title: Research and drug development activities in rare diseases: differences between Japan and Europe regarding influence of prevalence.
abstract: Orphan drug legislation has contributed enormously to promote drug development for rare diseases but further effective and sustainable approaches are required. This study focused on the difference of rare disease prevalence between Japan and Europe, classified the rare diseases comprehensively using cluster analysis and analyzed the influence of prevalence on research activity and drug development. Although overall strong correlative progress of research was found and absolute numbers of values were greater in Europe than in Japan, the regional higher prevalent diseases demonstrated more progress of research and development relatively in the region by examining clusters. Our findings suggest potential optimal drug development in consideration of regional differences. Moreover, an in-depth analysis of diseases that showed exceptional research achievements compared with prevalence speculated important determinants of progress.
authors.affiliation: Bio-Innovation Policy Unit, Department of Computational Biology and Medical Sciences, Graduate School of Frontier Sciences, The University of Tokyo, 5-1-5 Kashiwanoha, Kashiwa-shi, Chiba 277-8561, Japan. Electronic address: kk147342@mgs.k.u-tokyo.ac.jp.
authors.forename: Hirokuni
authors.initials: H
authors.lastname: Mizoguchi
authors.affiliation: Bio-Innovation Policy Unit, Department of Computational Biology and Medical Sciences, Graduate School of Frontier Sciences, The University of Tokyo, 5-1-5 Kashiwanoha, Kashiwa-shi, Chiba 277-8561, Japan.
authors.forename: Takayuki
authors.initials: T
authors.lastname: Yamanaka
authors.affiliation: Bio-Innovation Policy Unit, Department of Computational Biology and Medical Sciences, Graduate School of Frontier Sciences, The University of Tokyo, 5-1-5 Kashiwanoha, Kashiwa-shi, Chiba 277-8561, Japan.
authors.forename: Shingo
authors.initials: S
authors.lastname: Kano
mesh_terms: D035843:Biomedical Research; D016000:Cluster Analysis; D055808:Drug Discovery; D005060:Europe; D006801:Humans; D007564:Japan; D015995:Prevalence; D011643:Publishing / Q000706:statistics & numerical data; D035583:Rare Diseases / Q000188:drug therapy / Q000453:epidemiology*