An Exploratory Analysis of Pharmaceutical Drugs as Basic Research Tools
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An Exploratory Analysis of Pharmaceutical Drugs as Basic Research Tools
Drug Information Journal 46(2) 192-196 ª The Author(s) 2012 Reprints and permission: sagepub.com/journalsPermissions.nav DOI: 10.1177/0092861512436581 http://dij.sagepub.com
John E. Calfee1 and Elizabeth DuPre´2
Abstract Drug development is usually seen as following a more or less linear path from publicly subsidized basic research to costly preclinical and clinical research by for-profit firms, which usually fails but occasionally succeeds in bringing highly profitable drugs to market. This study describes an additional feedback process in which pharmaceutical drugs serve as research tools. Diverse examples illustrate that the tools function of pharmaceuticals supports important advances in basic research. These often lead in turn to practical uses including new drug development. Keywords basic research, pharmaceutical economics, drug development, research externalities
Introduction A 2004 report from the National Academy of Sciences pointed out that ‘‘many corporate laboratories conduct fundamental [basic] research whose results are published in the peerreviewed scientific literature,’’ and added, ‘‘the distinctions between basic and applied research or between science and technology have broken down.’’1 As an example, researchers at the biopharmaceutical firm Genentech have published seminal work on angiogenesis, including the discovery of vascular endothelial growth factor (VEGF-A), which has proved to be an important target for drug therapy.2 Pharmaceutical firms have clear incentives to engage in substantial amounts of basic research. Among the motivating factors are the attraction of talented researchers, the facilitation of productive collaboration with academic scholars, and the possibility that the research will yield results useful for drug development.3,4 This study focuses on another relationship between forprofit pharmaceutical research and development (R&D) and basic research: a feedback process in which pharmaceuticals serve as research tools in basic science. In medical research, an established pharmaceutical can play a central role when fundamental questions are best addressed by observing the results of large, lengthy clinical trials in which an essential variable is manipulated by that drug. Such trials are expensive, often costing hundreds of millions of dollars either for the trial itself or for investments necessary to develop a suitable drug. These trials often answer long-standing scientific questions and raise new hypotheses or otherwise revise scientific and medical thinking.
A second feedback path involves investigational compounds that never reach the marketplace, or clinical trials that fail to support an intended new use for an established drug. New compounds often fail because of ‘‘off-target’’ effects, as illustrated by the HDL-boosting drug torcetrapib. Successful drugs, on the other hand, tend to have relatively weak off-target effects, and such off-target effects sometimes turn out to be therapeutically
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