Congress

What Will ARPA-H Look Like?

by Matt Gontarchick

There’s a new federal agency in town.  The Fiscal Year (FY) 2022 omnibus appropriations bill officially created the Advanced Research Projects Agency for Health (ARPA-H), which the Biden administration first proposed last year to drive “transformational innovation” in health research.  However, the omnibus bill is scant on details, and lawmakers have much to decide about the structure of the new agency.

What’s in the spending bill? Beyond appropriating $1 billion in funds to the new agency through September 30, 2024, the FY 2022 omnibus bill allows the ARPA-H director to use those funds to make awards in the form of “grants, contracts, cooperative agreements, and cash prizes.”  Notably, the bill gave the Department of Health and Human Services (HHS) 30 days to decide whether the new agency would be independent or part of an existing institution, like the National Institutes of Health (NIH).

The issue of whether to make ARPA-H an independent agency within HHS or house the new agency within the NIH has been the subject of debate for months.  Public health experts who testified before a congressional panel on February 8 unanimously agreed that ARPA-H would need to foster an independent culture to be successful in delivering biomedical breakthroughs, and most witnesses and lawmakers felt that housing the ARPA-H within NIH would make it impossible to cultivate its own identity and operating structure. 

Since then, more details about ARPA-H have come into focus.  In a March 31 congressional hearing, HHS Secretary Xavier Becerra announced that ARPA-H would be placed within NIH.  However, Becerra did offer key details that suggests the administration wants to help foster an independent culture within the new agency.  For example, he said the ARPA-H director will be under the supervision of the HHS secretary, not the NIH director.  Additionally, Becerra clarified that ARPA-H would not be “physically housed” within NIH.  According to Becerra, the reason for the new agency’s placement within NIH is to allow ARPA-H to focus on research from the get-go while NIH handles all the administrative work like human resources and legal functions.  

However, the makeup and structure of ARPA-H won’t be up to the Biden administration.  That’s the job of Congress where a trio of authorizing bills are under consideration that flesh out the details of ARPA-H. The bills – the Cures 2.0 Act plus the House and Senate versions of the ARPA-H Act – propose similar requirements on the new agency, such as:

  • Presidential appointment of the APRA-H director for one five-year term, with the option to extend for one additional term.
  • Establish goals for delivering biomedical breakthroughs by prioritizing high-risk, high-reward innovations and identifying potential areas of health research advancement that industry stakeholders aren’t currently addressing due to technical or financial reasons.
  • Collaboration with other HHS entities like the Food and Drug Administration (FDA).

There’s one key area where the bills differ – the Cures 2.0 Act calls for APRA-H to be a part of NIH, while both versions of the ARPA-H Act say the new agency should be independent within HHS.  The House Energy and Commerce Subcommittee on Health held a legislative hearing on March 17 to review both the Cures 2.0 Act and the ARPA-H Act, and currently, House leadership supports the ARPA-H Act, which increases the odds the new agency will ultimately be independent from the NIH. 

As lawmakers continue their work, stakeholders outside of Washington are focusing on a different question: the specific location of ARPA-H.  While Becerra told lawmakers back in March that the new agency wouldn’t physically be a part of the NIH campus in Bethesda, Maryland – neither the FY 2023 omnibus bill nor the three authorizing bills say anything about where ARPA-H should be headquartered.  Currently, cities in California, Massachusetts, North Carolina, and Texas are lobbying to become the location of the new biomedical research agency.

Congress is in recess until the week of April 25, and neither chamber has indicated when it will markup and vote on their respective authorizing bills on APRA-H.  Until the finish line is in sight, conversations over the makeup of ARPA-H – and whether it should be a part of the NIH – are likely to continue. 

Congress

Why ARPA-H Needs to Be Independent from NIH to Be Successful

by Matt Gontarchick

The nation needs more breakthrough medical treatments.  While the National Institutes of Health (NIH) is the government’s leading biomedical research agency, unfortunately, it takes a long time to turn NIH-supported research into cures.  To bridge this gap between research and innovation, Democrats are proposing an Advanced Research Projects Agency for Health (ARPA-H) with the hope that it will deliver breakthrough medical treatments quicker. 

