease and to promote the expression of their stories through art. Enjoy your time exploring the Virtual Rare Artist Gallery. Explore artwork, artist statements, and videos that tell the story and share the voices of the 2020 Rare Artist Award Recipients, including FA community member Sophia Sieber-Davis!
Capitol Hill Updates 
BENEFIT Act Introduced in the House

The BENEFIT Act, H.R. 4472 was introduced in the House on July 16, 2021 by Representatives Doris Matsui (CA-6) and Brad Wenstrup (OH-2). Earlier this year, Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) introduced the identical Senate version, S.373. 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.
To learn more, click here
Newborn Screening Saves Lives Reauthorization Act Passes the House

The Newborn Screening Saves Lives Act, H.R. 482, passed the House with a 325-103 vote. The bill now moves to the Senate. The Everylife Foundation has provided this action alert to encourage advocates to ask their Senators to support the legislation. 
Our partner at Alliance for a Stronger FDA shares some
insight on ARPA-H

Admiration for the accomplishments of the Defense Advance Research Project Agency (DARPA) is longstanding and widespread in Washington. More than a decade ago, Congress created I-ARPA (history) to promote innovations in national intelligence, and subsequently, ARPA-E (history) to promote innovation in energy solutions.

Earlier this year, President Biden promoted the idea of an ARPA-C (White House announcement) for climate innovations, as well as ARPA-H for health innovation.

The initial proposal for an ARPA-H entity started a few years ago but has evolved in several ways. The concept of ARPA-H has also been buoyed by the success of the government’s efforts to rapidly stimulate private sector development of COVID-19 vaccines. This fits the DARPA model of taking on difficult problems, rapidly identifying possible solutions, then stimulating private development with the foreknowledge that there will be failures as well as successes. 

FARA will continue to monitor developments on ARPA-H.
Comments submitted on 21st Century Cures 2.0 Discussion Draft
NORD
NORD submitted comments to Reps. Diana DeGette (D-CO) and Fred Upton (R- MI) on their recently released Cures 2.0 discussion draft. NORD was largely supportive of the draft bill, particularly the provisions that focus on patient engagement in drug development and review. NORD expressed concern over some provisions, including the inclusion of language to create an Intercenter Institute for Rare Diseases within the FDA because of its potential to detract from FDA’s rare disease efforts. Read more here
Everylife Foundation
On July 16th, the EveryLife Foundation submitted written comments in support of the 21st Century Cures 2.0 Discussion Draft, particularly the inclusion of an additional Intercenter Institute for rare disease at FDA, the attention to new solutions for Medicaid coverage of DNA sequencing, and increased use of patient experience throughout the regulatory landscape. You can read more here.
Advocacy Training Webinars 
Tickets are now available!
Rare New England's 2021 Annual Conference will be held virtually on
October 23, 2021. It will be a day of education and information to Rare Disease patients, caregivers, students, and professionals.
Caregiver and Patient tickets are FREE this year.

View full agenda and register here.
Announcements 

On Tuesday, September 14, 10 a.m. – 3:45 p.m. ET, the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), an FDA-funded initiative, will have its public premiere during the annual meeting for the project. The platform will host, standardize and share rare diseases data as part of its functionality.

In addition to the demonstration of the platform itself, presentations throughout the day will show how RDCA-DAP is breaking down data silos, provide insight into the platform’s development and testing process, and showcase the importance of RDCA-DAP from the perspective of critical stakeholders, including academic, clinical, regulatory and patient communities. 
Newborn Screening State News

Rhode Island
SB 234 was signed into law on July 12, stating that Rhode Island must screen for all Recommended Uniform Screening Panel (RUSP) conditions. It also includes a two-year timeline, however it provides the Director of Health the ability to delay the timeline if they deem the two years impracticable. Please read the bill language here