Economic benefit of NINDS-supported clinical trials estimated at more than $15 billion

A comprehensive review of all phase III clinical trials supported by one Federal agency finds that, estimated conservatively, the economic benefit in the United States from just eight of these trials exceeded $15 billion over the course of 10 years. The study also found that new discoveries from the trials were responsible for an estimated additional 470,000 healthy years of life. The clinical trials were sponsored by the National Institutes of Health's National Institute of Neurological Disorders and Stroke (NINDS).

The study is one of the first to systematically analyze the impact of a publicly funded research program on medical care, public health, and health care costs. The analysis showed that the 10-year return on the investment in clinical trials research funding was 4600 percent. The researchers also found that the projected benefits of the clinical trial program during the period covered by this study were more than $50 billion far greater than the total budget of the NINDS ($29.5 billion) during that period. The investment in most of the trials was returned through health benefits within 1.2 years after the trial funding ended.

"The results of this analysis demonstrate the return of the public investment in NIH research for the American people not only in economic terms, but in additional healthy years of life," says Elias A. Zerhouni, M.D., Director of the NIH. "We are transforming the practice of medicine by moving into an era when treatment will increasingly become more predictive, personalized, and preemptive."

"This study strongly suggests that, for this institute at least, the economic benefit from clinical trials more than offsets the total expenditures on clinical and basic research," says Story C. Landis, Ph.D., director of the NINDS.

The investigators, led by S. Claiborne Johnston, M.D., Ph.D., of the University of California, San Francisco, evaluated the costs and public heath benefits of all 28 phase III clinical trials supported by the NINDS between 1977 and 2000. The total cost of funding these trials was $335 million. The investigators reviewed publications on treatment utilization, societal cost, and health impact. When necessary, they supplemented the published data with other publicly available information from organizations that pool sales data, the companies that manufacture the drugs and devices tested, and disease-based non-profit organizations. Information on the utilization of the tested therapies and their impact on societal costs and savings or quality of life was available for just eight of these trials. The costs of the other 20 trials were included in the analysis, but their potential benefits were not. The study appears in the April 22, 2006, issue of The Lancet.*

"We tried as best we could to be very systematic and to estimate conservatively whenever an estimation was required. In spite of all that, we found that there was a tremendous positive impact from the program of clinical trials at NINDS," says Dr. Johnston.

All cost and economic impact data were converted to 2004 U.S. dollars in order to generate 10-year projections of the impact of the trials, starting from the point at which the funding for each trial ended. The investigators used a statistical measure called "quality-adjusted life years" (QALY) to measure the impact of improvements in health and survival from changes in medical practice associated with the trials. Using this measure, a year of life in perfect health is considered equal to 1 QALY, and years of sub-optimal health are assigned numerical values between 0 and 1 based on the severity of impairment. The value of a single QALY was estimated at $40,310, which was the average economic productivity of a U.S. resident in 2004, regardless of employment or age, according to the U.S. Bureau of Labor Statistics. The total societal economic impact for the trials was calculated as the total net health benefit in QALY, multiplied by average economic productivity, minus the increase in costs related to the tested therapies and the total cost of the clinical trials.

Among the eight trials with adequate data for analysis are some of NINDS' best-known successes. One was a trial of tissue plasminogen activator (t-PA) for ischemic stroke that showed that t-PA could prevent brain damage if used within the first three hours after a stroke begins. Another was the Randomized Indomethacin Germinal Matrix/Intraventricular Hemorrhage Prevention Trial, which showed that using indomethacin in premature babies can prevent brain hemorrhage. Each of these studies had an estimated net benefit of more than $6 billion over 10 years.

In spite of this, the analysis did not include the benefits of many other trials for which information on impact was incomplete. Many of these trials have greatly changed clinical practice and have probably had a major public health impact, such as a study that established the use of methylprednisolone after spinal cord injury, the researchers note. Therefore, the total benefits of the research program are likely underestimated, they say.

The study also did not estimate the potential economic and health impact of less obvious results from the clinical studies. For example, clinical trial results might change the use of treatments similar to the one(s) that were tested. The trial results might also lead to new basic research discoveries. "We made no attempt to value the scientific discoveries and methodological advances from clinical trials; as some of the most highly cited publications in the literature, this impact is probably substantial," the researchers say.


The research was funded by the NINDS but, to reduce bias, it was performed independently and the sponsor had no control over the content of the analysis. Also, an independent panel of health policy experts audited the analysis and reviewed the manuscript.

The NINDS is a component of the National Institutes of Health (NIH) within the Department of Health and Human Services and is the nation's primary supporter of biomedical research on the brain and nervous system. The NIH is made up of 27 Institutes and Centers. It is the primary Federal agency for conducting and supporting basic, clinical, and translational medical research, and investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit

*Johnston SC, Rootenberg JD, Katrak S, Smith WS, Elkins JS. "The impact of an NIH program of clinical trials on public health and costs." The Lancet, April 22, 2006, Vol. 367, pp. 1319-1327.

Last reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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