Friedreich's Ataxia has Been Added to the DOD's Congressionally Directed
Medical Research Programs!
|
|
The CDMRP was created in 1992 in response to a group of grassroots advocates who wanted more research focusing on breast cancer. Congress created the CDMRP as a program where they could not only appropriate funds but specifically direct how those research dollars would be used. The Department of Defense runs the program and it now includes many other conditions. Congress decides each year which conditions are added and how much funding the program gets.
FARA, in partnership with NAF, initiated an advocacy campaign about eighteen months ago to tap into this resource. It was the top ask during our United Against Ataxia Hill Day last September and during Rare Disease Week this February. Additionally, many families joined the effort by meeting, emailing, or calling their Members of Congress to ask them to support this initiative. This amazing funding opportunity is a direct result of the collective power in raising our voices together through advocacy!
This is huge news for our researchers and our community. Today we celebrate! Tomorrow, we begin preparing our efforts to keep FA on the CDMRP next year, as well as supporting NAF’s effort to expand the language to include all hereditary ataxias. We will have to advocate yearly for funding through this program and will continue to need everyone’s help to make that happen. Stay tuned for future advocacy opportunities!
|
|
Rare Disease Day/Week Report |
|
Terminal Tower in Cleveland, OH |
|
Governor Mario M Cuomo Bridge in NY |
Zakim Bridge in Boston, MA |
|
FARA supports the NORD Light Up for Rare Campaign!
Buildings, monuments and landmarks across the US and around the world were lit up in the Rare Disease Day colors on February 28. FARA joined the effort by successfully illuminating the Pennsylvania Capitol Building in Harrisburg, PA and the Zakim Bridge in Boston, MA.
In addition, NORD’s #ShowYourStripes campaign was a huge success. Thanks to thousands of posts, #RareDiseaseDay was present on Twitter's top 10 trending list on February 28, with hundreds of posts published on LinkedIn, Facebook, Instagram, and TikTok as well.
|
|
Thirteen members of the FA community were among the 953 advocates in Rare Disease Week on Capitol Hill 2022, 664 of which participated in 370 Congressional meetings. Thank you for all who took the time to share your rare disease journey, demonstrating why legislative action is so important to expediting treatments! If you missed any of the EveryLife Foundation programs, you can view the recordings here.
|
|
Congratulations to FARA Ambassadors Shandra Trantham and Mary Nadon Scott for placing in the top 50 point earners of the EveryLife Foundation Rare Disease Week Scavenger Hunt. They earned $3500 in grant money for FARA. Thank you Shandra and Mary! |
|
Congratulations Kelly Hennessy! Kelly was one of ten people selected as a Rare Artist Award Recipient and her piece, Hope Through Trees, was showcased in the Rare Hub during Rare Disease Week.
|
|
FARA Ambassador Secures
Vermont Proclamation
Kudos to FARA Ambassador Mary Nadon Scott who secured an Executive Department Proclamation recognizing February 28, 2022 as Rare Disease Day in Vermont. Thank you Governor Scott for your support of rare disease patients!
|
|
The National Institutes of Health held Rare Disease Day at NIH 2022 on Monday, Feb 28. If you missed it, you can view the recording here.
|
|
FDA hosted a virtual public meeting on March 4, 2022 in observance of Rare Disease Day. The theme for FDA’s Rare Disease Day was “Sharing Experiences in Rare Diseases Together.” If you missed a session or want to revisit the day, you may watch the full recorded webcast here.
|
|
In recognition of Rare Disease Day 2022, the Oxford-Harrington Rare Disease Centre (OHC) hosted its 2nd annual Rare Disease Day Webinar on February 28, titled Advancing Novel Treatments for Neurological Rare Diseases.
The webinar featured a panel of experts discussing areas of unmet needs in neurological rare diseases including:
Richard Wade-Martins, MA, DPhil, Professor of Molecular Neuroscience, University of Oxford. Dr. Wade-Martins spoke about his research and therapeutic plan related to Friedreich's ataxia.
The Oxford-Harrington Rare Disease Centre (OHC) is a partnership between Harrington Discovery Institute at University Hospitals and the University of Oxford in the UK, combining world-leading strengths in research and therapeutics development, to address unmet need in rare diseases. Click here to watch the full webinar.
|
|
Fiscal year 2022 budget passed!
The FY22 omnibus budget package was passed by Congress on March 9th and signed by President Biden on March 15. The federal government is now funded through the remaining 2022 fiscal year, which ends on September 30th. Here are a few highlights we can all celebrate!
-
Friedreich’s Ataxia added as an eligible condition under the Peer Review Medical Research Program (PRMRP), a category of the CDMRP, which will allow FA researchers access to a new funding opportunity. The PRMRP has $370 million available to support accepted research.
-
$5 million in funding for the ACT for ALS research grants program, which gives the FDA funding to infuse into rare neurodegenerative disease clinical research.
-
$2.25 billion increase in medical research funding at the NIH.
-
$27 million increase in funding for important programs at the National Center for Advancing Translational Science (NCATS).
-
$102 million increase for programs at the FDA.
