Grant: Project Grant

The vast majority of new drugs tested in patients fail to demonstrate enough safety and efficacy to receive approval. Most of this failure occurs in "phase 2" clinical trials- a stage of testing aimed at showing drug efficacy in patients for the first time. Every time a drug fails in phase 2 trials, patients are exposed to a drug that is either unsafe or ineffective, and healthcare and research systems expend scarce resources pursuing blind alleys. The aim of this grant is to improve the way we make decisions about which phase 2 trials to pursue. Previous studies suggest that ethics committees and other decision-makers find it difficult to form judgments about risk, benefit and the clinical promise of new treatments tested in phase 2 trials. Moreover, the documentation used to support such evaluations often lacks critical information to help reviewers form judgments. Our study will proceed in four steps. First, we will examine what kind of information is presented to scientists and others who make decisions about whether to launch phase 2 trials. Second, we will observe how ethics and scientific committees make decisions about starting phase 2 trials. Third, we will interview experts to find out what sorts of information experts seek out when evaluating proposed phase 2 trials. Fourth, we will perform a series of experiments to test whether information formats used in trial documentation sometimes results in biased decisions. Once our four studies are complete, we will synthesize our findings into a series of recommendations for improving the information and processes used to make decisions about which phase 2 trials to pursue. We have lined up numerous partners to help ensure our findings translate into better protections for human volunteers in phase 2 trials, and more impactful drug development efforts.