| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 5521035 | Drug Discovery Today | 2016 | 9 Pages |
â¢Comprehensive analysis of R&D of orphan drugs between Japan and Europe.â¢Rare diseases were categorized into 4 clusters by regional different prevalence.â¢Diseases with higher regional prevalence have more progress of R&D in the region.â¢Orphan drug development that considers regional differences may be more effective.â¢Moreover progress factors of R&D of orphan drugs are discussed.
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.
