Five weeks out from brain surgery, I wasn't sure if I would be able to attend Rare Disease Week this year. My energy level is not yet back to 100%. Because this is a cause that is close to my heart, I decided to attend but prioritized my time and energy on the legislative conference and meeting with my representatives' offices, and I'm glad I did. It was wonderful catching up with my fellow rare disease advocates, and Rare Disease Week feels like a reunion with friends, especially with my amazing Pheo Para Alliance team.
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| Phearless Pheo Para Advocates |
Last year, the vibe at the rare disease legislative conference felt uncertain but determined. We were facing cuts to rare disease research and Medicaid, and key pieces of rare disease legislation were on the brink of being passed but then pulled at the last minute due to political crossfire. This year, we had several major victories to celebrate. Nearly all of our recent rare disease legislative asks were passed and signed into law in February (
https://everylifefoundation.org/congress-passes-five-year-reauthorization-of-rare-pediatric-disease-prv-program/). This included reauthorizing incentives for drug companies to develop rare disease treatments, additional funding for the NIH and their center for rare disease research, and streamlining access to out-of-state specialists for Medicaid patients.
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| Deep dive on policy asks at the Rare Disease Week legislative conference |
Rare disease legislation benefits everyone by accelerating development and approval of new therapies and eliminating hurdles to health care access, and many rare disease bills have both Republican and Democratic co-sponsors. Even with strong bipartisan support, these victories didn't happen overnight but took years of persistence and advocacy. There is still much more to do, but this past year has shown that even when Congress feels hopelessly dysfunctional, our voices matter and we can get things done.
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Celebrating the 5 year re-authorization of FDA Priority Review Vouchers,
an incentive to develop rare disease treatments |
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