250 Advocates + 100 Congressional Meetings =
SUCCESS!
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Over 250 people registered for the third United Against Ataxia Hill Day, resulting in 100 Congressional meetings covering 44 states! The time and attention that each and every advocate put into making this event a success was apparent from all the positive feedback we received.
"I did not know anything about Ataxia before today, thank you for sharing your story," was a comment we heard often. By combining our voices, we raised awareness and educated our legislators about our journey of being diagnosed and living with ataxia, and our search for treatments!
It was particularly exciting to highlight that our Hill Day followed National Ataxia Awareness Day! This accomplishment was a clear demonstration of the commitment the ataxia community has to elevate the conversation. FARA and NAF are very grateful for Senators Christopher Murphy (D-CT) and Cindy Hyde-Smith (R-MI) for co-sponsoring a Resolution declaring September 25, 2021, National Ataxia Awareness Day. (S.Res. 339).
Our primary “ask” for Hill Day was to add "Hereditary Ataxia" to the Congressionally Directed Medical Research Program (CDMRP), which would provide additional research funding. That request was well received. Many Members not only showed interest in supporting our CDMRP request but several offices offered to help us with the application. There is a lot of work to do over the next few months, but we are off to a great start. We will keep you posted so that you too can continue the conversation with your Members.
The Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813), a bill that has several provisions that would help the ataxia community, has continued to gain momentum. As of today, there are 326 co-sponsors in the House and 40 co-sponsors in the Senate. Some of whom signed up after our Hill Day. We can only imagine that hearing our personal stories regarding the need for more research in the degenerative, neuromuscular space helped with that effort. You can check if your Member is one of the 326 who have signed on in the House or 40 that have signed on in the Senate. If they did, thank them! If not, send them an email asking for their support for the ACT for ALS (H.R. 3537/S. 1813).
This incredible day concluded with the first United Against Ataxia Celebration. If you did not have a chance to see this short but powerful program, you can check it out below!
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NORD and Everylife Foundation Oppose Proposed Changes to the Orphan Drug Tax Credit and seek your help! |
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Right now, Congress is developing legislation that could include damaging changes to 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 of the Build Back Better Act in the House of Representatives 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.
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Tell Congress to Protect the Orphan Drug Tax Credit Today!
Contact your Members through NORD’s Action Alert here.
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NIH Director, Dr. Francis Collins, Resigns
Dr. Francis Collins, Director of the National Institutes of Health (NIH), will be stepping down from his position after more than 12 years of service. Under Dr. Collins’ leadership, NIH funding grew by 38%, enabling NIH’s investments in translational science, precision medicine, and more. Dr. Collins was committed to addressing racial disparities, clinical trials enhancements, and interdisciplinary data and spent decades unlocking the potential of the human genome to guide treatments and cures for diseases once thought incurable. No decision has been made on an interim director. Dr. Collins has stated that he plans to continue to lead his lab within the National Human Genome Research Institute.
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FARA supports robust and timely appropriations
FARA joined Research!America and over 200 other organizations in urging Senate and House leadership to complete the Fiscal Year 2022 appropriations process before the current continuing resolution (CR) expires on December 3, 2021. 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. Read the letter here.
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Our partners at Research!America offered a new insight about research appropriation this month. The National Retail Federation predicts Americans will spend $10.14 billion on Halloween this year; that’s almost double the $5.25 billion NIH will invest this year in research on dental/oral and craniofacial disease, diabetes, eating disorders, food allergies, nutrition, and obesity combined! Check out their Research Takes Cents page for more examples comparing consumer spending to the costs associated with conduction research.
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Senate Adds Co-Sponsors to the Newborn Screening Saves Lives Reauthorization Act
Senators Jeanne Shaheen and Debbie Stabenow joined the NBSSLA as co-sponsors. The Everylife Foundation is encouraging advocates to ask their Senators to support this important legislation through this action alert.
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Update on ARPA-H Listening Sessions from the
Everylife Foundation
Between July and August, the NIH and Office of Science and Technology Policy (OSTP) held fifteen listening sessions on the ARPA-H proposal. Ten sessions were open to the public and provided an opportunity for real-time question submission and response. The remaining five sessions, led by OSTP, were invite-only and were attended by various stakeholder organizations, including the EveryLife Foundation, representing patient advocacy groups, biomedical professional groups, venture capital firms, private industry, and other non-biomedical STEM professional organizations. Below are just a few of the highlights. A full report can be found here.