-
$1 billion start for ARPA-H.
|
|
House Energy & Commerce Hearings Held
The House Energy & Commerce Committee held a hearing on March 17th titled The Future of Medicine: Legislation to Encourage Innovation and Improve Oversight which discussed numerous health related bills including ARPA-H, Cures 2.0, PDFUA (Prescription Drug User Fee Act) and the BENEFIT Act to name a few. To view the recording of the hearing, click here.
|
|
Dr. Robert Califf Confirmed as
FDA Commissioner
On February 15, the Senate confirmed Dr. Califf as FDA Commissioner with a 50-46 vote. 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.
FARA joined multiple letters of support to Congressional leaders for Dr. Robert Califf to become the next FDA Commissioner, and now look forward to working with Commissioner Califf to expedite treatments for FA.
|
|
ARE YOU READY TO TAKE YOUR ADVOCACY JOURNEY TO THE NEXT LEVEL?
"Making an Impact on State and Local Policy”
A six-week seminar series for advocates with prior advocacy experience
hosted by the Rare Disease Legislative Advocates (RDLA)
When? May 9th – June 24th, 2022
Applications close April 1, 2022
|
|
An offering from the community, for the community
- Learn about rare disease state policy issues that affect the rare disease community.
- Identify opportunities to engage with key stakeholders on the local level.
- Develop a pathway toward year-round advocacy engagement.
- Receive a completion certificate.
|
|
Rare Advocacy Learning and all RDLA events and resources are offered free of charge to patient advocates. |
|
Rare Artist Webinar
Join Rare Artist in partnership with Rare Disease Legislative Advocates (RDLA) for a webinar on April 7, 2022 at 12:00 PM ET – 1:00 PM ET. The webinar will feature a panel of talented artists with rare diseases. Hear their stories, learn tips and techniques, and ask questions. Then, enjoy an interactive breakout session led by an artist to practice sharing your own story through art.
|
|
The fourth annual United Against Ataxia Hill Day will be held virtually on Wednesday, September 21, 2022. Mark your calendars now so that you will not miss this important event! |
|
Newborn Screening State News
The Governor of Mississippi signed HB 927/SB 2900 into law, ensuring that all RUSP conditions are added to their screening panel within three years. This action will save the lives of nearly 100 babies born in the state each year.
The Maryland legislation, HB 109 passed out of the Maryland House of Representatives in a 130-1 vote. SB 242, the Senate companion bill, passed out of the Senate in February and awaits action in the House.
The Iowa legislation, SF 2345 passed out of the Iowa House of Representatives with a unanimous vote. The bill is now headed to the Governor for her signature.
|
|
Living with a rare disease means managing unique challenges, including frequent doctor visits, rigorous treatment regimens and hospitalizations, and exposure risks. While quality and duration of life continues to improve thanks to improved diagnosis and treatment approaches, individuals living with rare diseases still face disparities in achieving traditional life milestones.
That’s why the EveryLife Foundation for Rare Diseases established the #RAREis Scholarship Fund – to enrich the lives of adults living with rare diseases by providing support for their educational pursuits. Thanks to the support of Horizon Therapeutics plc, one-time awards of $5,000 each will be granted to up to 53 recipients for the Fall 2022 semester. Apply here.
|
|
A Rare Report Card: NORD Grades All 50 States on
Rare Disease Policy Issues
The National Organization for Rare Disorders (NORD®) published the latest edition of its annual State Report Card, which rates all 50 states and Washington, DC on the most important issues affecting the more than 25 million Americans living with a rare disease. The State Report Card was compiled using data through November 2021 and provides detailed analysis in grading each state on its performance on nine major policies of importance to the rare disease community such as newborn screening, Medicaid eligibility and prescription drug out-of-pocket cost protections.
|
|
KEY TAKEAWAYS FROM THE NEW REPORT INCLUDE:
-
Only six states received an "A" grade on protecting patients from increasingly high prescription drug out-of-pocket costs.
-
Despite the critical importance of telehealth during the COVID-19 pandemic, 18 states received a failing grade when it comes to removing barriers for patient access to out of state providers via telehealth.
-
Short term, limited duration insurance continues to be unregulated in many states, as only 11 states received an A grade for their oversight and regulation of these plans, which fail to offer the comprehensive health care coverage vital to rare disease patients.
-
A total of 21 states have signed legislation into law creating a Rare Disease Advisory Council, including six in 2021 alone. These councils give rare disease patients a unified voice in state government and act as an advisory body to government leaders.
|
|
For more information and to view or download the full state-by-state report cards, including maps, patient stories, and resources, click here.
|
|
Research!America just released America Speaks! Volume 22, their annual poll data summary. Among other findings, the report highlights the public’s significant, sustained, bipartisan support for federal investment in science and technology – one of the strongest messages we can share as research advocates.
Even more persuasive is the United for Medical Research’s latest report, NIH’s Role in Sustaining the U.S. Economy, which looked at the national- and state-level impact of NIH research funding. In addition to lifesaving discoveries, NIH-funded research provides exponential return on investment; every $1 of NIH-funded research translates to $2.60 of economic activity. In FY21 alone, funding supported an estimated 552,444 jobs nationwide and produced $94.18 billion in new economic activity.
|
|
|
|
|
|
|
