By Paula Eichenbrenner, TMA Executive Director.
“Strength in numbers” is a cliche because it’s true, and every year, EveryLife Foundation for Rare Diseases assembles nearly 1,000 rare disease advocates during hashtag#RareDiseaseWeek. The Myositis Association is showing our stripes with advocates – myositis patients, care partners, and clinicians – from seven (!) states here in Washington for #RareDC2025. 🦓 💙
You can join our dazzle of zebras by contacting your Representatives and Senators. Ask that they support steady and robust leadership, federal biomedical research funding, and public health agencies. While many federal agencies support our rare disease ecosystem, the federal agencies housed within the Department of Health and Human Services (HHS) are essential to supporting timely diagnosis, conducting research, enabling therapy development, facilitating insurance coverage, and implementing public health programs.
hashtag#TheMyositisAgenda led by TMA aligns to #RareDC2025 and calls for:
🔵 🦓 Supporting the committed scientists, clinicians, and teams that are advancing our knowledge of myositis, especially at the National Institutes of Health’s (NIH) and the NIEHS, NIAMS, and NCATS institutes. We have to stand for science, now more than ever. Rare diseases need more research, not less.
🔵 🦓 Supporting the reauthorization of the Rare Pediatric Disease Priority Review Voucher program and include the Accelerating Kids’ Access to Care Act (AKACA). These efforts were supported with broad (even unanimous) support in past Congresses, and are important to so many Cure JM Foundation families.
🔵 🦓 Maintaining strong non-profit partnerships with agencies including NIH and FDA. Families work with private non-profit organizations like ours to fill critical research funding gaps, which can be a win when private funding significantly supplements federal appropriations and enhance government efficiency.
🔵 🦓 Enabling a better research ecosystem with policies that incentivize rare drug development (noting again the importance of incentives like Priority Review and Vouchers), and allow industry to innovate more easily on clinical trial design (for example, by evaluating outcome measures that actually reflect clinician experience and patient priorities).
🔵 🦓 Expanding access to therapies and potential treatments. TMA is encouraged that the Georgia Senate has passed the Georgia Hope for Patients Act (S.B. 72), and we’re tracking many innovative proposals in other states.
Learn more at links below. And DM me with ideas to drive additional myositis awareness! 💙 Let’s scale 🆙 and 🆙 and 🆙 again, from Rare Disease Day on February 28 to Autoimmune Awareness Month in March to Proclamation 50 and Myositis Awareness Month in May to World Myositis Day on September 21.
💻 https://www.myositis.org/blog/rare-diseases-need-more-research-not-less/
💻 https://www.myositis.org/blog/tma-board-vice-chair-testifies-in-support-of-georgias-hope-for-patients-act/
💻 https://everylifefoundation.org/rare-advocates/