Use of statistical and pharmacokinetic-pharmacodynamic modeling and simulation to improve decision-making: A section summary report of the trends and innovations in clinical trial statistics conference.

The application of modeling and simulation (M&S) methods to improve decision-making was discussed during the Trends & Innovations in Clinical Trial Statistics Conference held in Durham, North Carolina, USA on May 1-4, 2016. Uses of both pharmacometric and statistical M&S were presented during the conference, highlighting the diversity of the methods employed by pharmacometricians and statisticians to address a broad range of quantitative issues in drug development. Five presentations are summarized herein, which cover the development strategy of employing M&S to drive decision-making; European initiatives on best practice in M&S; case studies of pharmacokinetic/pharmacodynamics modeling in regulatory decisions; estimation of exposure-response relationships in the presence of confounding; and the utility of estimating the probability of a correct decision for dose selection when prior information is limited. While M&S has been widely used during the last few decades, it is expected to play an essential role as more quantitative assessments are employed in the decision-making process. By integrating M&S as a tool to compile the totality of evidence collected throughout the drug development program, more informed decisions will be made.