Drug Evaluation Committee Study to Promote the Utilization of the Registry for Pediatric, Rare and Intractable Diseases
Clinical Evaluation Subcommittee
April 2022
Although industry, government, and academia have been active in promoting the utilization of registries for drug development, there are only a limited number of cases in Japan where registries have been utilized for drug development.
In Task Force 1 of the Clinical Evaluation Subcommittee 2021, we focused on registries for pediatric, rare, and intractable diseases, which are considered to have particularly high needs for utilization in drug development, and examined the utilization of these registries in drug development. Based on publicly available information and the results of a questionnaire survey of member companies of the Clinical Evaluation Subcommittee, the Japan Pediatric Society Subcommittee, and related academic societies, the Subcommittee document summarizes the achievements and challenges in utilizing the registry in each drug development situation, and offers proposals for solving these challenges. We hope that this document will be of some help when considering the use of the registry in drug development for pediatric, rare, and intractable diseases.
Study to Promote the Utilization of the Registry for Pediatric Rare and Incurable Diseases (938KB)
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