Real‐World Data and Physiologically‐Based Mechanistic Models for Precision Medicine

Developing Clinically Relevant Dissolution Specifications (CRDSs) for Oral Drug Products: Virtual Webinar Series

“…Middle-out modelling approaches allow the estimation of variability and reverse translation of physiological parameters, leading to opportunities in virtual bioequivalence and mechanistic in vitro–in vivo extrapolation of drug formulations [ 70 , 71 , 72 , 73 ]. Real-world data (RWD) from healthcare and registries are combined with PBPK to inform drug development in special populations and derive inference from pharmacovigilance data [ 74 , 75 ]. Further, the wider adoption of advanced simulation methods in pharmaceutics modelling, such as global sensitivity analysis, allows more systematic interrogation of the impact of information on simulation outputs.…”

Data Science in Clinical Pharmacology and Drug Development for Improving Health Outcomes in Patients

“…There are also many opportunities where RWD can benefit clinical pharmacologists, including streamlining or even replacing clinical trials in a few instances, 6,7 informing on difficult-to-study populations, such as children, 7 or rare diseases, drug repurposing, 8 pharmacovigilance, 9 pharmacokinetic/pharmacodynamic modeling, 10 and physiologically-based pharmacokinetic modeling. 11 Given the range of opportunities, we must also understand the issues and challenges in how RWD should be used [12][13][14] and ensure that appropriate validation is undertaken for new methods. 15 RWD is also used commonly in the context of the explosion of data available from -omics, continuous, ambulatory (usually in the real world) patient-monitoring technology, including wearables and other high-capacity data capture and analytical methods (often referred to as Big Data).…”

“…Thus, there is a significant opportunity to provide societal impact and benefit by conducting carefully designed and hypothesis‐driven RWD studies. There are also many opportunities where RWD can benefit clinical pharmacologists, including streamlining or even replacing clinical trials in a few instances, informing on difficult‐to‐study populations, such as children, or rare diseases, drug repurposing, pharmacovigilance, pharmacokinetic/pharmacodynamic modeling, and physiologically‐based pharmacokinetic modeling . Given the range of opportunities, we must also understand the issues and challenges in how RWD should be used and ensure that appropriate validation is undertaken for new methods …”