Clinical Pharmacology Regulatory Sciences in Drug Development and Precision Medicine: Current Status and Emerging Trends

Abstract

In the regulatory setting, clinical pharmacology focuses on the impact of intrinsic and extrinsic factors on inter-patient and intra-subject variability in drug exposure and response. This translational science contributes to the understanding of the benefit-risk profile in individual patients and the development of relevant therapeutic monitoring and management strategies. Clinical pharmacology also plays a major role in the development and qualification of drug development tools. This article presented some recent examples to illustrate the important roles of clinical pharmacology in drug development and evaluation. In addition, emerging trends in clinical pharmacology regulatory sciences were also discussed, including the Model-Informed Drug Development (MIDD) pilot program, the use of real-world data to generate real-world evidence, and leveraging advances in basic, biomedical, and clinical science into useful tools for drug development and evaluation. Continued advances in clinical pharmacology can be the basis of more rational and efficient drug development and improved access to new drug treatments that are tailored to the patient to achieve better efficacy and safety.

Integrating Real-World Evidence in the Regulatory Decision-Making Process: A Systematic Analysis of Experiences in the US, EU, and China Using a Logic Model

“…RWE is also related to the overall development of regulatory science in these three countries/regions. In the US, Centers of Excellence in Regulatory Science and Innovation are the institutions that cooperated most with the FDA on RWE-related projects (57). Promoting use of high-quality real-world data (RWD) in decision-making is one of the core strategies of Regulatory Science to 2025 in EU.…”

Providing Australian children and adolescents with equitable access to new and emerging therapies through clinical trials: a call to action

“…34 It was not until the late 1990s that paediatric drug development became part of standard care. 33,35 This followed the necessary development of an ethical framework for involving children in research from the 1970s; 36 the introduction of pharmacometric techniques to allow increasingly accurate paediatric dose prediction, as well as confirmation using limited blood samples; 37 advances in trial design to minimise risk and burden to children; 38 and crucially, the scientific revolution of paediatric clinical pharmacology. 33 These developments have mapped out an understanding of maturational processes in infants and children that impact drug handling.…”

Clinical trial diversity: An opportunity for improved insight into the determinants of variability in drug response

“… Intrinsic and extrinsic factors contributing to interindividual variability in pharmacokinetics and pharmacodynamics, which may lead to clinically relevant differences in treatment response (adapted from Liu et al ., 2021) 101 …”