Regulators of new products confront a tradeoff between speeding a product to market and collecting additional product quality information. The FDA's Breakthrough Therapy Designation (BTD) provides an opportunity to understand if regulators can use new policy to innovate around this tradeoff. We find that the BTD program shortened clinical development times by 23 percent and did not impact the ex post safety profile of drugs with the designation. The BTD program had the greatest impact on less experienced firms and reduced clinical trial design complexity. The results suggest that targeted regulatory innovation can shorten R&D periods without compromising product quality.

Risk evaluation and mitigation strategy (REMS) programs help ensure that the benefits of certain high-risk medications outweigh their risks. With the routine use of REMS programs, however, concerns have emerged that they may delay the approval of generic alternatives. Using a data set of novel small-molecule drugs approved during the period 2000–13, linked to generic approvals through 2024, we assessed the impact of REMS programs on generic approval. We found that the existence of a REMS program was associated with a 25 percent delay in generic approvals after patent length, approval timing, approved indication, boxed warning status, orphan drug status, and drug use were controlled for. Our findings provide insight into future regulatory efforts that can facilitate timely access to safe, effective, and affordable generic drugs.

Academic Medical Centers (AMCs) — comprising medical schools, teaching hospitals, and research laboratories — play an important role in US biomedical innovation. The Balanced Budget Act of 1997 changed reimbursements for Medicare inpatient claims and subsidies for medical residents. We compare AMCs’ relative exposure to the reform, how these differences affect their researchers’ ability to attract NIH grant funding, and the quantity, impact, and content of their publications. We find that in response to the reform, research activity increased by approximately 6%. Changes in research composition suggest that hospitals responded to Medicare funding cuts by encouraging incumbent investigators to increase their research activities and by redirecting hiring efforts towards individuals attracted to AMCs (e.g., translational researchers). We find little effect on clinical outcomes.

This paper studies how competitive dynamics shape innovative firms’ voluntary disclosure of product quality information. Our empirical context is the pharmaceutical industry, where firms must decide whether to disclose private drug quality information acquired in clinical trials. Using a difference-in-difference strategy, we show that the approval of a competitor’s drug lowers the likelihood of a firm reporting its clinical trial results by 13%. We explore how these effects vary based on the project quality, competitor type, and firm experience. These findings suggest that strategic considerations play a role in firms’ disclosure decisions: in response to a competitor’s drug approval, firms may selectively withhold information to maintain and improve their competitive position.

The Food and Drug Administration’s (FDA’s) breakthrough therapy designation (BTD) program was created to increase patient access to safe and effective therapies by supporting the efficient clinical development of qualifying, clinically meaningful therapies. Using a new data set of key development milestones for drugs approved between 2006 and 2020, including both BTD drugs and a set of comparator drugs identified by FDA experts, we estimated the BTD program’s impact on time spent in late-stage clinical development, measured as the elapsed time between a drug’s end-of-Phase-II meeting with regulators and its approval for marketing. Our analysis suggests that the BTD program lowers late-stage clinical development time by 30 percent. Our findings provide insight into future regulatory and innovation policies aimed at driving efficiency in medical product development to ensure timely patient access to the most clinically meaningful therapies.

The COVID-19 pandemic highlighted the importance of timely access to clinical trial results for public health. Despite decades-long efforts to improve results reporting for clinical research, problems persist. Trial investigators have 3 key platforms to disseminate results: trial registries, medical journals, and medical conferences. These platforms vary in their accessibility, scope, and depth. Trials presented as abstracts at conferences are limited in word count length and audience (conference attendees). Additionally, while ClinicalTrials.gov offers publicly accessible trial result summaries, journal publications often require payment for more detailed trial reports. Accordingly, we characterized results reporting across these platforms for trials registered in ClinicalTrials.gov completed between 2008 to 2021 with an oncologic indication, the second leading cause of death in the United States.

The COVID-19 pandemic created a large, sudden unmet public health need for rapid access to safe and effective treatments. Against this backdrop, policy makers and researchers have looked to drug repurposing—using a drug previously approved for one indication to target a new indication—as a means to accelerate the identification and development of COVID-19 treatments. Using detailed data on US clinical trials initiated during the pandemic, we examined the trajectory and sources of drug repurposing initiatives for COVID-19. We found a rapid increase in repurposing efforts at the start of the pandemic, followed by a transition to greater de novo drug development. The drugs tested for repurposing treat a wide range of indications but were typically initially approved for other infectious diseases. Finally, we documented substantial variation by trial sponsor (academic, industry, or government) and generic status: Industry sponsorship for repurposing occurred much less frequently for drugs with generic competitors already on the market. Our findings inform drug repurposing policy for both future emerging diseases and drug development in general.

Public scientific data can uncover unexpected adjacencies across domains, enabling firms to redefine and expand the use of their existing assets. This paper examines the impact on private sector investment of large-scale public cancer genome mapping studies, which systematically identify the genetic abnormalities in cancer and reveal connections between previously unrelated diseases. Using newly constructed data from such studies and clinical trials, I find that publicly available mapping information increased private investments in clinical trials by 66%. These effects are driven primarily by incumbent firms repurposing existing drugs rather than developing novel ones and by firms with relevant domain expertise. The effects vary across markets and drug development stages, showing that cancer maps are also associated with improvements in firms’ decision-making as firms advance trials with a higher likelihood of clinical success. These findings highlight the role of public science in shaping firms’ R&D decisions by redefining the value of existing assets and reinforcing the advantage of those equipped to act on newly revealed scientific connections.

During research and development, firms must decide which types of users to engage to develop and test their products. We investigate how increasing representation of target users in product development shapes innovation, product quality and adoption. In 2000, Medicare expanded coverage of clinical trial costs, reducing participation costs for elderly enrollees—a historically underrepresented group. Reducing financial constraints significantly increased trials for diseases common among the elderly, led trial sponsors to broaden enrollment criteria, and may have increased elderly enrollment. This policy was associated with increased prescription utilization among elderly individuals. To explore potential mechanisms, we examine clinical outcomes and adverse events. Our findings highlight how financial incentives can improve representation in the innovative process and ultimately shape product quality and adoption.

In regulated markets, high entry barriers create incentives for firms to circumvent regulation or selectively avoid approval for certain products. We provide novel evidence of such regulatory arbitrage in U.S. pharmaceuticals: Firms conduct clinical trials in large patient populations but initially seek approval in smaller, lower-risk markets to reduce regulatory burden. They then rely on off-label drug use to expand demand beyond approved uses. While this approach expedites market entry, it raises policy concerns about treatment adoption without strong supporting evidence. Our findings highlight how regulatory design may induce strategic entry by firms and alter the diffusion of new technologies.