This paper summarizes and discusses key points from the 2024 symposium titled “The 3rd JSCPT Salon: Real World Data (RWD) and Pharmacometrics (PMx),” which was organized as part of an ongoing symposium series initiated in 2013 under the theme of “Pharmacometrics as Clinical Pharmacology,” aiming to explore how pharmacometrics can further contribute to individualized drug therapy in Japan. The symposium highlighted the value of RWD in understanding patient responses that are not adequately captured in clinical trials. PMx plays a critical role in providing scientific evidence for individualized dosing by quantitatively modeling the relationships between drug exposure, efficacy, and adverse effects. While PMx enables extrapolation to special populations, challenges remain when applying these approaches to patient groups with limited clinical data. Discussions during the symposium emphasized the importance of post-marketing efficacy and safety information derived from real-world clinical practice, particularly for patients with unique or complex backgrounds. Insufficient direct feedback from healthcare professionals to pharmaceutical companies was identified as a practical challenge, alongside ongoing global regulatory discussions and recent initiatives in Japan to enhance clinical data collection and utilization. Based on these discussions, this paper emphasizes the potential of integrating RWD with PMx approaches to identify patient subpopulations with heterogeneous responses and advocates for improved frameworks to effectively translate such insights back into clinical practice.
Tanigawa et al. (Thu,) studied this question.