After the success of bringing COVID-19 vaccines to the masses in less than a year, U.S. officials are wondering how those lessons can be applied to spur breakthroughs across medicine.
President Biden is proposing a new research agency to sit within the National Institutes of Health, called the Advanced Research Projects Agency for Health (ARPA-H). It would be modeled after the Defense Advanced Research Projects Agency, or DARPA, which has led breakthroughs for the Department of Defense for more than 60 years.
The NIH requested an initial $6.5 billion in funding “to develop breakthroughs—to prevent, detect, and treat diseases like Alzheimer’s, diabetes, and cancer,” according to a paper from several NIH and White House officials published Tuesday in the journal Science.
“The United States stands at a moment of unprecedented scientific promise and is challenged to ask: What more can we do to accelerate the pace of breakthroughs to transform medicine and health?” wrote NIH Director Francis Collins and two of his deputies, Tara Schwetz and Lawrence Tabak, along with Eric Lander, director of the White House Office of Science and Technology Policy.
Like DARPA, the new health project would work to advance fundamental research at university, nonprofit and government labs, which is typically funded by the federal government already.
DARPA has shown the model works: The agency contributed to the development of the internet, GPS and self-driving cars to name a few. One other breakthrough DARPA had a hand in? Messenger RNA vaccines, just like the ones being delivered to arms around the world right now to combat the COVID-19 pandemic.
The officials said every new medicine approved by the FDA can be traced back to NIH-supported discoveries, at least in part. The pharmaceutical industry focuses on R&D and marketing of specific products to bring new therapies and devices to patients. Biotechs do this using “abundant capital,” so long as the investment can be recouped through profits and protection of intellectual property. This two-tiered system has spawned a pipeline about 8,000-medicines strong, including 1,300 for cancer.
But while this model has been effective, some innovative medicines simply never make it off the drawing board because they don’t fit into existing mechanisms, the NIH and White House officials wrote.
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“NIH support for science traditionally favors incremental, hypothesis-driven research, whereas business plans require an expected return on investment in a reasonable time frame that is sufficient to attract investors,” the paper said.
ARPA-H will therefore be an incubator for “bold ideas,” that are riskier, more costly and time-consuming, involve complex coordination across stakeholders and may not immediately bring in enough cash for a company because of a small market size or difficulty implementing the new treatment in the health system.
“ARPA-H should expect that a sizable fraction of its efforts will fail; if not, the organization is being too risk-averse,” the officials wrote. “The best approach is to fail early in the process, by addressing key risks upfront.”
NIH took inspiration from DARPA to respond quickly to the COVID-19 pandemic with a new program to accelerate therapeutics and vaccines. The same was done for diagnostics.
“Although these programs have been successful, they required bespoke solutions and herculean efforts to get them off the ground,” the paper said. “Because NIH lacks a regular framework for such projects, many bold ideas are hard to realize. That’s where ARPA-H can help.”
ARPA-H would have a broad mission that goes beyond just developing therapeutics. The officials said that equity in race, ethnicity, gender, sexual orientation, disability and income should be front and center to ensure positive health outcomes for all patients.
The officials proposed a director who would serve a single term of five years with extension possible, but rare, to keep the new organization brimming with fresh ideas.