The 10th Annual Rare Disease Week started July 14, and runs through July 22! It is virtual this year, so regardless of where you are, you can do it! It is a great opportunity to learn about pending US legislation impacting rare disease, gain some advocacy training and, if US based, speak directly to your Members of Congress &/or their staff.
There is still time for you to register and tune in for many of the excellent programs offered during Rare Disease Week.
If you are registering now, it is too late to sign up for Congressional meetings. However, do not despair! Your voice can still be heard! Send your Members an email on July 20 or 21, the days your rare disease colleagues will be holding meetings. You can find contact information for your US Senators here and your US Representative here.
For those who have already registered and have meetings scheduled, here are the three items that FARA is currently working on which you can advocate for in your meetings!
Please let Brigid Brennan, brigid.brennan@curefa.org, know how it goes, especially if your Member agrees to support one or more of FARA’s initiatives!
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1. National Ataxia Awareness Day Resolution
Senators Elizabeth Warren (D-MA) and Cindy Hyde-Smith (R-MI) co-sponsored the first National Ataxia Awareness Day Resolution which occurred on September 25, 2020. Senator Warren is unable to do it again this year. Help FARA and NAF find a Democratic Senator to co-sponsor with Senator Hyde-Smith. Here is a one pager with more information.
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2. Congressionally Directed Medical Research Program (CDMRP)
FARA and NAF has been working to get “hereditary ataxia” added to the CDMRP, which is run by the Department of Defense. There are over 45,000 veterans who suffer from ataxia and that number multiplies when you add family members who have ataxia. 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 for research. If added, this would provide a new source of funding for FA researchers.
The request was submitted to the Defense Appropriation Sub-Committee for consideration for FY22 but it did not make it out of Committee. We are starting early in building a coalition of support for FY23 and already have several Members’ support. Share this information (CDMRP) with your Members and ask them to support this important initiative.
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3. Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813)
On May 25, 2021, US Representatives Jeff Fortenberry (NE-01) and Mike Quigley (IL-05), with Senators Chris Coons (D-DE) and Lisa Murkowski (R-AK) re-introduced the bipartisan, bicameral Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813).
Although this bill is directed primarily towards ALS, there are very important parts that help all rare neuromuscular diseases like FA. The three initiatives in this legislation that would help FA research are:
· Establish a Health and Human Services (HHS) Public-Private Partnership for Rare Neurodegenerative Diseases between the NIH, the FDA, and eligible stakeholders with a connection to the patient population(s) to advance the understanding of rare neurodegenerative diseases and foster the effective development and evaluation of treatments;
· Commission the publication of an FDA Action Plan on rare neurodegenerative diseases;
· Implement an FDA grant program to fund research and therapy development for life-threatening or severely debilitating rare neurodegenerative diseases.
Let’s help get this bill passed! You can check if your Member is one of the 273 who have signed on in the House or five that have signed on in the Senate. If they did, thank them! If not, ask them to support Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537/S. 1813). Share this one pager developed by our partners at MDA which explains why these important initiatives would expedite the search for a cure for FA.
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Supreme Court upholds the Affordable Care Act
In June, the US Supreme Court issued a decision effectively upholding the Affordable Care Act (ACA) by a 7-to-2 vote. This ruling is a triumph for all patients with a rare disorder and their loved ones, and will ensure that patients retain the vital protections and access to care enabled by the ACA. Read NORD's statement celebrating this announcement.
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A New Cabinet Member: The Senate has confirmed Dr. Eric Lander to be Director of the Office of Science and Technology (OSTP), the first director to be part of the President’s cabinet. Dr Lander, a well-respected mathematician and geneticist, is the first life scientist to hold this position.
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Support in the House and Senate for increasing investment in science and technology
The National Science Foundation for the Future Act (H.R.2225) and Department of Energy Science for the Future Act (H.R.3593) were both passed earlier this month. Part of what President Biden calls a “once in a generation” investment, these bills put forth important policies and authorize new funding levels designed to drive faster innovation in science and technology at NSF and DOE. The House bills are expected to be conferenced with the Senate-passed United States Innovation and Competition Act (S.1260).
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Newborn Screening Saves Lives Reauthorization Act Passes the House
On June 24th, the Newborn Screening Saves Lives Act, H.R. 482, passed the House with a 325-103 vote. The bill now moves to the Senate. The Everylife Foundation has provided this action alert to encourage advocates to ask their Senators to support the legislation.
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The Verifying Accurate Leading-Edge IVCT Development (VALID) Act Re-introduced in Congress
On June 24th, the VALID Act was reintroduced in the House and Senate, by Reps. DeGette (D-CO) and Bucshon (R-IN) and Sens. Bennet (D-CO) and Burr (R-NC). The legislation seeks to alter the regulatory oversight of in vitro diagnostics, including laboratory-derived tests (LDTS) such as newborn screening and many genetic tests. The legislation would create a single, diagnostics-specific regulatory pathway for the first time at the Food and Drug Administration (FDA). For more information, please read here.
