The FA community learned this month that the FDA pivoted and asked Reata to request a Pre-NDA meeting for Omaveloxolone. This action was a direct result of your advocacy! Sharing your story was influential in educating and engaging those who received FARA’s petition. Your voice was heard! There is now another opportunity for action.

On May 25, 2021, US Representatives Jeff Fortenberry (NE-01) and Mike Quigley (IL-05), with Senators Chris Coons (D-DE) and Lisa Murkowski (RAK) re-introduced the bipartisan, bicameral Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813). This legislation has 99 original co-sponsors in the House.

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.

Let’s help get this bill passed! Reach out to your representatives and ask them to support Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813). Find your US Senators here and your US Representative here. You can call their office or send them an email. Explain why these important initiatives would expedite the search for a cure for FA.

Sample language you can use:

As your constituent and an advocate, I urge you to cosponsor the ACT for ALS (H.R. 3537 / S. 1813). Those with rare neurodegenerative diseases have waited too long for promising therapies and drugs and the ACT for ALS is an important step in bringing promising new treatments to the market as quickly as possible.

<Insert personal story, explain FA>

Although this bill is directed primarily towards ALS, there are key provisions that would help all individuals with rare neurodegenerative diseases like FA. First, it would speed up development of potential lifesaving therapies by creating a brand-new grants program that invests in promising neurodegenerative disease research and coordinates private and public research and development efforts. Second, this legislation would commission the publication of an FDA Action Plan on rare neurodegenerative diseases. Finally, the ACT for ALS will facilitate greater coordination of federal government efforts by creating an HHS Public-Private Partnership for Rare Neurodegenerative Diseases.

Please cosponsor the ACT for ALS today (H.R. 3537/ S. 1813)!
Capitol Hill Updates 
President’s Budget for FY 2022: The Biden Administration released a short version of President Biden’s FY 2022 budget. The proposed budget is $118 billion, or 8.4% higher than the FY 2021 level. This 8.4% increase would account for increases among the Department of Education, Department of Commerce, Department of Health and Human Services, and the Environmental Protection Agency’s budgets. Once the Biden Administration’s full FY 2022 budget request is sent to Congress, which is expected on May 28, Congress will hold hearings and develop and consider a budget resolution. After the hearings are held, the Appropriations Committee drafts the 12 appropriations bills and Congress must pass all 12 to fund the government. To learn more about the appropriations process, check out RDLA’s tip sheet here or video tutorial here.
Congressionally Directed Medical Research Program (CDMRP): FARA and NAF have a particular interest in the defense appropriations bill this year. Together, we have submitted an appropriation request for “hereditary ataxia” to be added to the CDMRP, which is run by the Department of Defense. The CDMRP is focused on funding high impact, high risk and high gain research projects. Since its origin in 1992, the CDMRP has funded $17.8 billion dollars of research. If added, this would provide a new source of funding for FA researchers.
FARA is making a special outreach to community members who either served in the US military or have a family member (spouse, parent, child, sibling, grandparent) who served.

If you have a connection to the military, your participation in this quick survey would be very helpful.
Ask your Senators to support robust NIH funding

Increases to NIH funding return dividends in terms of both human benefits and economic impact. The American Society for Human Genetics released a report this week examining the impact of the genetics and genomics sector on the economy and on human lives. In terms of economic impact, every dollar of federal investment in genetics and genomics returned more than $4.75 (a 4-1 ROI).

Senators Bob Casey (D-PA) and Richard Burr (R-NC) are circulating a "Dear Colleague" asking their fellow Senators to sign onto a letter supporting robust funding for the NIH. Contact your Senator and ask them to join the fight for strong NIH funding.
Nomination to Watch: On a bipartisan vote, Dr. Eric Lander, President Biden’s nominee for the Office of Science and Technology (OSTP) Director, advanced from the Senate Commerce, Science, and Transportation Committee. His nomination will now move to a full Senate vote. If confirmed by the Senate, Dr Lander would be the first life scientist to hold this position, and the first director to be part of the President’s cabinet. Dr. Lander is a well-respected geneticist and the last of President Biden’s Cabinet nominees awaiting confirmation.
Rare Disease Week
RDLA, a program of the Everylife Foundation, has opened registration for Rare Disease Week. It will be virtual this year, so regardless of where you are, you can do it! Although the focus of the program is on US legislation, individuals outside of the US who are interested in participating can sign up for most of the events. The one exception - legislative meetings will only be available for those living in the United States.

This is a great opportunity to learn about pending US legislation impacting rare disease, gain some advocacy training and, if US based, speak directly to your Members of Congress &/or their staff. Plus, an extra bonus, Everylife just announced:
Advocacy + Fun = $100,000
During Rare Disease Week on Capitol Hill 2021, advocates can win a grant for their rare disease organization of choice! By participating in the events, attending virtual Hill meetings, and taking part in other activities, advocates can win points. The top 50 point-earners from the week will win a grant --from $1,000 to $5,000 each--totaling $100,000 awarded by the EveryLife Foundation.

Register today to be a part of this incredible week of learning, networking, and advocating AND for an opportunity to win up to $5000 for your favorite rare disease organization!

The schedule of events and registration information is here.
Advocacy Training Webinars 
So Your State Just Enacted an RDAC - Now What?

Rare Disease Advisory Councils (RDAC) help to address the needs of rare disease patients and families by educating and advising state government officials on ways to address the unique challenges faced by the community. Do you want to help your state implement a strong and effective RDAC? NORD is thrilled to launch our latest RDAC toolkit entitled, “Implementing a Rare Disease Advisory Council in Your State.” This toolkit will help states with newly signed RDAC legislation establish a high-functioning RDAC by sharing best practices from existing councils as part of this step-by-step guide to implementing an RDAC.

Date: June 10, 2021 Time: 1:00pm - 2:00pm ET
The Living Rare, Living Stronger NORD Patient and Family Forum featuring the Rare Impact Awards is officially virtual for 2021! Join us for networking, education, inspiration, hope and fun this June. Be sure to take advantage of the special rate for patients, caregivers and NORD member representatives. Information on registration and scholarships can be found here.
EveryLife Publishes COVID-19 Vaccine Survey

The EveryLife Foundation has posted the results of the Rare Disease COVID-19 Vaccine Survey conducted last fall. The data acquired helped guide the actions EveryLife and their advocacy partners, including FARA, took in response to the current public health emergency. The Rare Disease COVID-19 Vaccine Survey was designed to better understand the rare disease community’s perceptions and information needs about a vaccine for COVID-19. Access the survey summary here.
Newborn Screening State News

The Governor of Georgia signed HB 567 in law!
This legislation will eliminate the unnecessary delay in screening for diseases, ensuring the Georgia babies born with debilitating and life-threatening diseases are diagnosed and treated at the earliest age possible and without devastating delays. The legislation would help save the lives of more than 700 babies in Georgia annually. The bill passed the State House 166-2 and unanimously passed the State Senate.

Ohio HB 110 passed out of one chamber
This bill is currently waiting for passage out of the full Assembly. Ohio advocates can contact their state representatives here.

North Carolina NB 736 passed in NC House of Representatives
North Carolina advocates can ask their State Senator to support this bill here.