Advocacy Update – February 2024: Redefining “Rare” For the World, Funding for 2024 & Beyond

Rare Disease Day
On February 29, we’ll join the nearly 300 million affected patients and their loved ones across the world in marking Rare Disease Day.

It’s a day where we collectively raise awareness and generate support for those living with rare disease — but perhaps just as important, it’s a time when we help redefine “rare” by showing the true impact of such diseases extend to all of us. It’s not a patient and their family. It’s coworkers, classmates, friends, neighbors, and much more.

That’s why organizations like UMDF exist. To ensure no one is alone on this journey. To ensure no one hears the word “rare” and thinks it means forgotten or neglected.

You’ll find lots of ways you can get involved in Rare Disease Day on UMDF social media pages, with perhaps the most powerful of those being letting Capitol Hill hear your voice.

You can take action today at, where you can ask your local House Representative to join the Mitochondrial Disease Caucus, ask for support for the Medical Nutrition Equity Act, encourage regulatory action on mitochondrial therapies, and more.
There are a variety of virtual advocacy events happening over the next week or so, including:

February 23, 1 p.m. ET – Rare Disease Day Policy & Advocacy Town Hall presented by National Organization for Rare Disorders

February 28, 5:30-7:30 p.m. ET — White House Rare Disease Forum Livestreamed at:

February 29, 9 a.m. ET – National Institutes of Health’s Rare Disease Day

February 29, 10 a.m. ET — Energy and Commerce, Health Subcommittee Legislative Hearing

March 1, 9 a.m. ET — U.S. Food and Drug Administration’s Rare Disease Day

For those of you in the D.C. area, there are opportunities in-person on Capitol Hill, as well.

Advocating for Mitochondrial Disease Priorities in the FY 2025 Appropriations Bill
Congress has only several working days ahead of the first of two upcoming deadlines to prevent a partial government shutdown. Lawmakers continue to negotiate details of the fiscal year (FY) 2024 spending bills, as current funding for federal agencies is set to expire on March 1 for some agencies, including the Food and Drug Administration (FDA), and March 8 for the remainder, including Health and Human Services. These are also significant dates for several essential healthcare programs that either run out of funding or face dramatic cuts to existing funding once those deadlines pass. Accordingly, negotiations continue for the potential inclusion of healthcare priorities in the FY 2024 spending package.

Looking ahead to the FY 2025 budget, preparations for FY 2025 will be soon underway, with President Joe Biden sending Congress his fiscal 2025 budget proposal on March 11, days after his State of the Union address and the aforementioned deadline to avoid a government shutdown.  The release of the President’s budget request is seen as the official kick-off to appropriations season.

Notably, the appropriations season is when UMDF advocates with help from the Congressional Mitochondrial Disease Caucus, co-chaired by Reps. McGovern (D-MA) and Fitzpatrick (R-PA), for the inclusion of mitochondrial disease as an area of medical research eligible for the Department of Defense (DoD) Peer-Reviewed Medical Research Program. Due to this inclusion, there has been at least $60 million in funding for mitochondrial disease through DOD’s Peer-Reviewed Medical Research Program.  We will continue to advocate for this critical provision in the Fiscal Year 2025 appropriations package.  We will also focus our appropriations efforts on language that directs the National Institutes of Health (NIH) to prioritize mitochondrial disease research.  Please be on the lookout for a forthcoming action alert on how UMDF members can support these efforts with their members of Congress.