Advanced Research Projects Agency for Health (ARPA-H): Considerations for Congress

Advanced Research Projects Agency for Health (ARPA-H): Considerations for Congress

INSIGHTi

Advanced Research Projects Agency for
Health (ARPA-H): Considerations for
Congress
May 18, 2021
On April 9, 2021, the Office of Budget and Management (OMB) submitted President Biden’s FY2022
discretionary budget request to Congress. This “skinny” budget request proposed the creation of an
Advanced Research Projects Agency for Health (ARPA-H) within the National Institutes of Health (NIH),
an agency in the Department of Health and Human Services (HHS). The budget request included $6.5
billion for ARPA-H to “drive transformational innovation in health research and speed application and
implementation of health breakthroughs.” Its initial focus would include “cancer and other diseases such
as diabetes and Alzheimer’s.”
While the current publicly available information on the proposal lacks specifics, ARPA-H would likely
follow the model of other “ARPAs,” especially the Defense Advanced Research Projects Agency
(DARPA) and the Advanced Research Projects Agency—Energy (ARPA-E). Stakeholders identify a
number of “DARPA model” characteristics for ARPA-H, including a flat and nimble organizational
structure, tenure-limited program managers with a high degree of autonomy to select and fund projects,
and a milestone-based contract approach. In contrast, NIH relies predominantly, with some exceptions
discussed below, on the scientific peer review process to award most of its funding. Some evidence
suggests that this investigator-driven and consensus-based process is less likely to fund high-risk, high-
reward projects.
Novel approaches outside of the traditional peer-review process have been successful at funding medical
research and development (R&D). Notably, DARPA invested in Moderna’s mRNA vaccine technology—
reportedly at a time when it was viewed with skepticism. The Biomedical Advanced Research and
Development Authority (BARDA) has supported medical countermeasure R&D through flexible,
innovation-focused R&D awards—including for Coronavirus Disease 2019 (COVID-19) products.
NIH currently has programs with some DARPA model characteristics—the Common Fund for high-risk,
short-term and milestone-driven innovative projects and the National Center for Advancing Translational
Sciences (NCATS), which focuses on innovation in medical product development. Additionally, NIH has
major drug R&D efforts involving private partners and other agencies for the diseases mentioned in the
President’s request—Alzheimer’s disease, cancer, and diabetes—notably through the Alzheimer’s disease
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research portfolio, the Beau Biden Cancer Moonshot Initiative, and the Accelerating Medicines
Partnership (AMP). During congressional hearings, some Members have questioned if ARPA-H would
replicate existing NIH efforts.
ARPA-H could potentially expand NIH’s role helping researchers and companies overcome barriers (e.g.,
lack of capital) to the commercialization of biomedical discoveries. Figure 1 depicts a generalized
process for funding and commercializing pharmaceutical drugs. Some recent economics research
indicates that pharmaceutical firms may underinvest in novel innovation, which may justify establishing
ARPA-H. However, the Congressional Budget Office (CBO) has noted a risk of federal funding
displacing private sector investments in pharmaceutical R&D.
Figure 1. Common Process and Funding Sources for Transitioning a Discovery into a
Pharmaceutical Drug

Source: Adapted by CRS from Jeffrey Cummings, Carl Reiber, and Parvesh Kumar, “The Price of Progress: Funding and
Financing Alzheimer’s Disease Drug Development,” Alzheimer’s & Dementia: Translational Research & Clinical Interventions,
vol. 4 (2018), p. 3337.
Notes: Figure represents a generalized process that may not apply to every pharmaceutical drug. Abbreviations:
SBIR/STTR = Small Business Innovation Research/Small Business Technology Transfer programs; AMC= Academic Medical
Center; Biotech Start-Up = small biotechnology companies; Pharma = large pharmaceutical companies and large- and
medium-sized biotechnology companies.
DARPA’s primary mission is national security, and its primary customer is the Department of Defense. As
such, the agency does not have to consider commercialization—though DARPA investments do
sometimes lead to commercial technologies (e.g., autonomous vehicles). ARPA-H would fund biomedical
R&D and technologies that would transition primarily to the largely private health care sector, which is
influenced by public and private health insurance and coverage, as well as patient and provider
acceptance and uptake. Many ARPA-H-supported technologies would require preclinical and clinical
testing to support Food and Drug Administration (FDA) approval.
Clinical trials (i.e., testing in humans) follow a process designed to limit exposure to potentially unsafe or
ineffective products and can take over 10 years to complete. Some studies suggest that issues with clinical
trials, such as trial design or barriers to participation, also pose key challenges for developing new drugs
for Alzheimer’s disease and cancers. Additionally, the acting FDA Commissioner and other stakeholders

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have called for greater federal clinical trial coordination and infrastructure based on experience with
COVID-19 R&D. ARPA-H could potentially aid in developing innovative technologies used in clinical
trials (e.g., devices to measure patient outcomes) or by expanding NIH’s role in supporting clinical trials.
However, clinical trial infrastructure programs may be more akin to NIH’s existing clinical trial networks
or BARDA’s clinical studies network.
As Congress examines the ARPA-H proposal and the role of the federal government in the
commercialization of biomedical products, it may consider the following options:
 Establishing a new ARPA-H entity either within NIH or within HHS, in addition to
existing programs.
 Consolidating existing related programs (e.g., the Common Fund) into a new ARPA-H.
 Expanding upon existing efforts instead of establishing ARPA-H.
 Taking no action.
If Congress decides to create ARPA-H, it may consider a number of questions:
 Are some of the goals of ARPA-H more appropriately accomplished through other policy
options (e.g., tax incentives to encourage private investment)?
 How should ARPA-H determine its priorities? Are cancer, diabetes, and Alzheimer’s
disease the appropriate initial focus areas for ARPA-H, especially given existing NIH
efforts?
 How should ARPA-H consider implementation and access to medical products and
drugs? For example, cost, manufacturing capacity, provider training needs, and complex
clinical administration have affected access to cancer CAR-T therapy.
 Is the proposed $6.5 billion budget appropriate? That amount would place ARPA-H
among the top-funded NIH Institutes and Centers (IC) and potentially affect the agency’s
longstanding emphasis on basic research.
 What are the appropriate metrics for measuring the success of ARPA-H?
Establishing a new entity may overcome existing institutional or cultural barriers to innovative
approaches to biomedical R&D. At the same time, any new entity may need to coordinate with existing
programs to avoid duplication. Congress may also consider whether to incorporate or align ARPA-H with
other legislative proposals, such as the NIH innovation provisions in H.R. 3.

Author Information

Kavya Sekar
Marcy E. Gallo
Analyst in Health Policy
Analyst in Science and Technology Policy

Disclaimer
This document was prepared by the Congressional Research Service (CRS). CRS serves as nonpartisan shared staff
to congressional committees and Members of Congress. It operates solely at the behest of and under the direction of

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