Happy New Year Advocates! 2021 ended with some amazing accomplishments thanks to your hard work. You may have been in legislative meetings, sent emails or simply shared your story with someone who did not know about ataxia. Every action, whether big or small, helps to raise awareness of FA and the resources needed to expedite treatments! Here are just a few highlights of what our community successfully undertook together: |
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74,000 people signed a petition asking the FDA and Reata Pharmaceutics to move forward on a New Drug Application (NDA) for Omaveloxolone - that NDA submission is expected to be filed in the first quarter of 2022. |
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FARA joined with many organizations to support the Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813). This bill had several provisions that would help expedite ataxia research. The ACT for ALS was passed by the House and Senate in December and signed into law by President Biden on December 23, 2021 (Public Law No: 118-79).
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For the second year, a Senate Resolution was passed recognizing September 25, 2021 as National Ataxia Awareness Day. |
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FARA advocates attended many legislative meetings throughout the year, including Rare Across America in February, Rare Disease Week in August, and United Against Ataxia Hill Day in September to name a few. This strong community participation made it possible to educate legislators about ataxia and successfully advocate for our initiatives. |
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Leaping into 2022, there are also promising initiatives on the horizon. FARA will continue to share information as they develop. It will take all of us to stay updated and engaged on the issues so that our collective voice will be heard. 2021 was great, let's make 2022 even better! Here are a few topics currently under consideration: |
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Our primary “ask” for the 2021 Hill Day was to add "Hereditary Ataxia" to the Congressionally Directed Medical Research Program (CDMRP), which would provide additional research funding. FARA is continuing the work on this issue with various legislative offices and will have material ready for you to meet with your Members during Rare Disease week (details below!) |
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PDUFA VII: Fiscal Years 2023-2027
Before PDUFA's enactment in 1992, Americans’ access to innovative, new medicines lagged behind other countries. FDA's premarket review process was understaffed, unpredictable, and slow. The Agency lacked sufficient staff to perform timely reviews or develop procedures and standards to assure a more rigorous, consistent, and predictable process. To tackle these challenges, Congress passed PDUFA, which authorized FDA to collect industry user fees to hire additional staff and upgrade its information technology systems. In return, it committed the Agency to speed the application review process for new drugs without compromising its high standards for new drug safety, efficacy, and quality. PDUFA must be renewed every five years.
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Reps. Diana DeGette, D-Colo., and Fred Upton, R-Mich., unveiled a draft of follow-up legislation to the 21st Century Cures Act that would authorize the Advanced Research Projects Agency for Health, change how Medicare pays for new treatments and technologies and broadens access to telehealth.
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The Orphan Drug Tax Credit is still in danger!
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Right now, the US Senate is debating the Build Back Better Act which currently contains a provision to change the Orphan Drug Tax Credit that could harm rare disease patients and their ability to have access to new therapies and cures. The Orphan Drug Tax Credit provides incentives for research and development of new treatments for rare disease patients by allowing drug manufacturers to claim a 25% tax credit for clinical testing expenses associated with rare disease drug development. The current version would change the Orphan Drug Tax Credit to only allow drug manufacturers to claim this tax credit for a drug’s first rare disease indication and not any subsequent rare indications. Given that 90% of rare diseases lack an FDA approved treatment, these changes could have a devastating impact on the millions of rare disease patients who continue to hope for a safe and effective therapeutic option to treat their specific condition.
Tell Congress to Protect the Orphan Drug Tax Credit Today!
Contact your Members through NORD’s Action Alert here.
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Congress passes another Continuing Resolution (CR)
The current CR extended FY21 funding levels until February 18, 2022. Our advocacy partner Research!America is keeping track of the exactly how much time has passed without Congress finalizing FY22 appropriations. When Congress delays in passing a timely budget, they slow desperately needed research. CRs hamstring the full array of R&D-critical federal agencies, stall momentum for new and innovative programs and breed uncertainty in recruiting and retaining the best researchers and doctors.
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National Defense Authorization Act (NDAA) passes
Another program impacted by a CR is the CDMRP, a research program within the Department of Defense. The House passed the 2022 National Defense Authorization Act (NDAA) by an overwhelmingly bipartisan 363-70 vote in early December, and the Senate later passed the bill by a bipartisan 88-11 vote. President Biden signed the bill on December 27, 2021.
However, the NDAA is a policy bill and doesn’t authorize any actual spending. Therefore, Congress needs to pass an appropriations bill to fund the Department of Defense for FY22.
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Update on the nomination of Dr. Robert Califf as FDA Commissioner |
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During the hearing, Dr Califf said his top three priorities once confirmed include helping with pandemic emergency preparedness and response, improving consumer and patient protection and keeping up with innovation and modernization. Dr Califf, a cardiologist, was briefly the agency’s commissioner under the Obama administration beginning in 2016 and was nominated by President Biden on Nov 12, 2021.
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The National Organization for Rare Disorders (NORD) is proud to be the official US partner for Rare Disease Day. NORD works with EURORDIS (Rare Disease – Europe) and others all around the world to drive an international campaign that shines a light on rare diseases and advocates for health equity for all those living with a rare disease. Visit the International Rare Disease Day site for additional information and resources to get involved in events and activities taking place worldwide.
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Registration is open for Rare Disease Day at The National Institutes of Health 2022. Register here for this virtual event, which will take place on Monday, Feb. 28, 2022.
