Comparative analysis of correlations of research and development indicators for rare diseases among Japan, the US, and Europe

Hirokuni Mizoguchi () and Shingo Kano
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Hirokuni Mizoguchi: The University of Tokyo
Shingo Kano: The University of Tokyo

Scientometrics, 2019, vol. 120, issue 2, No 1, 374 pages

Abstract: Abstract There are many rare diseases and biomedical research efforts for treatment of each disease have been ongoing. However, few reports are available to analyze overall trends for how research and development have been performed for rare diseases generally. In this research, the correlations between research and development indicators of rare diseases were examined with international comparisons among Japan, the US, and Europe. The correlation between the number of clinical studies and orphan drug designations was lower in Japan than that in the US and Europe, while the correlation between the number of orphan drug designations and orphan drug approvals was higher in Japan than in the US and Europe. This analysis clarified differences in the orphan drug designation system, operational characteristics, and beneficial points in the regions. Based on the findings of the analysis of the research/regulatory system, earlier time of orphan drug designation in Japan was proposed as a policy implication.

Keywords: Rare disease; Orphan drug; Research and development; Regulatory science; Correlation; Regional comparison; 62-07 (search for similar items in EconPapers)
JEL-codes: I18 (search for similar items in EconPapers)
Date: 2019
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DOI: 10.1007/s11192-019-03129-5

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