February 28, 2022 marks the fourteenth Rare Disease Day! Since it was first launched by EURORDIS, thousands of events have taken place across the world, reaching millions of people and resulting in a growing public awareness of rare disease. We invite you to add your voice to this global initiative and participate in one, or more, of the many rare disease events listed below.
FARA also encourages our U.S. residents to mark Rare Disease Day by reaching out to your Members of Congress. So far, there are twelve FARA advocates participating in Rare Disease Week on Capitol Hill. If the time commitment of that program is not possible with for you, emailing your Members is a great option to raise awareness about FA, as well as support FARA’s CDMRP effort!
For the past year, FARA and the National Ataxia Foundation (NAF) have been working to get “hereditary ataxia” added to the Congressionally Directed Medical Research Program (CDMRP), a research program run by the Department of Defense. 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. Congress decides which conditions to include and how much money to appropriate for each area of research. If “hereditary ataxia” was added to the CDMRP, it would provide a new source of funding for FA researchers.
FARA and NAF are currently building a coalition of support for this initiative among Congressional Members. Your email could be the first step in getting your Members to join that effort!
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First, find your two Senators here and your House Representative here. You should have an option to “contact” them and that will usually take you to a form for their office.
- Share your rare disease story including how it has impacted you and your family, and make sure to include that there are currently no treatments for FA.
- Ask them to support FARA’s initiative to add “hereditary ataxia” to the CDMRP
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Let Brigid Brennan know if they are interested! FARA will follow up with supporting documentation and would be happy to meet with them to discuss further.
Thank you for celebrating Rare Disease Day through advocacy!
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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.
NORD’s Rare Disease Day event will be on Monday, February 28 at 1:30 pm ET. It will be a global celebration of our rare community, featuring incredible patient stories and surprise celebrity appearances!
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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!
No prior advocacy experience is needed! Training webinars available! For more information, click here.
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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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FDA will host a virtual public meeting on March 4, 2022 to join the global observance of Rare Disease Day. The theme for FDA’s Rare Disease Day is “Sharing Experiences in Rare Diseases Together.”
Patients, patient advocates, researchers and medical product developers may benefit from attending this public meeting on rare disease product development. During presentations and panel discussions various stakeholders will share their perspectives on and experiences in rare disease product development.
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Appropriations & Another Continuing Resolution (CR)
The current CR extended FY21 funding levels until February 18, 2022. Congress is expected to pass another short-term continuing resolution (CR) until March 11th. The extension allows Congress to continue negotiating on what to include in the FY22 budget. There is optimism coming from Congress that they will pass an omnibus in either March or April, however negotiations must still continue to proceed to ensure that occurs. Until a FY22 budget is passed, the federal government will remain operating at FY21 levels.
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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House Energy & Commerce Hearings Held
The House Energy & Commerce Committee held a hearing on PDFUA (Prescription Drug User Fee Act) on February 3rd. To view a recording of the hearing, click here. You can learn more about PDFUA and it's impact on the rare disease community from the EveryLife Foundation here.
The committee also held a hearing on ARPA-H (Advance Research Projects Agency on Health) on February 8th. To view a recording of the hearing, click here. You can watch an EveryLife presentation on the basics of ARPA-H and what it would mean for the rare disease community here.
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Breaking News! Senate Confirms
Dr. Robert Califf as FDA Commissioner
Tuesday, the Senate confirmed Dr. Califf as the 25th FDA Commission. The vote was 50-46. Dr. Janet Woodcock has been the Acting FDA Commissioner for the last 391 days. FARA thanks Dr. Woodcock for her stewardship and looks forward to working with Dr. Califf to advance treatments for FA.
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Your help is needed to protect the
Orphan Drug Tax Credit!
Right now, the US Senate is debating the Build Back Better Act which currently contains a provision to change the Orphan Drug Tax Credit. 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 future treatments by reducing the number of FDA-approved drugs for rare disease. Read statements opposing this action by NORD and the EveryLife Foundation.
Tell Congress to Protect the Orphan Drug Tax Credit Today!
Contact your Members through NORD’s Action Alert here.
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Regenerative medicine therapies, such as cell and gene therapies, hold promise for transforming medicine and creating options for patients living with difficult, rare, and often incurable diseases. The FDA plays a vital role in facilitating the development of regenerative medicine therapies, as well as in ensuring approved products are safe and effective for patients.
This webinar will bring together patients, caregivers, advocates, and other important stakeholders to celebrate and recognize the contributions that patients with rare diseases have made advancements in regenerative medicine.
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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 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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Director of Office of Science & Technology Policy (OSTP) Resigns
Eric Lander, OSTP director and President Biden’s cabinet-level science advisor, resigned on Feb 8, 2022 following reports that a White House internal investigation found he "bullied and demeaned his subordinates and violated the White House’s workplace policy" OSTP plays a critical role in setting policy and funding priorities for all federal science and technology initiatives and responds to global threats such as the Covid-19 pandemic, climate change; and, most recently, President Biden’s ARPA-H and Moonshot Cancer Initiative.
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Newborn Screening State News
Maryland, Mississippi and Iowa introduce Newborn Screening RUSP alignment legislation. This legislation expands newborn screening and ensures that all federal Recommended Uniform Screening Panel (RUSP) conditions are added to each state’s screening panel within a reasonable amount of time.
The Maryland legislation, HB 109/SB 242, would ensure that all RUSP conditions are added to their screening panel within two years. Maryland residents are encouraged to ask their state representatives to support this legislation using the EveryLife’s online form.
The Mississippi legislation, HB 927/SB 2900, would ensure that all RUSP conditions are added to their screening panel within three years. Mississippi residents are encouraged to ask their state representatives to support this legislation using the EveryLife’s online form.
The Iowa legislation, HSB 690/SSB 3122, provide a thoughtful approach to expanding newborn screening in Iowa and ensure that all conditions on the RUSP are implemented by the state within a reasonable amount of time. Iowa residents are encouraged to ask your state representatives to support this legislation by using the EveryLife’s online form.
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Economic Burden Data Published in Health Affairs
The EveryLife Foundation has co-authored an article published in Health Affairs entitled, “The Economic Burden of Rare Diseases: Quantifying the Sizeable Collective Burden and Offering Solutions.” In the piece, the authors synthesize a quartet of recent studies and reports, including the Foundation’s National Economic Burden of Rare Disease Study, shedding new light on the collective cost impacts posed by rare diseases, disorders and conditions. The authors propose several recommendations that, if taken, will address the gaps and challenges they identify.
Thanks to the engagement of the community, we now have a growing set of data that show what we have long known: rare diseases present a public health crisis and demand additional research funding, enhanced awareness, and improved access to diagnosis, care, and treatment. Learn more and view the article, click here.
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