Drug Evaluation Committee Contribution of Clinical Pharmacology to Ensuring Diversity in Drug Development - Toward Closing the Gap in Patient Backgrounds between Clinical Trials and Real-Life Clinical Trials
Clinical Evaluation Subcommittee
April 2026
Pharmacokinetics, efficacy, and safety of a drug are affected by various patient backgrounds. However, the patient populations included in pivotal studies do not always fully reflect the patient populations in post-approval clinical trials. One of the factors that make it difficult to include certain populations in clinical trials is the lack of efficacy and safety information to determine whether or not to include them. Clinical pharmacological approaches, including modeling and simulation (M&S), can be one way to address these challenges.
Therefore, the Ongoing Issues Team-8 (Clinical Pharmacology) will organize the issues involved in including populations with specific backgrounds in pivotal trials, establish selection and exclusion criteria to promote diversity of trial participants, and conduct M&S and other clinical pharmacology approaches to evaluate efficacy and safety in these populations and to provide information in the package inserts. We discussed how clinical pharmacological approaches, including M&S, can be used to evaluate efficacy and safety in this population and to provide information in the package inserts. Based on the results of the discussion, the subcommittee compiled recommendations from the perspective of clinical pharmacology for the entire development team involved in clinical development planning, clinical trial execution, and regulatory affairs.
We hope that these results will promote the "acquisition of actual data on efficacy and safety of target patients in clinical trials" throughout the entire drug development process and contribute to supplementing information on efficacy and safety in "certain populations with specific backgrounds for which information may be lacking," thereby contributing to the appropriate provision of information through package inserts and other means. We hope that this will help to provide appropriate information through the package inserts and other documents.