For 80 years, federally supported research in the United States has fuelled scientific and technological advancements, including the Internet, space exploration and the development of semiconductors and biotechnologies. In biomedicine, innovations have led to vaccines and therapeutics, breakthroughs in cancer and cardiovascular care and gains in life expectancy and quality of life.
How data can help to guide NIH funding policy
Yet, the accelerating pace of innovation, alongside the complexity and scale of today’s societal challenges, requires a shift in how research investments are prioritized and governed. For example, artificial intelligence, big data and continuously collected health information are enabling more-precise prediction and prevention strategies. Such advances could reduce the burden of disease by detecting risks earlier, offering personalized interventions and improving treatment outcomes.
The roll-out of breakthroughs in the United States has conventionally followed the ‘linear’ framework laid out in science adviser Vannevar Bush’s 1945 report to then US president Harry S. Truman1. In Bush’s model, the federal government distributes money through its agencies; academics generate discoveries that companies translate into commercial applications; and intellectual-property rights boost private-sector investment and markets. This framework has catalysed decades of economic and technological gains, and has been copied worldwide. However, the private sector’s growing emphasis on reducing risk and maximizing returns is distorting research priorities in ways that Bush did not anticipate.
Companies are focusing on some diseases at the expense of others. For example, in 2022, more than half of US Food and Drug Administration (FDA) approvals of new drugs were for those targeting cancer and rare diseases2. Such areas generate high revenues because drug prices are high, people are willing to pay for the latest treatments and accelerated approval pathways shorten the time to market. For well-understood diseases such as these with clear therapeutic targets, the development risk for sponsors is low.
By contrast, new psychiatric therapies accounted for only 4.8% of FDA approvals from 2018 to 20223, despite more than one in five US adults living with mental illness in 2022 (see go.nature.com/4tygtd5). Developers might have been put off from entering this area because the understanding of complex disease mechanisms is limited and clinical trials often fail — which increases cost, uncertainty and risk.
A mainly market-driven approach also encourages companies to prolong monopolies and limit competition from generic versions of drugs. A product’s commercial life can be extended by varying its formulations, routes of administration and methods of use. Maintaining patent protections and market exclusivity hinders access by patients to affordable treatments, and contributed to an average price increase of 68% for the 12 best-selling drugs in the United States between 2012 and 20194.
Addressing the societal challenges of health security, chronic disease and population wellness requires a long-term, multidisciplinary approach, which is difficult to achieve through typical four-to-five-year government grants. Translation into the clinic and follow-up trials can take more than a decade.
Making progress in US biomedicine will require balancing market incentives and public needs.Credit: Wolfgang Hoffmann/Design Pics Editorial/Universal Images Group via Getty
Reforms are needed to ensure that the public is able to benefit appropriately from the research investments that are paid for by taxation. A coordinated strategy that balances market incentives with public needs is necessary to shape the next era of biomedical progress.
Here, we set out how the United States can adopt a mission-based strategic framework for biomedical innovation. It has three components: a clear, well-defined mission; a regulatory and incentive structure that aligns with the mission; and expanded partnerships across public and private sectors.
Define a clear mission
Historically, key innovation successes have shared common characteristics. These include clear, goal-oriented missions tied to pressing societal needs and a collaborative, cross-sectoral approach.
For example, the Apollo space programme landed astronauts on the Moon by mobilizing the public and private sectors — including the aerospace industry — in a government-led operational structure. Operation Warp Speed, a public–private partnership (PPP) spearheaded by the US health and defence departments, accelerated the development and distribution of COVID-19 vaccines. The Human Genome Project combined US agency leadership with international collaborations and with infrastructure and technologies from the private sector to sequence the first complete human genome.
