Guidelines for Grant Proposals on Evidence-Based Biopharma Policy
The Information Technology and Innovation Foundation (ITIF), based in Washington, DC, will offer grants for new research that helps to inform the relationship between the expectation of a financial reward for an approved new drug or new indication in the United States and the (1) investment in research and development for new and existing drugs, and/or (2) the approval of new drugs and new indications.
The purpose of this funding opportunity is to address the lack of sufficient credible and recent evidence to inform public policy changes impacting biopharmaceutical drug development. Funded research will support a better understanding of how the expectation of a financial return from investment in clinical biopharmaceutical development affects the level of investment in all stages of clinical development and for different types of potential drugs, such as for rare diseases, oncology, or vaccines.
ITIF awarded three grants of $50,000 each for research to be completed within 18 months, with a preference for the research to take one year in duration. Research will focus on the U.S. market or analogs of sufficient size that could inform how changes in U.S. policy will affect drug development globally. The expected output of each grant is a paper that can be submitted to a special issue of Health Affairs Scholar on this topic, following their guidelines and subject to their review process.
The preferred topics for these proposals were as follows:
- Impact on post-market development: Policy that affects the financial return from a drug or vaccine later in the lifecycle, such as the Medicare Drug Price Negotiation Program in the Inflation Reduction Act, is expected to affect investment in and conduct of post-market study. Much of the existing evidence examines the impact of market size on new drug development, neglecting post-market development. Research is of interest that improves understanding of incentives for investment in and/or conduct of post-market development in studies such as those for new indications, new formulations, or for special populations such as children or individuals with co-morbidities.
- Updated R&D cost and complexity: Current estimates of the relationship between expected financial returns and R&D investment rely on older estimates of the cost and time needed to develop a new drug or indication. Drug development has evolved significantly over time, with more specialized drugs and complex development protocols, affecting both development risks and costs. Peak revenues for approved drugs and prices have also evolved. Updated data and modeling that better reflect the current complexities of development of new indications and new drugs would provide more relevant insights. This could include how changes in trial enrollment rates, population sizes for drugs studied, study complexity, diversity requirements, or innovative study designs such as adaptive or remote-enabled trials may affect the relationship between investment in drug development and the expected financial return.
- Disparate impact on certain disease states: Drug development has shifted significantly, with oncology, targeted therapeutics, biologically based therapies and other new modalities now comprising a much larger share of R&D investment compared with two decades ago. Existing studies have not evaluated the effect of market size or financial reward on investment in clinical study for emerging modalities such as cell and gene therapies and RNA vaccines. Research of interest should examine the investment risks and opportunities across different therapeutic areas, particularly treatments for rare diseases or diseases for which there is no standard of care previously and should consider how the magnitude of the relationship may differ in different disease states.
- Variable risk tolerance in capital investment: The organizations involved in drug development are distinct along the drug development lifecycle and there is little understanding of how decisions are made to invest or not invest at different stages and who is making the decisions and their risk tolerance. Such evidence should better reflect capital mobility and diverse investment strategies throughout the drug development lifecycle. More comprehensive data, including insights from public and industry investments—such as quantitative firm data and qualitative research to better understand the risk profiles of different stakeholders, combined with modeling methodologies that account for heterogeneity across firms and investors—would help to capture variations in risk tolerance, investment behavior, and decision-making, and can provide a more realistic representation of industry dynamics.
- Effect on competitor drugs: Price setting or other polices may reduce the revenue of competitor drugs in the same therapeutic area, leading to lower prices, lower expectation of reward, and a change in clinical development within that therapeutic area. This effect, akin to the dynamics of generic drug entry, remains uncertain and requires further investigation.
- Competitive market for generics and biosimilars: Updated evidence is needed to assess the impact of government price setting of branded drugs on incentives for the entry and pricing of generics and biosimilars.
Call for Proposals
Proposals were accepted starting November 1, 2024. Responses to the call for proposals were due to ITIF by December 15, 2024, and awardees were announced at the end of January 2025. The applications were required to include:
- Research Team: Full name and affiliation of the lead researcher who will receive the funds and the research team and their affiliated institutions. Brief bios of lead researcher and research teams, not to exceed 150 words each (can include links to CVs).
- Abstract: Brief overview (200 words or less) of the proposed study including rationale, objective, methods, implications for research or policy.
- Approach to Study: Description of the research, the methods, and timelines not to exceed 800 words, may include up to three charts or tables not in the word count. Grantees will be required to deliver an interim report on progress at a midpoint in the project and can recommend a time for the interim report then provide a target date for the final report to be submitted to Heath Affairs Scholar.
- Statement of Willingness to Comply with Health Affairs Scholar Format: Include a statement that the lead researcher and team are willing to agree to the submission guidelines in Health Affairs Scholar. If the team prefers another outlet, they can describe that outlet in lieu of this statement and explain their rationale for that publication venue and how the content will be accessible.
The proposals were evaluated by Sandra Barbosu (ITIF), Stephen Ezell (ITIF), and Kirsten Axelsen (DLA Piper), and possibly additional experts depending on the topic of the application. All proposal materials were kept confidential and were not shared with anyone who was not a part of the review process. They were evaluated on the ability of the research to address an important gap in the evidence, the clarity and soundness of the methodology as described, and the novelty of the approach. Candidates were asked to provide additional information or address questions about their submissions prior to final grantmaking decisions.
Other publications in this series are available through ITIF’s Center for Life Sciences Innovation. To read the whole series, search #BiopharmaPolicyEvidence on itif.org.
