Cost–utility analysis conducted alongside randomized controlled trials: Are economic end points considered in sample size calculations and does it matter?

SAGE: 12/11/12

Background Many randomized controlled trials (RCTs) collect cost-effectiveness data. Without appropriate sample size calculations, patient recruitment may cease before the cost-effectiveness of the intervention can be established or continue after the cost-effectiveness of the intervention is established beyond doubt.

Purpose We determined the frequency with which cost-effectiveness is considered in sample size calculations and whether RCT-based economic evaluations are likely to come to inconclusive results at odds with the clinical findings.

Methods We searched the National Health Service Economic Evaluation Database (NHS EED) to identify RCT-based cost-utility analyses. RCTs that collected individual patient data on costs and quality-adjusted life years (QALYs) were eligible. Studies using models to extrapolate the results of RCTs or with insufficient information on incremental costs and QALYs were excluded. Read more