In a commentary published in Science, White House Office of Science and Technology Policy Director Eric S. Lander, Ph.D., National Institutes of Health Director Francis S. Collins, M.D., Ph.D., and other leaders describe a vision for a new science entity, the Advanced Research Projects Agency for Health (ARPA-H). Included in the President’s fiscal year 2022 budget with a requested funding level of $6.5 billion, ARPA-H would accelerate biomedical innovation and adoption of technologies and approaches to revolutionize healthcare and medicine.
Progress in medicine and health in recent decades has been driven by two powerful forces: pathbreaking fundamental research and a vibrant commercial biotechnology sector. Fundamental research is typically performed in university, nonprofit, and government labs, and is mostly funded by the federal government, largely through the National Institutes of Health (NIH). The commercial sector is largely focused on research, development, and marketing of specific products, to bring sophisticated therapies and devices to patients. In many cases, these two components are all that’s needed to drive progress towards clinical benefit – though subsequent regulatory approvals, reimbursement, and adoption in healthcare systems can also be optimized.
It’s becoming clear, though, that some of the most innovative project ideas, which could yield bold breakthroughs, don’t always fit existing support mechanisms: NIH support for science traditionally favors incremental, hypothesis-driven research, while business plans require an expected return on investment in a reasonable time frame that is sufficient to attract investors. As a result, some of the most significant ideas may never mature, representing substantial lost opportunity.
[Market failures] Bold ideas may not fit existing mechanisms because: (i) the risk is too high; (ii) the cost is too large; (iii) the time frame is too long; (iv) the focus is too applied for academia; (v) there is a need for complex coordination among multiple parties; (vi) the near-term market opportunity is too small to justify commercial investment, given the expected market size or challenges in adoption by the healthcare system; or (vii) the scope is so broad that no company can realize the full economic benefit, resulting in underinvestment relative to the potential impact. Evaluations by companies also may not consider the impact of projects on inequities that persist in our health ecosystem. In short, projects with a potentially transformative impact on the ecosystem may not yet be economically compelling or sufficiently feasible for a company to move forward. At the same time, we lack public mechanisms to propel these public goods at rapid speed.
Many such bold ideas involve creating platforms, capabilities, and resources that could be applicable across many diseases. Whereas most NIH proposals are ‘curiosity-driven’, these ideas are largely ‘use-driven’ research — that is, research directed at solving a practical problem.
The mission of ARPA-H will be to make pivotal investments in break-through technologies and broadly applicable platforms, capabilities, resources, and solutions that have the potential to transform important areas of medicine and health for the benefit of all patients and that cannot readily be accomplished through traditional research or commercial activity.
ARPA-H will be a distinct division within the NIH, with a unique culture and organization.
o Creating ARPA-H within the NIH, rather than as a stand-alone entity, makes sense because the ARPA-H mission falls squarely within the overall NIH mission (“to seek fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability”) and because it is important that ARPA-H be closely coupled to the vast range of biomedical and health knowledge, expertise, and activities within NIH.
o ARPA-H will focus on time-limited projects with goals, benchmarks, and accountability.
o ARPA-H Director will be selected based on extraordinary technical and leadership skills, such as proven innovation and partnership-building track-records, and will be responsible for setting the culture, including injecting the principles of risk-tolerance, urgency, equity, and innovation into every decision.
o A diverse cohort of ARPA-H program managers will be recruited from industry, academia, or other sectors based on demonstrated scientific vision, judgment and management skills, and they will have broad autonomy to drive transformational change.
o ARPA-H will establish partnerships and collaborations with key stakeholders from industry, government, non-profits, and academia to leverage critical skills and expertise in tackling the systemic challenges that the current systems were not designed to address.
Press release: https://www.whitehouse.gov/ostp/news-updates/2021/06/22/media-availability-lander-collins-set-forth-a-vision-for-arpa-h/
Concept paper: https://www.whitehouse.gov/wp-content/uploads/2021/06/ARPA-H-Concept-Paper.pdf
Fact sheet: https://www.whitehouse.gov/wp-content/uploads/2021/06/ARPA-H-Fact-Sheet.pdf
"ARPA-H: Accelerating biomedical breakthroughs," Science, June 22, 2021
Francis S. Collins, Tara A. Schwetz, Lawrence A. Tabak, Eric S. Lander