The sessions emphasized that the program should focus on technologies with wide-ranging application, collapsing data siloes to collect uniform and interdisciplinary data, and AI. Other considerations include wearables/digital technology, holistic programs, and new treatment technologies. There was also considerable attention given to ensuring that the program is diverse, multidisciplinary, and equitable.
While these listening sessions have concluded, OSTP and NIH will continue to seek perspectives from stakeholders on ARPA-H. Public comments can be submitted to ARPAHcomments@nih.gov. The Administration has also convened a Fast Track Action Committee (FTAC) of the National Science and Technology Council (NSTC) to identify synergies between ARPA-H and the work of NIH and other Federal agencies and to promote interagency coordination, where appropriate, in the design of ARPA-H’s approach.
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House Agriculture/FDA Appropriations Subcommittee Ranking Member Jeff Rotenberry
Temporarily Steps Aside
Congressman Jeff Fortenberry (R-NE) has temporarily stepped aside from the House Appropriations Committee and his position as ranking member of the Agriculture/FDASubcommittee. Rule 25 of the House Republican Conference Rules requires "A member of a standing, select, joint or ad hoc committee, or any subcommittee thereof, who is indicted for a felony for which a sentence of two or more years imprisonment may be imposed, shall submit his or her resignation from any such committees to the House promptly." Congressman Fortenberry has been indicted on a charge of concealing information and lying to the FBI regrading an investigation into illegal campaign contributions. Congressman Andy Harris (R-MD) was named this week as the Acting Ranking Minority Member on the House Ag/FDA Subcommittee.
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Advocacy Training Webinars
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JOIN NORD'S UPCOMING RARE DISEASE ADVISORY COUNCIL (RDAC) STAKEHOLDERS MEETING
All rare disease advocates are invited to join NORD for Project RDAC's Stakeholders Meeting, which will be held virtually on Tuesday, November 16th from 2:30-4:00pm ET. Join to learn more about how to engage in the RDAC process. You can check the status of your state’s RDAC here.
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NORD Calls On Governors To Protect Access
To Telehealth
NORD, along with FARA and 235 other organizations representing patients are calling on governors to prevent the flexibilities that have allowed patients to access necessary health care providers via telehealth from expiring. These licensure flexibilities are critical to ensuring that patients, particularly patients with compromised immune systems that are still vulnerable to contracting COVID-19, can access health care across state lines as the pandemic continues. Read the letter sent to all 50 governors here!
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Health care costs higher for people with rare diseases NCATS Releases Economic Burden of Disease Study
On October 22nd, NCATS released a study on the impact of rare diseases on patients and healthcare systems. The study found that health care costs for rare disease patients are underestimated and that they are three to five times higher than costs for individuals without rare diseases. The study used patient medical records to estimate average medical costs for rare disease patients and to track the diagnostic journeys of four patients. A link to the study can be found here.
GAO Releases Study on the Cost of Rare Diseases
On October 18th, 2021, the Government Accountability Office (GAO) released its study on the cost of undiagnosed and untreated rare diseases. The GAO study was based on literature review and interviews and found that “those with undiagnosed, misdiagnosed, or untreated rare diseases may face various negative outcomes,” that “a person’s health can suffer when appropriate, timely interventions are not provided or when interventions based on misdiagnoses cause harm,” and that “multiple diagnostic tests, medical appointments, and ultimately unwarranted interventions can add to the costs of the disease.” View the GAO study here.
Earlier this year, the Everylife Foundation also released the National Economic Burden of Rare Disease Study. These landmark reports will help ensure that the experience of the rare disease community is reflected accurately in policy discussions and serve as powerful tools that underscore the public urgency and awareness of the public health crisis of rare disease.
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Newborn Screening State News
Delaware
The Delaware Governor signed into law legislation that will require dried blood spots destroyed once screening and testing is complete, allows for newborn screening fees to be used to defray expenses associated with the newborn screening program and any other program that ensures optimal health across the lifespan of the maternal and child health, and that the Department of Public Health can only notify the physician of record of abnormal test results.
Wisconsin
On October 13th, an amendment was offered to AB 181, a bill that would add Krabbe disease to Wisconsin’s NBS panel. The amendment would delay the requirement that the Department of Health Services determine if they should add lysosomal storage disorders to state NBS by one year. The amendment was also offered to the companion bill to AB 181, SB 194. Neither amendment has been voted on.
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Rare Artist Finalist from FAmily! |
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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."
Congratulations Kelly Hennessy on being selected as one of the top 20 finalists in the Rare Artist contest. Awardees will be announced by the Everylife Foundation in the second week in November. Good luck Kelly!
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