Abstract and Keywords
Health economic evaluations face the issues of noncompliance and missing data. Here, noncompliance is defined as non-adherence to a specific treatment, and occurs within randomized controlled trials (RCTs) when participants depart from their random assignment. Missing data arises if, for example, there is loss-to-follow-up, survey non-response, or the information available from routine data sources is incomplete. Appropriate statistical methods for handling noncompliance and missing data have been developed, but they have rarely been applied in health economics studies. Here, we illustrate the issues and outline some of the appropriate methods with which to handle these with application to health economic evaluation that uses data from an RCT.
In an RCT the random assignment can be used as an instrument-for-treatment receipt, to obtain consistent estimates of the complier average causal effect, provided the underlying assumptions are met. Instrumental variable methods can accommodate essential features of the health economic context such as the correlation between individuals’ costs and outcomes in cost-effectiveness studies. Methodological guidance for handling missing data encourages approaches such as multiple imputation or inverse probability weighting, which assume the data are Missing At Random, but also sensitivity analyses that recognize the data may be missing according to the true, unobserved values, that is, Missing Not at Random.
Future studies should subject the assumptions behind methods for handling noncompliance and missing data to thorough sensitivity analyses. Modern machine-learning methods can help reduce reliance on correct model specification. Further research is required to develop flexible methods for handling more complex forms of noncompliance and missing data.
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