For ARPA-H to be successful, its placement within the structure of the federal government may be key.  In a February 8 hearing by the Health Subcommittee of the House Energy and Commerce Committee, witnesses testified to make the case that for ARPA-H to be effective, it cannot be housed within the NIH – instead, ARPA-H must be an independent agency within outside the purview of the Department of Health and Human Services (HHS).

Democrats modeled ARPA-H after the Defense Advanced Research Projects Agency (DARPA), an independent research and development agency within the Department of Defense, that’s charged with the development of emergency technologies for use by the military.  According to former Assistant Secretary for Health Brett Giroir – a member of the witness panel and a former Director of Defense Science Office at DARPA – the independent status of DARPA was crucial in its ability to bring about innovations like cellular technology and the Global Positioning System (GPS).

During the hearing, members of the witness panel discussed several reasons why ARPA-H must be independent of NIH to be successful.

  • Culture.  Like DAPRA, ARPA-H would need to have a distinct culture, vision, and approach to problem solving to deliver breakthroughs.  This is only possible if ARPA-H is separate  from NIH – otherwise, the new agency will be unable to develop a culture distinct from NIH. 
  • Operations. Unlike NIH researchers, project managers at ARPA-H would follow timelines with specific deliverables that witnesses at the hearing suggested being publicly posted.  DARPA follows a similar model
  • Interaction with private sector.  Project managers at ARPA-H would be required to engage with the private sector to ensure that there is no overlap between research being conducted within the new agency and drug developers.  Witnesses at the hearing additionally suggested that ARPA-H hold listening sessions and/or engage with specific communities to ensure health equity is a focus at the new agency.

What comes next? House and Senate appropriators proposed funding for ARPA-H in Fiscal Year (2022) appropriations.  However, the new agency’s future is increasingly in doubt as lawmakers move to pass another continuing resolution to keep the government funded at FY 2021 levels through March 11 – which mean no funding for new initiatives like ARPA-H.  And it’s not just appropriations – for ARPA-H to become a reality, Congress needs to approve authorizing legislation, and there’s currently no timetable for when either of the bills that authorize ARPA-H will move forward. 

Administration

What Happened to the Cancer Moonshot?

by Matt Gontarchick

In his final State of the Union address in January 2016, then-President Barack Obama asked then-Vice President Joe Biden to lead the Cancer Moonshot initiative, a new national effort to accelerate research in cancer treatment.  Over the past five years, however, attention on the Cancer Moonshot has seemingly waned to the point that it barely pops up in the national discourse on health policy.  Is the Cancer Moonshot still around, and if so, what has it even accomplished?

Background

The Cancer Moonshot is far from being the federal government’s first push to deliver new cancer treatments.  The National Cancer Institute (NCI) was first established in 1937 to support cancer research.  Incorporated within the National Institutes of Health (NIH) in 1944, NCI remains the oldest center and has the largest budget among the other 27 centers and institutes of NIH. Decades later, President Richard Nixon signed the National Cancer Act of 1971 into law, which provided NCI with more autonomy within NIH and authorized $1.6 billion in funding for NCI over three years.  Additionally, the Orphan Drug Act of 1983 spurred many pharmaceutical companies to invest in drugs to treat rare diseases like cancer. 

Beyond NCI,  the federal government has done little to invest large sums of money focusing on research into the prevention and treatment of cancer, which  has limited the progress in the war against cancer.  The overall death rate from cancer has only declined 5% since 1950, and new cancer drugs produced between 2003 and 2013 has only boosted overall survival by 3.4 months.  Given the technical and scientific difficultly of developing cancer treatments, a more concerted effort rather than a piecemeal approach may be necessary to deliver progress. 

