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.

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.

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.

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. In 2000, Medicare extended coverage for clinical trial costs, lowering the cost of participation for elderly enrollees. This policy shifted the rate and direction of clinical research, leading to a 24 percent increase in trials targeting diseases common among the elderly, compared to those affecting younger populations. Trial sponsors expanded the enrollment criteria of trials to include more elderly participants. This policy was also associated with an increase in drug utilization for elderly drugs and a reduction in adverse events.

We analyze the relationship between firms’ ability to leverage regulatory slack and their market entry strategies. Using newly constructed genomic measures of disease market similarity, we systematically document evidence of forum shopping, whereby pharmaceutical firms seek the most lenient regulatory environment for approval when drugs have multiple potential therapeutic uses. Firms seek regulatory approval in smaller disease markets to lower the costs of regulation and rely on complementary, non-regulatory pathways—in the form of unapproved, “off-label” drug use—to expand demand. Our data allow us to characterize the degree to which new technologies can exploit such opportunities, shedding light on how firms navigate regulatory environments to speed the entry of new products to market.