Last week, UMDF hosted a congressional briefing with the U.S. House of Representatives’ Mitochondrial Disease Caucus, which featured speakers from across the mito community, including Caucus Co-chairs Rep. Brian Fitzpatrick & Rep. Jim McGovern; Andy Dearth, Director of Advocacy, Marketing, and Communications, UMDF; Kira Mann, CEO, MitoAction; Emily Milligan, Executive Director, Barth Syndrome Foundation; Dr. Hilary Vernon, Associate Professor of Medical Genetics at Johns Hopkins University School of Medicine; and Reenie McCarthy, CEO, Stealth BioTherapeutics. 

The briefing focused on transforming patient outcomes for mitochondrial patients, including raising awareness, enabling innovation, and driving change.

The caucus, which traditionally ranges from 20 – 30 members, was the brainchild of UMDF leadership more than a decade ago to help Congress understand the needs of the mitochondrial disease community

You can watch it here:

UMDF Washington Update

House FY 2025 Labor-HHS Spending Bill – NIH Research Language Supported by UMDF Included

We are pleased to announce that the Fiscal Year (FY) 2025 Labor, Health and Human Services, Education, and Related Agencies (Labor-HHS) bill, advanced by the U.S. House of Representatives Appropriations Committee, includes language supported by the congressional mitochondrial disease caucus. We deeply appreciate the House committee’s efforts to advance crucial medical research supported by the National Institutes of Health (NIH) related to mitochondrial disease.

Mitochondrial Disease.—The Committee recognizes the need for the NIH to prioritize research on primary and secondary mitochondrial disease. A constellation of rare diseases linked to impaired mitochondrial function needs further research while potentially promising interventions work through the FDA approval process. At the same time, research continues to validate the substantial connections between mitochondrial function and major conditions such as Alzheimer’s, Parkinson’s, ALS, Muscular Dystrophy, heart failure, and long COVID. The Committee strongly encourages NIH to advance primary mitochondrial disease research, continue its ongoing outreach and collaboration with the FDA related to research that may lead to future mitochondrial disease-related drug approvals, ensure that support for long COVID research includes opportunities for studies to explore the role of mitochondrial impairment, and ensure that opportunities remain available to support collaborative research on mitochondrial disease to centralize a critical mass of research, clinical care, and provider education.

Supreme Court Overturns Chevron Doctrine

On June 28, 2024, the Supreme Court, in a 6-3 decision along ideological lines, overturned the four-decade-old Chevron deference doctrine. This ruling, stemming from litigation involving fishing industry regulation, removes the ability of federal agencies to interpret ambiguous laws passed by Congress. Previously, under the Chevron deference, judges would defer to regulatory agencies when congressional intent was unclear. The decision will have a significant impact on the highly regulated healthcare industry. The decision is also likely to have a significant impact on legislative advocacy. The deference afforded to agencies via the Chevron doctrine was only applicable when Congressional intent was not clear and “the statute is silent or ambiguous with respect to the specific issue.” The ruling now places greater emphasis on both more precise text as well as more express delegations of agency authority where Constitutionally permissible.

 House Energy & Commerce Committee Advances the Accelerating Kids’ Access to Care Act

Last month, the Energy and Commerce full committee marked up 13 bipartisan bills, including UMDF endorsed, Accelerating Kids’ Access to Care Act (S. 2372/H.R. 4758).  A markup memo with details on each of the bills can be found here, and a recap of the votes can be found here. This progress is a major milestone in the legislative process, as the bill will now be considered by the full House of Representatives. The bill would be of great benefit to children on Medicaid with rare and medically complex conditions like mitochondrial disease who often must travel to different states to receive care from medical specialists not available in their home state.  The bill creates a new pathway for pediatric providers to enroll in multiple state Medicaid programs if specific requirements are met. 

Missing from the markup were several bills that advanced out of the Health Subcommittee last month, including legislation addressing the Medicare telehealth flexibilities that expire at the end of calendar year 2024 and the pediatric rare disease priority review voucher program that expires at the end of fiscal year 2024. Committee leadership emphasized that they are working to advance fully paid for packages ahead of the programs’ expirations and noted that part of the delay regarding the telehealth package is due to the lack of an official Congressional Budget Office (CBO) score. UMDF continues to advocate for the passage of these vital bills.

Administration Acts on Clinical Trials

The FDA issued draft guidance to increase the enrollment of historically underrepresented patient populations in clinical studies by helping medical sponsors submit diversity action plans. This guidance outlines the format and content of these plans, the situations requiring them, and the submission process. It also details the criteria and process for evaluating waiver requests. The draft guidance is open for public comment until September 26, 2024. Read the press release here. On the same day, the White House hosted a clinical trials forum to identify strategies for increasing access, lowering participation barriers, and accelerating the discovery of effective therapies. The forum emphasized the benefits of patient-centered approaches, such as integrating patient voices into trial design, ensuring equitable trials, and treating patients as collaborators.