Angelman Syndrome Advocacy in the Current Federal Policy Environment
As we prepare for the second Angelman Syndrome Congressional Advocacy Day (March 5th), the FAST and ASF teams want to assure you of our steadfast commitment and focus on the priorities that matter most to families.
The new administration is swiftly focusing on issues highlighted during the campaign, and the 119th Congress is gearing up. The government usually acts in a deliberate and incremental way, but recent actions under President Trump have been rapid and far-reaching, giving Washington DC and the country a lot to consider.
Legislative priorities going into March 5 Advocacy Day
Medical Research
To further medical research, during last year’s Advocacy Day we proposed the inclusion of legislative language that provides guidance to the National Institutes of Health (NIH) on AS and we requested funding eligibility for AS researchers through the Department of Defense Medical Research Program in the FY2025 appropriations measures.
These requests were included in a bipartisan manner in the House and Senate versions of these bills. Unfortunately, the FY2025 measures are not final. In December, Congress passed what is called a Continuing Resolution (CR), which sustained the FY2024 funding levels until Congress passes a final set of appropriations bills or another CR. The next funding deadline is March 14 and Congress has not yet determined how to resolve the funding impasse.
On February 7, the National Institutes of Health issued a directive to limit the indirect costs that are funded on new and existing grant to 15% of the direct expenses.[1] This deep cut to research funding went into effect immediately, raising concerns from research institutions and patient organizations every corner of the country. Shortly thereafter, a U.S. District Court ordered a temporary pause to the caps pending more complete judicial review of the policy later this month.[2] Senator Susan Collins (R-ME) rightly noted, “(t)here is no investment that pays greater dividends to American families than our investment in biomedical research…”[3] Our organizations are coordinating advocacy on this issue with our researchers and will keep the community apprised of developments and how you can help urge Congress to protect NIH funding for AS.
Medicaid
Potential changes to Medicaid have been making headlines this year. A confusing set of administrative actions temporarily shut down Medicaid systems across the country. While jarring, as of February 18, 2025, no immediate cuts have been passed by Congress or the White House. The federal affairs team is closely monitoring for changes. That said, Medicaid is a partnership between states and the federal government. States may be taking individual action that is not happening at the federal level.
FAST and ASF work every day to protect Medicaid for our community. We engage with leaders in the space, and look to support legislation that affirms the value of Medicaid. This includes at our upcoming Advocacy Day with a dedicated session on Protecting Medicaid, and materials to share with Hill offices on the importance of Medicaid services for individuals with Angelman syndrome.
Caregiving
A key tenet of our legislative priorities will be focused on caregiving. In the 118th Congress, FAST and ASF supported the Credit for Caregiving Act, a bipartisan bill that provides a non-refundable tax credit to eligible family caregivers. The tax credit can be used for things like respite care, assistive technologies, and supplies for daily life (e.g., incontinence supplies). A key ask in 2025 is urging offices to support this legislation. This is bipartisan legislation that President Trump supported on the campaign trail. FAST and ASF are looking forward to reintroduction and are eager to support this policy in March.
Ensuring the Angelman syndrome community is heard in the therapy development and FDA review process
In our FY2025 appropriations priorities to drive new treatments, FAST and ASF proposed provisions to encourage FDA to focus attention on development of Angelman syndrome clinical endpoints and novel biomarkers, including assessment of developmental gains through the use of growth scale values and encouraging data sharing. For FY2026, we are redoubling these efforts by urging Congress to recognize the importance of patient experience insights in the drug development and regulatory review processes. The proposed appropriations language encourages FDA to use this vital data in their decision-making for treatments, recognizing improvements in activities of daily living in domains such as dressing, eating, and grooming.