小児医薬品臨床試験の開始前に確認すべき事項を含めたコンセプトシートの開発

Background: There are many factors to be considered when planning intervention trials（hereinafter referred to as "clinical drug trials"）in the pediatric field, which aim at establishing the practical application of a pharmaceutical product and accruing evidence to support it. However, there are many trials that are planned without sufficiently considering these issues. Objective: We conducted a questionnaire survey of Researchers and Research Supporters belonging to the network of Japanese Association of Children,s Hospitals and Related Institutions, to develop a concept sheet that identifies points to be considered when planning a clinical drug trial in the pediatric field. Methods: Relying on past experience as well as domestic and international guidelines, our team of three experts （including myself）, who are currently responsible for providing developmental research consultation at the National Center for Child Health and Development, drafted a concept sheet, which identifies points to focus on and check before planning a pediatric clinical drug trial. Based on the contents of the concept sheet, we conducted a survey of pediatric Researchers and Research Supporters to determine what they should consider before planning a pediatric clinical drug trial. The concept sheet was revised and finalized based on the results of the survey. Results: We identified the following items to be checked when planning a pediatric drug clinical trial: "Sufficiency of non-clinical data"", Natural history of the disease"", Availability and appropriateness of pediatric dosage forms （for drugs）"", Status of approval for pediatric use in Japan and overseas"", Availability of guidelines"", Validity of pediatric dosing"", Validity of assessment index"and"Necessity of pharmacokinetics/pharmacodynamics evaluation". Based on the results of the aforementioned survey, "Progress of domestic and foreign research and clinical trials for pediatric patients"was added as an item to the concept sheet, which was then finalized. Conclusion: We developed a concept sheet that includes items to be confirmed when planning a drug clinical research in the pediatric field. We believe that this concept sheet will help Researchers and Research Supporters to achieve their goals, such as regulatory approval and inclusion in guidelines, when planning pediatric drug clinical trials, by enabling them to conduct a thorough preliminary review. We will be revising the concept sheet as necessary based on future use experience.