Value-based clinical trials: selecting trial lengths and recruitment rates in different regulatory contexts

Health systems are placing increasing emphasis on improving the design and operation of clinical trials, with a view to increasing the rate of innovation and adoption of health technologies in a ‘value-based’ world. We present a value-based, Bayesian decision-theoretic model of a two-armed clinical trial and health technology adoption decision in which the recruitment rate and duration of the recruitment period are optimised. We account for a wide range of regulatory and practical contexts, addressing questions of how health is valued (considering discounting, the horizon of an adoption decision, and the endogenisation of outcomes for patients in the trial), and the state of clinical practice prior to commencement of the trial (we consider both exploratory trials for pharmaceutical research and pragmatic trials which compare technologies currently in use). We apply the model using research and treatment cost data from an existing trial and health technology assessment and challenge traditional perceptions concerning the efficiency, length and knowledge that may be gained from clinical research when trial teams are charged with delivering ‘value’ efficiently.