Improving Realism in Clinical Trial Simulations via Real‐World Data

Simulation validity depends on how well sampling distributions used reflect real‐patient characteristics, such as drug adherence, disease progression, and pharmacologic handling in the body. We challenge the current use of growth charts from nondisease‐specific pediatrics in simulations for drug development. Complementary use of data from clinical trials and the real‐world is expected to achieve a more realistic representation of clinical outcomes for decisions in drug development, regulatory approval, and health technology assessment.