10 tips on performing economic evaluation in kidney disease

Nephrology has benefited from a growing body of high-quality clinical evidence, including clinical trials of pharmacological therapies and health service research on alternative care approaches. Consequently, there is an increasing need to perform economic evaluations in kidney disease to inform reimbursement decisions and optimise healthcare spending, thereby improving patient care within budget constraints. Cost-effectiveness assesses if the additional health gains are worth any additional costs by estimating differences in the quality and quantity of life, and the costs, from the point of intervention over observed but also longer (even lifetime) timelines, capturing the entire patient pathway through healthcare, e.g. from early-stage chronic kidney disease (CKD) through to dialysis or transplantation. Working with stakeholders to define the decision problem, merging evidence from a range of sources, including clinical trials complicated by limited follow-up and non-generalisable participants, surrogacy studies to estimate the intervention's impact on longer-term kidney failure risk, quality of life data collected ideally using instruments sensitive to kidney disease progression and other real-world data are required to make extrapolations sufficiently far into the patient's lifetime to capture kidney failure. Consideration of disadvantaged populations and how interventions may operate differently in certain groups may be indicated. Failure to capture competing risks of cardiovascular disease and death will bias estimates of kidney failure. Application of our tips, combined with an understanding of how decision-makers use cost-effectiveness results and information about factors like rarity and disease severity maximises the likelihood of new kidney treatments and care approaches being adopted.