National Academy of Medicine

Targeted Research: Brain Disorders as an Example--A Vital Direction for Health and Health Care

By Alan Leshner, Steven E. Hyman, and Story Landis
September 19, 2016 | Discussion Paper

Much discussion surrounds the question of the most appropriate strategies for bringing the power of science to bear on the nation’s pressing problems. Some problems, such as an emerging infectious disease, are urgent and must be addressed immediately. Others, such as the increasing global burden of dementia and other neurodegenerative diseases as populations grow older, become apparent with time but can be just as pressing in their implications. Advances in science and technology are often critical for progress, and circumstances can make it imperative for major science-based initiatives to deal with problems. We argue here that now is the right time for a substantial science-based assault on disorders of the brain. Our thesis is based on the conjunction of a growing worldwide societal burden of brain disorders with scientific opportunity driven by the maturing of neuroscience and related disciplines, by the recent and continuing emergence of relevant tools and technologies, and by the quality and number of personnel in the field.

Policy Strategies

There is no simple recipe for the planning and conduct of science-based initiatives that would ensure advances in both scientific progress and their application to societal problems. Much, however, has been learned from prior science initiatives. The temptation is always great, particularly when funding is constrained, to focus research funding in explicit or targeted ways, specifying in detail the exact problems to be solved and even the research approach to be taken. But the history of American science shows that stipulation of details can be counterproductive. What has generally been proved most effective is a combination of approaches to the support of research and development that involves diverse strategies. Moreover, it should be emphasized that increased funding, although almost always a necessary condition for progress, will not by itself yield solutions to critical problems. The science must be tractable—even if difficult—and there must be an appropriate workforce in the field in question or workers willing to enter from related fields. Both those conditions prevailed in the response to HIV/AIDS that began in the 1990s.

In addition to substantial increments in funding, policy and regulatory initiatives may be required to advance relevant science and to apply it effectively to the pressing problems that motivated the investment. Policy initiatives involving regulatory and possibly legislative bodies, the academic and industrial sectors, and journal publishers can markedly increase the likelihood of successful research and societal outcomes. Examples include the sharing of data (in conjunction with appropriate ways of protecting the privacy of individuals), the sharing of methods and key reagents by scientists, increased incentives for scientific rigor (as opposed to premature publication), and decreased barriers to partnerships between academic and industrial researchers that address the issue of conflicts of interest.

About the Vital Directions for Health and Health Care Series

Vital DirectionsThis publication is part of the National Academy of Medicine’s Vital Directions for Health and Health Care Initiative, which called on more than 100 leading researchers, scientists, and policy makers from across the United States to provide expert guidance in 19 priority focus areas for U.S. health policy. The views presented in this publication and others in the series are those of the authors and do not represent formal consensus positions of the NAM, the National Academies of Sciences, Engineering, and Medicine, or the authors’ organizations.
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Suggested Citation

Leshner, A., S. E. Hyman, and S. Landis. 2016. Targeted Research: Brain Disorders as an Example – A Vital Direction for Health and Health Care. NAM Perspectives. Discussion Paper, National Academy of Medicine, Washington, DC. doi: 10.31478/201609f


Disclaimer: The views expressed in this paper are those of the authors and not necessarily of the authors’ organizations, the National Academy of Medicine (NAM), or the National Academies of Sciences, Engineering, and Medicine (the National Academies). The paper is intended to help inform and stimulate discussion. It is not a report of the NAM or the National Academies. Copyright by the National Academy of Sciences. All rights reserved.