The Truth About Drug Innovation: Thirty-Five Summary Case Histories on Private Sector Contributions to Pharmaceutical Science

The increasingly important role of prescription medicines as both complements to and substitutes for other medical procedures, as well as rising costs for newer and more effective medicines, has precipitated an array of proposals for reducing private and public spending on drugs. Some prominent observers have questioned whether the current system of research and development is as cost-effective as alternatives might be, and, in particular, whether the central role of private pharmaceutical firms in drug research and development produces commensurate social benefits. One contention that recently has attracted considerable attention can be summarized as follows: most of the scientific advances that yield new and improved medicines are the fruit of research financed or conducted by public agencies, the National Institutes of Health (NIH) foremost among them, rather than the pharmaceutical companies that produce and market them.

The goal of this study is to test the accuracy of this proposition. To do so, we compiled summary case histories of thirty-five drugs and drug classes (a group of drugs used to treat a given medical condition in similar ways) identified in the scholarly literature as important and/or that were among the most prescribed in 2007. Our conclusions can be described as follows: the literature on the histories of drugs makes it clear that the scientific contributions of the private sector were crucial for the discovery and/or development of virtually all of the thirty-five drugs and drug classes examined in this study. Such scientific advances can be classified as the basic science of biology and disease processes relevant for given medical conditions; the applied science of discovering compounds that treat particular conditions; and the development of compounds with improved clinical (medical) effects, of large-scale manufacturing processes, and the like.

Three examples of advances yielded by private-sector research are, respectively, the discoveries in basic science that led to the development of the modern drugs used to treat serious bacterial infections; the discoveries in applied science yielding drugs used to treat hypertension; and the advances in recombinant genetic science that allowed largescale production of such drugs as Epogen (used in treatment of anemia).

More generally, among our thirty-five drugs and drug classes, private-sector research was responsible for central advances in basic science for seven, in applied science for thirty-four, and in the development of drugs yielding improved clinical performance or manufacturing processes for twenty-eight. In short, all or almost all of the drugs and drug classes examined in this study would not have been developed—or their development would have been delayed significantly—in the absence of the scientific or technical contributions of the pharmaceutical firms.

Table S1 summarizes these findings, derived from the thirty-five summary case histories presented in this study.

Scientific research efforts funded, respectively, by the NIH and by pharmaceutical firms occupy very different—but complementary—niches in the process of drug development. Research conducted at government or university laboratories (often funded by the NIH or other agencies) tends to be concentrated in the basic science of disease biology, biochemistry, and disease processes. A major goal of that work is the identification of biologic targets that could prove susceptible to future drug candidates. Basic research often yields advances that cannot be patented and that often are made long before the subsequent scientific and clinical work that leads to viable new therapies.

The scientific contributions of the private sector have been weighted heavily, though not exclusively, toward the applied science of discovering ways to exploit the findings of basic science. This scientific work can be characterized as the discovery, synthesis, testing, and (often complex) manufacturing of candidate compounds intended to exploit biologic targets for the purpose of curing medical conditions or mitigating their adverse effects.

In short, although basic research occurs in both the public and private sectors, the applied science of drug development and clinical refinement of compounds occurs almost exclusively in the private sector. It is those efforts that ultimately allow new scientific discoveries to be translated into new medicines.

READ FULL REPORT