Clinical trials are the way the medical field tests whether a new therapeutic product performs as expected and actually makes a difference in treating disease. Hundreds of innovative therapies are generated in laboratories, but few survive early development to reach the point of human testing. Clinical trials in patients suffering from a specific condition represent the crucial link between scientific discovery and medical utility.

To plan and execute a clinical trial today can take years and cost hundreds of millions of dollars. In the past, the United States was considered the best place to conduct clinical trials because of the right mix of clinical and scientific expertise and an understanding of the research process. However, many believe that the clinical research enterprise in the United States has failed to keep pace with that in the rest of the world because of this time and cost burden. To evaluate the state of clinical research in the United States and identify strategies for enhancing the effectiveness and efficiency of clinical trials, the Institute of Medicine’s Forum on Drug Discovery, Development, and Translation convened a public workshop on October 7–8, 2009, titled Transforming Clinical Research in the United States. Clinical trial experts from academic research centers, pharmaceutical companies, contract research organizations, government, nonprofit research networks, and patient advocacy groups came together to discuss their clinical trial successes and failures, the challenges they face in conducting clinical research, and strategies for improving the efficiency of clinical trials while maintaining the highest standards for the data generated.

The intent of the workshop was to engage stakeholders in an honest discussion of the state of clinical trials today and to gain an understanding of what has and has not worked in planning and executing trials. The workshop was focused on four disease areas: cardiovascular disease, depression, cancer, and diabetes. Although “clinical research” is a generic term, a clinical trial in breast cancer, with 5-, 10-, or 15-year outcomes, is quite different from a clinical trial in cardiovascular disease, where the outcome of interest may occur in a month or less. The disease being studied also affects the kind of patients needed and how they are recruited and retained. Gaining an appreciation of the differences in clinical trials by disease helped generate ideas for improving the clinical research enterprise as a whole.

This workshop is part of a broader initiative of the Forum addressing different aspects of clinical research. Future Forum plans include the following: further examining regulatory, administrative, and structural barriers to the effective conduct of clinical research; developing a vision for a stable, continuously funded clinical research infrastructure in the United States; and considering strategies and collaborative activities to facilitate more robust public engagement in the clinical research enterprise.

As the starting point for the Forum’s work in the area of clinical research, it is our hope that this workshop summary will serve as a resource for all organizations and individuals seeking a greater understanding of how the clinical research enterprise works and how it can improve. The workshop showcased the best examples from clinical research conducted to date and developed novel ideas for organizing and conducting clinical trials. Ultimately, as the health care system moves forward, we hope our work can serve as a source of information and inspiration to those involved in clinical research as sponsors, investigators, clinicians, patients, and policy makers.

Jeffrey M. Drazen, Co-Chair

Forum on Drug Discovery, Development, and Translation