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Join hundreds of advocates virtually for Rare Disease Week on Capitol Hill from February 22nd to March 2nd. This is a wonderful opportunity to learn about the issues and meet with your Congressional Members. Connecting with your Members on a regular basis helps to build a relationship and develop a deeper understanding of FA. While in your meetings, you can advocate for legislation that helps everyone with rare disease, like increased funding for the FDA, or for more specific issues, like adding “hereditary ataxia” to the CDMRP, or both! View the agenda and register here.
No prior advocacy experience is needed! Simply register for the training webinars.
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General Training Webinar: February 2nd at 2:00pm ET
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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.)
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Share Your Story with Policymakers Webinar: February 10th at 2:00pm ET
- Share Your Story is a new webinar for 2022! This webinar will teach you how to best share your rare disease story with your policymakers and provide coaching on how to tell your story.
EXTRA BONUS TO PARTICIPATE!
Advocates will once again have the opportunity to participate in the Points for Advocacy Scavenger Hunt and the EveryLife Foundation will award grants totaling $100,000 to the top-50 point earners' rare disease non-profit organization of choice! FARA had three grant recipients in 2021 – you could win one in 2022!
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Meet rare disease advocates from your state and region, get to know our YARR members, and win prizes!
Register for this fun opportunity for the rare disease community in each region and state to get together before Rare Disease Week to foster community and camaraderie. Whether you are new or a veteran of the rare disease community, there will be something for everyone. We want to take time to get together socially before the excitement of Rare Disease Week! See below for details on the virtual session in your region.
Virtual Regional Meet and Greet Sessions:
January 11 at 5-6 pm PST: AK, AZ, CA, CO, HI, ID, MT, NV, NM, OR, UT, WA, WY, U.S. Territories
January 11 at 6-7 pm CST: AR, IL, IN, IA, KS, MI, MN, MO, NE, ND, OK, SD, WI
January 13 at 6-7 pm CST: AL, FL, GA, KY, LA, MS, OH, SC, TN, TX, WV
January 13 at 6-7 pm EST: CT, DC, DE, ME, MD, MA, NH, NJ, NY, NC, PA, RI, VT, VA
No prior experience necessary. Registration for this event and all RDLA events are free for rare disease advocates.
RAFFLE DETAILS: To be entered to win the raffle, registration AND attendance to one Regional Meet and Greet 2022 session is required. Only one winner per session. No repeat winners for attending multiple sessions.
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The fourth annual United Against Ataxia Hill Day will be held virtually on Wednesday, September 21, 2022.
Just a reminder, by participating in Rare Disease Week (detailed above), and then in United Against Ataxia Hill Day in September, you have two complimentary ways to raise awareness about FA and effect change throughout the year!
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THE UNITED NATIONS (UN) ADOPTS FIRST-EVER RESOLUTION TO ADDRESS CHALLENGES OF PEOPLE LIVING WITH RARE DISEASE
On Thursday, December 16, the UN adopted a groundbreaking resolution recognizing the specific challenges of Persons Living with a Rare Disease and their families. Read more here about this incredible grassroots global effort!
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Newborn Screening State News
North Carolina Passes RUSP Alignment Legislation
On November 19th, the Governor of North Carolina signed HB 736 into law. HB 736 will save the lives of nearly 700 babies born in the state each year by implementing a three-year timeline in which the screening must begin for new conditions added to the federal RUSP and requires that the North Carolina Department of Health and Human Services shall provide a report on the state of the addition at 18 months and every six months the addition is delayed past three years.
2022 Update on the EveryLife Foundation’s RUSP Alignment Efforts
After working alongside our community to successfully pass RUSP alignment legislation in Ohio, Georgia, Arizona, and North Carolina in 2021, the EveryLife Foundation is excited to announce that we will continue to pursue this important legislation. In 2022, we have committed to leading efforts to pass RUSP legislation in Iowa, Maryland, and Mississippi.
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Hope Through Trees
2021 Award Recipient
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Congratulations Kelly Hennessy! Kelly was one of ten people selected by the EveryLife Foundation as a 2021 Rare Artist Award Recipient.
The Rare Artist Program was established in 2010 to exhibit the unique gifts of individuals with, or affiliated with, a rare disease to tell their story through art. Now in year 11, the Rare Artist Program is focused on providing a national platform for artists to advocate through visual artwork and powerful artist statements. Rare Artist finalists participate in individualized artist-to-advocate coaching to strengthen their advocacy voice. Rare Artist awardees are presented with checks and invited to speak during Rare Disease Week on Capitol Hill in Washington, D.C. Their artwork is showcased throughout the year at various patient and biopharmaceutical conferences, displayed at the Rare Hub, social media, and website posts.
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Getting treatment and especially a cure would mean everything to me and my family. My brother also has this rare disease so the impact would be immense. My diagnosis was suspected as my brother is older; the disease is genetic. This art shows the path to hope; we cannot give up hope though we struggle daily. Hope is needed for all those suffering from any rare disease; my art highlights possibilities. The location in the trees is one that brings me a sense of calm and peace, but also shows the light at the end of the journey. COVID impacted me greatly as I was furloughed from my work decorating cupcakes at a local bakery. I also could not attend art classes so it has been isolating and limited some creative outlets. In the past year, I redirected my energies to creating a blog that highlights my life with a disability (kellonwheels.com). I hope it provides insight for others and raises awareness. Our annual fundraiser which is a bike ride, turned virtual, which limited participation. That decreased other community gathering possibilities to strengthen our cause. I hope my art provides thoughts of hope and peace for others. |
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