Problems and Possible Solutions for Investigator-initiated Clinical Trials in Children

Abstract

Pharmaceutical companies are not necessarily willing to develop drugs for children, and consequently, conducting investigator-initiated clinical trials (ICTs) is essential for pediatric drug development in Japan. Since ICTs were introduced in 2003, there have been efforts to decrease the burden on clinical trial sites, but there are apparently still problems and hurdles. We have conducted a survey about these issues and possible solutions targeting those with experience regarding ICTs in children. A web-based questionnaire was sent to physicians, clinical research coordinators, and other staff involved with ICTs. The results of the questionnaire revealed several problems and hurdles, including protocol design, invasive or inappropriate laboratory testing procedures, limited blood sampling volume, lack of reference values for laboratory data, inappropriate dosage forms, and insufficient research funding. We examined the background and causes of the problems, reviewed the published papers, sought opinions from experts, and proposed solutions in this paper.