When cost data are collected in a clinical study, interest centers on the between-treatment difference in mean cost. When censoring is present, the resulting loss of information can be limited by collecting cost data for several pre-specified time intervals, leading to censored longitudinal cost...

Risk sharing arrangements relate to adjusting payments for new health technologies given evidence of their performance over time. Such arrangements rely on prospective information regarding the incremental net benefit of the new technology, and its use in practice. However, once the new...

Background:Background: Assessment of health state and health-related quality of life (HR-QOL) are limited by a child's age and cognitive ability. Parent-proxy reports are known to differ from children's reports. Simultaneous assessment using a parent-child dyad is an alternative approach....

Previous application of value-of-information methods to optimal clinical trial design have predominantly taken a societal decision-making perspective, implicitly assuming that healthcare costs are covered through public expenditure and trial research is funded by government or donation-based...

The parent-child dyad approach demonstrated moderate to strong performance characteristics in generic and disease-specific questionnaires suggesting it may be a valuable alternative to relying on parent proxies for assessing children’s utility and HR-QOL. Future research in additional...

Previous application of value-of-information methods to optimal clinical trial design have predominantly taken a societal decision-making perspective, implicitly assuming that healthcare costs are covered through public expenditure and trial research is funded by government or donation-based...