|Economic Benefit of NINDS-Supported Clinical Trials Estimated
at More Than $15 Billion Over Ten Years
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 a 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
"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
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.
The National Institutes of Health (NIH) — The Nation's Medical Research
Agency — includes 27 Institutes and Centers and is a component of
the U.S. Department of Health and Human Services. It is the primary federal
agency for conducting and supporting basic, clinical and translational medical
research, and it investigates the causes, treatments, and cures for both common
and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.