Regulators of new products confront a tradeoff between speeding a new product to market and collecting additional product quality information. The FDA’s Breakthrough Therapy Designation (BTD) provides an opportunity to understand if a regulator can use new policy to innovate around this tradeoff—i.e., whether it improved regulator productivity by allowing products to come to market more quickly without compromising quality. 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. In exploring mechanisms, we find that the BTD program had the greatest impact on less experienced firms and was associated with reduced BTD clinical trial design complexity. The results suggest that targeted regulatory innovation can shorten R&D periods without compromising the quality of new products.

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.

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.

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 and how these differences affect their researchers' ability to attract NIH grant funding, as well as the quantity, impact, and content of their publications. We find that in response to the reform, research activity increases by approximately 6%, with larger effects observed for “translational” and clinical research. We find little effect on clinical outcomes.

How does comprehensive basic scientific information shape private sector research investments among heterogeneous firms? I assess the impact of large-scale public cancer genome mapping studies, which systematically map the genetic abnormalities in cancer. Using newly-constructed data from cancer genome mapping studies and clinical trials, I find that publicly available mapping information increases private investments in clinical trials by 66%. The large-scale public release of such information has nuanced effects: it disproportionately increases research among incumbents with previously tested drugs for related diseases and spurs research activity among firms with limited access to private mapping information. Cancer maps are associated with improvements in firms’ decision-making: when genetic data becomes available, firms are more likely to initiate and advance research investments that are likely to yield promising clinical results.

We investigate the causes and consequences of demographic disparities in product development. We focus on how insurance policies affect elderly enrollment in clinical trials. In 2000, Medicare extended coverage for clinical trial costs. This policy shifted the rate and direction of clinical research, leading to an 18 percent increase in trials targeting diseases common among the elderly, compared to those affecting younger populations. We find trial sponsors expanded the enrollment criteria of trials to include more elderly participants. This policy was also associated with increase in drug utilization for elderly drugs.

We analyze firms’ product line decisions in the presence of entry regulation. We find that pharmaceutical firms prioritize smaller disease markets for a drug’s initial regulatory approval and larger markets for its subsequent approvals. These patterns are consistent with a model of strategic entry in which firms—facing high costs of regulation—exploit regulatory loopholes to expand into novel product markets. Drug regulation, in particular, features a loophole where firms can rely on off-label drug use—the practice of using an approved drug for unapproved uses—as a non-regulatory pathway for reaching new markets. These findings raise important considerations for firm managers, highlighting opportunities for utilizing non-regulatory entry pathways to expand into costly product markets, and for regulators, who must balance the trade-off between expedient access to innovative products and the need for sufficient information about their quality.