Advocacy Update – February 2023: Could ARPA-H Change the Research Landscape for Rare Diseases?

For many years, UMDF has worked to encourage greater mitochondrial disease research funding through the National Institutes of Health (NIH). And we have also worked to direct mitochondrial disease research funding through the Peer Reviewed Medical Research Program of the Department of Defense.  Now we are closely tracking the development of a new federal biomedical research entity — the Advanced Research Projects Agency for Health or “ARPA-H”.

ARPA-H was proposed early in the Biden Administration and is in the process of being implemented.  The concept behind the new Agency is borrowed from the military which several decades ago created the Defense Advanced Research Projects Agency (DARPA).  The idea behind DARPA was to fund very ambitious and forward-leaning research projects and to aim at making transformational breakthroughs.  These lofty objectives were combined with a culture that acknowledged and accepted that many projects would fail and that funding would be turned off and redirected to other initiatives quickly where metrics were not met.

For the past two years, Congress and the Administration have been standing up ARPA-H based on the DARPA concept.  ARPA-H received initial funding of $1 billion last year (federal fiscal year 2022) and $1.5 billion in fiscal year 2023.  It is unclear how much funding will be requested and provided in the fiscal year 2024 budget process.  While this is a significant sum, it is much less than the current resources provided to the NIH ($47.5 billion).  However, there is the potential for ARPA-H to grow substantially in the coming years.

Within DoD, Independence is a hallmark of DARPA.  It falls under none of the service branches of Pentagon offices but instead is separate and reports directly to the Secretary of Defense.  ARPA-H is trying to reflect that model.  While the new Agency will receive administrative support from the NIH, its Director will report straight to the Secretary of HHS and the physical facilities of ARPA-H are to be established in at least three geographic locations (as required by Congress), none of which may be on the campus of the NIH.

ARPA-H is also mimicking DARPA’s administrative structure — a single director, appointed from outside the ranks of government, oversees a cadre of highly independent “program managers” each with broad latitude to establish priorities and fund (and de-fund) projects.  Dr. Renee Wegrzyn — an applied biologist who worked at DARPA at one time and most recently for a cutting-edge biologics firm, Ginko Bioworks — was appointed by President Biden as ARPA-H’s first Director.  She is now in the process of recruiting and installing program managers to handle portfolios of work.

It is expected that, like DARPA, ARPA-H will try to bring together teams of academic researchers and industry experts to address major challenges in health care research, such as the difficulty in bridging the gap between scientific insights and therapies, treatments and cures; and fundamental impediments to progress on cancer, diabetes and Alzheimer’s.  UMDF will be encouraging a focus on Mitochondrial function and genomics as part of the new Agency’s agenda.