To apply the same focusing power to modern biomedical research to meet societal health needs, the United States must now produce a strategic national vision. Other countries do so. For example, Singapore sets its national research strategy every five years (see go.nature.com/4bktucq). China outlines measurable goals for its science and technology investments up to 15 years in advance5. The United Kingdom defines priorities for its partnerships between the life-sciences industry and the National Health Service6. And the European Union’s EU Missions, which fall under the Horizon Europe research programme, set goals around topics that are pursued through public–private collaborations. These are established by advisory boards comprising experts and policymakers with input from industry and citizens (see go.nature.com/4bzrwyt).
NIH pivots away from agency-directed science
As a first step for the United States, we suggest following the recommendations of a 2024 report by the US National Academy of Medicine7, which called on the president and Congress to establish an advisory body to develop such a strategic national vision (M.H.L. was an editor and V.J.D. was on the committee tasked with producing this report).
The advisory body would comprise leading experts from a variety of disciplines and sectors, including academia, industry, economics, philanthropy and federal agencies. Its responsibilities would include issuing national research priorities, identifying opportunities for PPPs, advising federal agencies on funding allocations and developing mechanisms for public engagement in setting research agendas. We recommend a seven- to ten-year renewable charter and mandates to ensure continuity beyond federal elections and congressional budget cycles. The body should work with federal agencies and the National Economic Council and Domestic Policy Council to ensure the engagement of all relevant groups.
To align research priorities with national health needs, the advisory body should instigate data-driven assessments to quantify and assess disease burden, disease impact and unmet medical needs — as proposed in a 2025 report by the National Academies of Sciences, Engineering, and Medicine8. The body would produce an annual report for Congress and a decadal review to inform national priorities and strategy. Priority areas would include health burdens that have a high economic impact; unmet medical and societal needs; and persistent gaps in translation and access to the benefits of innovation when industry investment is low and deemed to be risky with low returns.
US companies are working on innovations such as injectable micro-robots that deliver drugs.Credit: Robyn Beck/AFP via Getty
This approach differs from the current system in two important ways. First, priorities would be set through an integrated, data-driven assessment across agencies rather than emerging mainly from institute-level agendas, congressional interest or market incentives. Second, they would emphasize coordinated, multi-agency and cross-sector strategies.
Such a mission-based approach is intended to augment, not diminish, curiosity-driven and investigator-led research and the normal operation of companies. By activating funding across government, academia, philanthropy and industry, this approach would help to de-risk work in areas in which market forces alone have not identified a sufficient return on investment. It would also offer extra opportunities for scientists to work together irrespective of their institution.
Align funding, regulations and incentives
Once an evidence-based strategic mission is set, a balanced regulatory and governance framework is essential — one that safeguards against risks while enabling innovation. Given rapid advancements in biotechnology, AI and digital health, regulations must be adaptive, ensuring safety, security and ethical integrity without stifling progress.
Governance must combine protections to minimize risk in areas such as ethics, data standards, biosecurity screening and transparency obligations, with adaptive oversight mechanisms that evolve as evidence emerges. It should direct public investment towards outcomes that advance health, economic resilience and national competitiveness.
Achieving this balance would require coordinated action by federal agencies, funders, regulators and health systems to align incentives with public value.
A standardized framework for assessing societal return on investment — similar to models used in infrastructure planning, defence innovation and energy systems — should be developed through an interagency process. This would be led by the White House Office of Science and Technology Policy in collaboration with the US National Institutes of Health, the National Science Foundation and the Office of Management and Budget, as well as relevant economic agencies.
What drugs are safe during pregnancy? There’s a shocking lack of data
For example, the Advanced Research Projects Agency–Energy uses techno-economic assessment to evaluate the technical feasibility, economic viability and potential benefits of a product. A similar approach in health could combine epidemiological modelling, health economics and implementation projections to estimate potential long-term population impacts under different adoption scenarios.
The federal government can leverage its own purchasing power to catalyse demand for priority health products, much as it did for the early semiconductor and aerospace industries. These might include next-generation vaccines and antimicrobial agents, advanced diagnostics and early-detection technologies, secure health-data infrastructure, resilient domestic manufacturing platforms for essential medicines and digital health tools. Creating viable markets for these innovations would ensure that they receive sustained investment.
