Current Issues and Future Evolvement of Pediatric Drugs Development in Japan: From a Survey of Medical Institutes on the Development of Pediatric Drugs

Although pediatric drugs are essential in treating children with severe diseases, there is a notable delay and absence of their clinical development in Japan; consequently, acceleration of the pediatric drug development process is an urgent issue. To reveal the underlying causes of this inadequacy of pediatric drug development in Japan, we conducted a questionnaire survey involving physicians, clinical research coordinators, and others involved in pediatric clinical trials at medical institutions. The lack of profitability for companies, small number of pediatric patients, and difficulty in conducting pediatric clinical trials are the dominated responses of the reasons for hesitating drug development for pediatric oncology and rare intractable diseases, with response rates of 72.3％ (180/249), 56.6％ (141/249), and 53.0％ (132/249) for pediatric oncology, and 77.5％ (193/249), 63.1％ (157/249), and 45.4％ (113/249) for pediatric rare and refractory diseases, respectively. The lack of knowledge and experience on the part of medical institutions is the dominant reason for the difficulty of study implementation, with 83.1％ (123/148 patients) for pediatric cancer and 86.6％ (110/127 patients) for rare intractable diseases. 83.5％ (208/249) of the respondents were in favor of encouraging multinational trials, respectively. Clinical trials need to be concentrated in base hospitals to improve knowledge and experience regarding multinational trial. As suggestions for other measures, some respondents mentioned incentives for companies, while others mentioned making pediatric clinical study obligatory. Obligatory and incentives need to be considered together. On the contrary, only 63.9％ (159/249) of the respondents were positive about strengthening patient-requested therapy system (“Kanja moushide ryoyo”).