As plan for new U.S. health agency to speed treatments takes shape, doubts remain

By Jocelyn Kaiser May. 29, 2021 , 11:00 AM

Those closely following President Joe Biden's plan to create a huge agency to fund cutting-edge, transformative health projects welcomed the release this week of new details about the ambitious proposal. But for some research advocates, worries remain that the new agency won't be significantly different from the rest of the National Institutes of Health (NIH), where it would be housed.

The proposed Advanced Research Projects Agency for Health (ARPA-H) "will need to be audacious, nimble, and have unique authorities," says Ellen Sigal, chair and founder of Friends of Cancer Research. "It's an incredible opportunity, but at the moment there are many unknowns that will need to be discussed and debated in the near future."

First proposed by Biden early this year, ARPA-H would be modeled after the similarly named Defense Advanced Research Projects Agency (DARPA), which has a reputation for accelerating the development of breakthrough technologies for the military. DARPA's funding approach depends less on traditional peer review of ideas, and more on hard-charging program managers empowered to award contracts that can be abruptly cancelled if researchers don't meet desired milestones. DARPA has been lauded for, among other things, helping develop the Internet and radar-evading stealth technologies. Biden and others believe a similar model of placing informed bets on high-risk but potentially high-payoff ideas could also produce biomedical advances.

Some proponents of ARPA-H have argued it should be a stand-alone agency within the Department of Health and Human Services (HHS) because it would be so different from NIH, which funds basic research with long-running grants evaluated by peer reviewers. Last month, however, Biden released a budget outline that proposed placing ARPA-H – which he wants to launch with a budget of $6.5 billion – squarely within the NIH . That plan raised doubts that, if Congress approved ARPA-H, it would be able operate as originally envisioned.

Yesterday, Biden's full budget request to Congress offered a bit more insight into the administration's vision for ARPA-H. The agency "will have a distinctive culture and organizational structure," according to budget documents , "and will complement NIH's existing research portfolio, providing an agile and flexible arm to advance biomedical science quickly and robustly." The budget also describes an external advisory board that will help ARPA-H coordinate with other agencies and generate ideas.

Those things sound like DARPA, says Michael Stebbins, a consultant and former White House science office official. But he's troubled by a statement that ARPA-H will use "traditional and nontraditional mechanisms." "That sounds like grants and not milestone-driven contracts," Stebbins says.

NIH director Francis Collins added more details earlier this week at hearings in the Senate and House of Representatives on NIH's 2022 budget. Lawmakers were generally supportive of ARPA-H but had questions, including about who would run the agency. Collins told a Senate panel that he expects it to be led by "a visionary person" appointed for a 5-year term that could be renewed only once. Like at DARPA, projects would be pitched to the director by a hundred program managers, who would then find partners in industry and academia to pursue them.

Collins said NIH has shown it is capable of adopting "a DARPA-like attitude" by quickly disbursing billions of dollars to develop diagnostics, treatments, and vaccines to battle the COVID-19 pandemic. As an example, he pointed to the Operation Warp Speed effort that produced two cutting-edge mRNA vaccines for COVID-19.

As for concerns that $6.5 billion would be too much for the agency to handle in its first year, Collins said the spending would be spread over 3 years. And there's no need to worry that ARPAH-H would mean smaller funding increases for NIH's 27 institutes and centers, he said. "It is going to be a synergistic relationship," Collins told the Senate panel.

To some onlookers, however, giving NIH's institutes and other agencies a role in shaping ARPA-H's activities sounds less like a dramatic break from traditional approaches, and more like a larger version of the Common Fund, a central NIH pot of money that critics complain has failed to fund enough out-of-the-box research.

Another concern is that instead of allowing ARPA-H to decide which diseases it will target, Collins will yield to demands from patient advocacy groups that the agency include funding for their priorities. Collins seemed to suggest at the House hearing last week that ARPA-H won't ignore any disease. "The intention is for this model to be applied to all of the diseases that are ready for this scientifically," he said.

Stebbins, for one, hasn't been reassured by such comments. "I think we need a lot more information here before we should be comfortable with [ARPA-H] being under the NIH," he says.

Yet another unknown is what happens if ARPA-H is created as a standalone NIH institute, as it appears to be in the president's budget. That would bring the total number of NIH institutes and centers to 28, one more than allowed under current law.

One option may be to combine ARPA-H with NIH's National Center for Advancing Translational Sciences (NCATS), which has a similar mission of funding translational science that moves basic discoveries towards treatments. Collins told the House panel that NIH needs to consult with "stakeholders" about whether some of NCATS' components could be folded into ARPA-H.

How Congress will handle the many questions about ARPA-H could become clearer in early June, when a House committee that oversees NIH policies intends to release a draft bill to create the agency.