How the Moonshot Got Started

Shaped by his oldest son’s death from brain cancer in 2015, Biden declared after the 2016 State of the Union that the goal of the Cancer Moonshot was to double the rate of progress towards a cure for cancer. Shortly thereafter, Biden convened a Cancer Moonshot Task Force that established a BlueRibbon Panel charged with outlining research priorities to help the new initiative meet its goals.  Comprised of clinicians, scientists, cancer patients, and advocates, the panel issued a report in October 2016 that established seven working groups to identify research opportunities and offer recommendations on how the Cancer Moonshot can support those opportunities.  Since then, Congress authorized the Cancer Moonshot through enactment of the 21st Century Cures Act in December 2016, which set up the initiative within NCI and provided $1.8 billion funding over seven years.  For FY 2021, the Cancer Moonshot was appropriated $195 million.

Progress So Far

The Cancer Moonshot initiative uses its funding to invest in research that aligns with the initiative’s key priorities, such as generation of human tumor atlases, expanded use of early detection strategies, and establishing a network for direct patient engagement.  According to a midpoint progress update issued in January 2021, the initiative has so far funded over 240 research projects and more than 70 cancer science programs.  The Cancer Moonshot cites over a dozen projects on its website that have yielded progress, including:

  • The Immuno-Oncology Translational Network, whose research has led to insights into ovarian cancers responses to immunotherapy and the use of immunotherapy in certain types of head and neck cancers.
  • The My Pediatric and Adult Rare Tumor Network, which has developed a pipeline for biospecimen collection/analysis rare cancers and established several new specialized rare tumor clinics around the country.
  • The Fusion Oncoproteins in Childhood Cancers Consortium, whose researchers have developed several novel cancer models to study rare cancers and have made significant advances in understanding how each fusion affects the protein’s function and localization in the cell.

Room for Improvement

However, some scientists say Cancer Moonshot has the potential to deliver on greater progress.  To achieve this, a group of scientists proposed early this year in Lancent Oncology, a “Cancer Moonshot 2.0” that can meaningfully improve outcomes for cancer patients with a “scaled up, redoubled, and accelerated” approach.  Among the research areas suggested by the group include:

  • New database formats to capture biological data.
  • Non-invasive liquid biopsy approaches that can be used to screen high-risk individuals to identify precancers and early cancers.
  • Machine learning innovations that link the molecular structure of cancer pathways to more specific and effective drugs.
  • New drug delivery systems that target specific cancer vulnerabilities, such as bispecific antibodies and nanotechnologies.

While the Cancer Moonshot is still alive and kicking, the initiative may not be subject to as much attention when it was first conceived in 2016, likely due to less-than-stellar advancements and the domination of other issues in the public health discourse.  As difficult as cancer research may be, a redoubling of efforts and additional funding could pave the way for new treatments that exemplify the term “moonshot.”

Administration

ARPA-H, the Proposed Agency to Transform Biomedical Research

by Matt Gontarchick

The internet.  GPS.  The computer mouse.  These are some of the technological innovations made possible by the Defense Advanced Research Projects Agency (DARPA), a Defense Department agency tasked with advancing research and development of science and technology programs.  Now, the Biden administration wants to replicate DARPA’s success in biomedical research with a newly proposed entity called Advanced Research Projects Agency for Health (ARPA-H). 

What Is ARPA-H?

The Biden administration’s Fiscal Year (FY) 2022 budget request provided a $6.5 billion investment for APRA-H, which would be directed to drive “transformational innovation” in health research and speed application and implementation of health breakthroughs.  The initial focus of ARPA-H would be cancer, disabilities, and Alzheimer’s disease.  According to a concept paper provided by the White House, examples of projects that could be pursued by ARPA-H include:

  • mRNA vaccines to prevent most cancers.
  • Molecular “zip codes” that target drugs only to specific tissues and cell types, to eliminate serious side effects.
  • Highly accurate, inexpensive, wearable monitors for blood pressure and blood sugar that provide real-time data to patients and providers.
  • Holistic systems that eliminate racial disparities in maternal morbidity and mortality rates and premature births.
  • A process to design, test, and approve a vaccine against any newly emerging human virus in 100 days.