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21st Century Cures 2.0 Discussion Draft Released
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President’s FY22 Budget increases funding for federal agencies supporting research
National Institutes of Health (NIH)
The President's budget proposal recommends increasing the base budget of the National Institutes of Health (NIH) by approximately $2.5 billion, or 5.9%, and providing $6.5 billion for the establishment of a new science and technology (S&T) initiative called “ARPA-H.”
Food and Drug Administration (FDA)
The FDA is responsible for evaluating the safety and effectiveness of prescription and tobacco products, as well as nearly 80% of the nation’s food supply; FDA-regulated products account for 20 cents of every dollar American consumers spend. The President’s request includes approximately $3.6 billion for FDA (exclusive of mandatory funding for FDA under the 21st Century Cures Act and one-time funding items). This increase of nearly 10.6% over 2021 will help assure FDA can meet its crucial, multi-faceted mission.
Centers for Disease Control and Prevention (CDC)
Funding for the Centers for Disease Control and Prevention (CDC) would increase by $1.6 billion, or 11%. The ongoing COVID-19 pandemic, other recent outbreaks of infectious diseases, and challenges like antimicrobial resistance underscore the vital role played by the CDC.
Agency for Health Research and Quality (AHRQ)
AHRQ is the lead federal agency tasked with the essential mission of translating medical and public health research into more effective and affordable health care for Americans across the country. The President’s request proposes a budget of $380 million, an increase of 12%, over 2021.
National Science Foundation (NSF)
President Biden’s budget request also includes an increase of $1.7 billion, or 20% for the National Science Foundation (NSF). Decades’ worth of NSF research was utilized in the COVID-19 response, such as computer software simulations of the molecular structure of the virus. NSF’s portfolio is intentionally diverse; investments yield unknown discoveries, spanning biology, economics, engineering, mathematics, computational science, data analytics, the social and behavioral sciences, and other high-impact disciplines.
Congress will now hold hearings and develop and consider a budget resolution. After the hearings are held, the Appropriations Committee drafts the 12 appropriations bills and Congress must pass all 12 to fund the government. To learn more about the appropriations process, check out RDLA’s tip sheet here or video tutorial here.
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Advocacy Training Webinars
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Tickets are now available!
Rare New England's 2021 Annual Conference will be held virtually on
October 23, 2021. It will be a day of education and information to Rare Disease patients, caregivers, students, and professionals.
Caregiver and Patient tickets are FREE this year.
View full agenda and register here.
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The Rare Artist Program was established in 2010 to exhibit the unique gifts of individuals affected by rare disease and to promote the expression of their stories through art. Now in its 11th year, the Rare Artist Contest provides a national platform for artists with, or affiliated with, rare diseases to showcase their artwork and highlight their stories. |
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BONUS ADVOCACY TRAINING!
In the spirit of Advocacy, the Everylife Foundation will be offering individualized coaching for the twenty Rare Artist finalists. Before sharing finalist profiles to the public, the EveryLife staff will work with artists on their advocacy goals, create an artist’s profile and provide training on how to share your artwork with the community for the public vote. We hope to empower artists in sharing their advocacy story and provide them with the proper tools to turn their story from an Artist to an Advocate.
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Newborn Screening State News
Arizona and Ohio pass RUSP alignment legislation!
The Governors of Ohio and Arizona have signed into law legislation that will help save the lives of hundreds of babies born in these states each year. Due to broad legislative and executive support, both laws, HB110 in Ohio and SB1824 in Arizona, were passed through the state budget process. The laws require the states to screen newborn babies for any disorder on the federal Recommended Uniform Screening Panel (RUSP), implements a timeline in which the screening must begin, and ensures that resources will be available to fund all conditions added to the RUSP in the future. Each year, new screening tests are developed to diagnose diseases before it is too late to receive treatment. Thank you to all the advocates who helped to make this possible. For more information, please read the Everylife Foundation press release here.
North Carolina NB 736 passed in NC House of Representatives
HB 736 is newborn screening legislation that implements a timeline on when the screening must begin for a condition following its addition to the Recommended Uniform Screening Panel (RUSP) that will eliminate the unnecessary delay in screening for diseases, ensuring the North Carolina babies born with debilitating and life-threatening diseases are diagnosed and treated at the earliest age possible and without devastating delays.
North Carolina advocates can ask their State Senator to support this bill here.
Texas and North Carolina
Texas and North Carolina added spinal muscular atrophy to their state newborn screening panel. Click to read more about Texas and North Carolina.
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