Shoyo Shibata

To stimulate clinical development, the Japanese government introduced “pricing premium for the promotion of new drug development and elimination/resolution of off-label use” in 2010. Using statistical analysis, we aimed to verify the acceleration of clinical development in therapeutic areas that are positive factors for receiving reward premiums. We defined “encouragement for clinical development” as the Pharmaceuticals and Medical Devices Agency (PMDA) review time in each therapeutic area compared with that in all therapeutic areas together. The dataset for this research was created from publicly available information on the PMDA website. New molecule entities (NMEs) between 2000 and 2016 in Japan were selected as the drugs of interest. Univariate regression analysis, Wilcoxon signed-rank test, and logistic regression analysis were conducted. The number of NMEs has been decreasing over time except for B (blood and blood-forming organs). The review time was significantly shorter for A (alimentary tract and metabolism), B, J (anti-infective for systemic use), and L (antineoplastic and immunomodulating agents) compared with that for all anatomical therapeutic chemical (ATC) codes together. The review time was significantly longer for C (cardiovascular system), and S (sensory organs). From logistic regression analysis, A, B, J, and L were identified as significant positive factors to shorten the review time. In conclusion, the present study has demonstrated that clinical development in Japan has been encouraged in therapeutic areas with unmet medical needs as the PMDA review time is shortened. This study assessing the review time per ATC code is consistent with our first report where the review time per office of new drug was investigated.

In 2010, a premium rewards system for the promotion of innovative drug discovery was introduced in Japan, which aimed to further the development of innovative new medicines to meet the high level of unmet medical needs present in Japan. Previous research indicated that anti-cancer agents, immune-suppressants, and neuroscience drugs comprised the drug categories that significantly contributed to receive this reward premium. In this study, the number of new molecule entities (NMEs) approved in Japan between 2000 and 2015, along with their review times by the Pharmaceuticals and Medical Devices Agency (PMDA), were investigated to elucidate the actual clinical development status in Japan under this reward system. The dataset used in this study was created from publicly-available information on the PMDA website. For analysis, univariate regression analysis and Wilcoxon signed-rank test were used. Significant upward trends were observed in the total number of NMEs approved by the Offices of New Drug III, IV, V, and the Vaccines and Blood Products Office. No significant differences in the number of NMEs between 2000 and 2015 for each New Drug Office were observed. The median review time was 14 months; the maximum review time was 21 months (Office of New Drug III) and the minimum review time was 12 months (Offices of New Drug IV and V), excluding the Office of Vaccines and Blood Products (11 months). The review period was significantly shortened over time in the Offices of New Drug I and II. Our study suggests that the development of new drugs in therapeutic areas with high unmet medical needs has been adequately promoted in Japan, along with reasonably shortened PMDA review times.