Exploratory Research and Science A Survey of Scientific Sources of Pharmaceutical Innovation and Their Economic Impact (1)

Sadao Nagaoka (Professor, Tokyo Keizai University, Former Professor, Center for Innovation Research, Hitotsubashi University)
Junichi Nishimura (Associate Professor, Faculty of Economics, Gakushuin University, Visiting Fellow, Pharmaceutical and Industrial Policy Research Institute)
Koichi Genda (Former Senior Researcher, Pharmaceutical and Industrial Policy Research Institute)

(No. 66: Published in August 2015)

This paper summarizes the results of a survey on the scientific sources of pharmaceutical innovation and their economic effects, which was conducted on exploratory research. The survey covered exploratory projects aimed at creating New Molecular Entity (NME) drugs, and the main findings are as follows. First, the contribution of science in exploratory research was comprehensively understood. Second, the mechanisms of science utilization and the location of the utilized science were investigated. Third, we investigated the relationship between the degree of uncertainty in exploratory projects and the degree of progress of science at the start of the project, the frequency of selection of projects with high uncertainty, the role of individual free research, and the (ex ante) response at the start of the project to uncertainty.

The main conclusions from the research in this paper are as follows. First, Science has a very important contribution to make in exploratory research for drugs containing new active ingredients. Science inspires and helps to implement R&D projects, serves as a source of product innovation, and provides an important means for companies to engage in highly original R&D. Second, in a group of exploratory projects that are more original, the relative importance of the scientific and technological literature declines and the importance of industry-academia collaboration with human interaction becomes more important. The more original the project is, the more incomplete the science is: "the disease mechanism of the indication is unknown," "the target molecule is unknown," and "the relationship between the target molecule and the disease mechanism is unknown. Third, even in the case of drugs that have been registered and launched, about 60% of them have experienced crises that almost led to discontinuation, and in this respect, there is not much difference from cancelled or pending projects. Drug discovery projects are in the process of overcoming unexpected difficulties, and scientific progress often contributes to the resolution of these difficulties. Fourth, individual initiative plays an important role in exploratory research, which is highly original and at the same time highly uncertain, and individual independent research ("dark research") plays an important role in resolving unanticipated difficulties. These are also important elements of the ability to absorb and utilize science.

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