Abstract
The current status of pediatric drug approval and the problems of off-label drug use in Japan are described. Investigation reports published by the Pharmaceuticals and Medical Devices Agency(PMDA)showed that of the 740 drugs that had been approved from April 2001 to March 2012,154(20.8%)had pediatric indications. As short of medicines for children,we are obliged to prescribe off-label drugs in our daily practice. The most important problem of the off-label drug use is that we have to use drugs that have no description of adequate effect-efficacy for children. In addition,there are no clinical data concerning PK/PD and safety in children and the adequate dosage can not be determined. Furthermore,if a serious adverse event occurs in a patient treated with an off-label drug,the patient could not receive official relief aid by the PMDA. Another problem of off-label drug use is that no repayment could be applied to the medical institution in the health insurance system.
Clinical trials are indispensible to address these issues. However,clinical trials appear hard to perform in children. Another option to obtaining approval is filing an application based on the official notification describing procedures for approval of off-label drugs. This notification,published by the Ministry of Health,Labor,and Welfare in 1992 in order to approve offlabel drugs without a clinical trial,appears helpful in promoting pediatric indications for off-label drugs.