The National Institute of Health (NIH) further explains that ARPA-H will predominantly focus on “time-limited” projects with goals, metrics, and accountability.  It also states that the director of ARPA-H will be selected based on having a “proven innovation and partnership-building track-record,” and ARPA-H program managers will be recruited from industry, academia, or other sectors based on “scientific vision, judgement, and management skills.”

ARPA-H is also included in the Cures 2.0 discussion draft that was released by Reps. Diana DeGette (D-CO) and Fred Upton (R-CO).  However, the discussion draft is still a work in progress, and it contains far fewer details on the proposed agency than those provided by the Biden administration. 

How Would ARPA-H Advance Biomedical Research?

Ideally, ARPA-H would need many of the same authorities used by DARPA to accomplish its mission to delivery breakthroughs in biomedical research.  Key authorities would include:

  • The ability to hire individuals rapidly based on “a unique skill set” outside the typical civil services hiring system and pay those individuals a competitive wage, as well as the ability to recruit experienced program managers from the private sector. 
  • Broad, flexible funding authority that make it possible to mix and match the best ideas with minimal bureaucracy that allow for projects that can be funded for multiple years or don’t necessarily fit into one-year intervals.
  • Exemptions from the traditional review process for biomedical research, which can take up to 18 months or longer, to get from an idea to a scientific review before the actual work begins.

According to a July 26 Federal News Network interview with NIH Director Francis Collins, an ARPA-H with “some new authorities” could make a difference for experimental drugs that show a lot promise during clinical trials but fail to attract investment from pharmaceutical companies due to an experimental products being perceived as too risky or having a limited market share.  In these cases, Collins explained ARPA-H could chose to invest in certain experimental drugs and hand them over to the private sector when necessary.

The Structure of APRA-H

The Biden administration has stipulated that ARPA-H would be a “distinct division” within NIH, with a “unique culture and organization.”  However, some medical experts have argued ARPA-H should be a stand-alone entity within the Department of Health and Human Services (HHS), including a former chair from DARPA’s advisory committee, who suggested NIH’s approach to research and innovation is too time-consuming and bureaucratic.  In response, Collins told The Washington Post in mid-June that putting ARPA-H outside of NIH would be a big mistake because it would lead to “administrative duplications” and preclude the chance for ARPA-H program managers to develop synergy with their colleagues at NIH.  While the Cures 2.0 discussion draft has yet to define ARPA-H’s structure and home, Collins has said that DeGette and Upton are leaning towards having the new agency withing NIH.

Collins also threw cold water on the notion that ARPA-H could create internal jealously within NIH in his Federal News Network interview by countering that many NIH employees are excited about the prospect of a new research agency and that “unfriendly competitiveness” exists in other parts of the government.  It’s worth noting that the administration’s FY 2022 budget request includes a major increase in NIH funding rather than diverting away funds to create a new agency. 

Next Steps

APRA-H only becomes a reality when FY 2022 appropriations bills are signed into law, and the FY 2022 appropriations process has gotten off to a slow start.  The House passed an appropriations minibus for FY 2022 on July 31 that includes $3 billion for ARPA-H, while the Senate only began reviewing its FY 2022 spending proposals on August 2. , Notably, the proposals to create a new biomedical research agency been favorably viewed by members of both parties, meaning ARPA-H is unlikely to be cut from the legislation. However, given lawmakers’ slow progress on appropriations and long list of other items that require their attention in the next 60 days, all FY 2022 spending measures are unlikely to be finalized by the end of the FY 2021 on September 31.  This means NIH may not be able to take the first steps in building up ARPA-H until Congress finalizes the FY 2022 appropriations bills later in the fall or winter.

Once FY 2022 funding is squared away, Collins hopes to hit the ground running.  In his Federal News Network interview, the NIH director said he hopes to recruit 50-100 program managers for the agency’s first year, with results on research projects expected in the following one to two years as ARPA-H continues to “staff up and ramp up.”  Collins also projected to have 50-60 projects going at ARPA-H by the end of FY 2022, assuming all FY 2022 spending bills are enacted by December 2021.