Winter | 2021
Big ACT for ALS News!
Your efforts paid off!
The ACT for ALS passed the US Senate! This initiative was one that Ataxia advocates from NAF and FARA talked to lawmakers about during 2021 Ataxia Hill Day. This bill would accelerate therapeutic development for all neuromuscular diseases. Thank you to all the advocates who made this happen!

Policy Updates
Department of Defense Funding for Brain Research is at Risk!
The U.S. Department of Defense (DoD) Congressionally Directed Medical Research Program (CDMRP), which has funded over $15 billion in research into many diseases and conditions, including several brain and central nervous system (CNS) diseases. Funding for these programs is in serious jeopardy if the government is operating under a year-long continuing resolution for FY22.

Continued funding under a continuing resolution (CR) is not particularly good for FDA either. Under the CR, FDA has only the amount to spend that it did in FY 21 and cannot undertake certain new initiatives. There is a substantial gap between the agency’s needs and its available funds under a CR.

Please urge Congress and the Administration to complete Fiscal Year 2022 appropriations before the February 18, 2022, deadline established under the current Continuing Resolution.

Contact Your Local Representative
Defend the Orphan Drug Tax Credit (ODTC)
NORD, along with nearly ninety other organizations representing patients with rare diseases, are urging the Senate not to include harmful changes to the ODTC found in the final version of the House-passed Build Back Better Act. The ODTC is an important incentive that encourages clinical trials to be conducted for rare disease treatments. Curtailing this incentive could reduce the number of FDA-approved drugs for rare diseases.

Your legislators need to hear from you! NORD urges all patients and advocates to take action NOW.
New Legislation
BENEFIT Act Needs Your Support
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.
Cures 2.0
Representatives Degette (D-CO) and Upton (R-MI) introduced the Cures 2.0 bill, H.R 6000, on November 16th, 2021. 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 diseases. Cures 2.0 builds on the successes of the 21st Century Cures Act, passed in 2016, to advance biomedical research, regulatory science, public health, and payment policy innovation so critical for rare disease patients and families. You can read more about the bill here
Access to Rare Indications Act
Congresswoman Doris Matsui (D-CA) and Reps. Mike Thompson (D-CA), Mike Kelly (R-PA), and Markwayne Mullin (R-OK) introduced the Access to Rare Indications Act of 2021, legislation to support patients with rare and ultra-rare diseases in getting evidence-based, medically necessary care covered by their health insurance. Here is Rep. Matsui’s press release about the bill:

A bill introduction is a huge accomplishment, but we need your help to get us from a bill to a law!!

Please use your social media - Facebook, twitter, LinkedIn - to Call for Cosponsors and thank the leaders of the bill. We made it easy for you – here are some materials to make it simple! The attachment has sample tweets and images.
New Letters of Support
NAF has signed on in support of the following legislative issues:

  • Congressional Caucus for Social Determinants of Health (Read more)
  • Disability Employment Incentive Act (S.630)
  • Ending the Diagnostic Odyssey Bill (S.2022)
Advocacy News
New Bespoke Gene Therapy Consortium (BGTC) has Launched
The newly launched Bespoke Gene Therapy Consortium (BGTC) aims to speed the development of gene therapies to treat Rare Diseases. The program was created by the National Institutes of Health (NIH), U.S. Food and Drug Administration and 15 private organizations and will be co-led by NCATS and managed by the Foundation for the National Institutes of Health. Learn about NCATS’ role, the partners involved and what the program plans to accomplish.

Advocacy Events
Virtual Rare Disease Week on Capitol Hill
June 22 - March 2, 2022
Join other advocates virtually to give rare disease patients a voice on Capitol Hill. Advocates will once again have the opportunity to participate in the Points for Advocacy Scavenger Hunt and the EveryLife Foundation will award a total of $100,000 to the top-50 point earners' rare disease non-profit organization of choice! This is valuable training for advocates interested in participating in our next United Against Ataxia Hill Day being held in September 2022.

Rare Disease Week Training Webinars 
Click here to register
  • General Training Webinar: February 2nd at 2:00pm ET
  • Team Coordinator Webinar: February 9th at 2:00pm ET (There is no registration for this webinar. The link to attend will be sent to all team coordinators once the coordinators have been selected.)
  • Share Your Story with Policymakers Webinar: February 10th at 2:00pm ET
Rare Disease Day - Show Your Stripes!
February 28, 2022
It’s a day to raise awareness of the over 7,000 rare diseases that impact over 300 million people globally! Find ways to get involved in events and activities taking place across the United States

Current Legislative Issues
Legislative Issues NAF is Following:
Legislative Initiatives NAF is Following:
Letters of Support NAF Has Joined:
More Issues can be found on the Advocacy webpage
Find Your Local Representatives:
763-553-0020 |