Cost-utility analysis of difelikefalin for the treatment of moderate to severe Chronic Kidney Disease associated-Pruritus (CKD-aP) in adult patients receiving haemodialysis in Spain.

Abstract

Background and objectives: Chronic Kidney Disease-associated Pruritus (CKD-aP) is a disabling condition that affects around 60% of patients with end-stage kidney disease undergoing dialysis. Current off-label treatment options are neither effective nor appropriate for all dialysis patients, leaving a clear unmet need. This study aimed to evaluate the cost-effectiveness of difelikefalin - the only drug approved in Europe for the treatment of moderate to severe CKD-aP adult patients in haemodialysis - compared to the best supportive care (BSC) from the Spanish NHS perspective.

Methods: A Markov model was developed with seven health states: five health states representing levels of pruritus intensity over time (No, Mild, Moderate, Severe and Very severe CKD-aP), kidney transplant and death as the absorbing state. The model included patients with moderate to severe CKD-aP at baseline, in line with difelikefalin approved indication and clinical trials. Local costs, utilities, mortality rates and kidney transplant probabilities were obtained from published literature. Costs and quality-adjusted life-years (QALYs) were discounted at a 3% annual rate with a lifetime horizon (36 years).

Results: Difelikefalin was associated with an increased in QALYs (+0.49) and higher costs (+12,300€) compared to the BSC over a lifetime horizon. At a provisional cost estimate of 270.6€per 28-days for difelikefalin (based on a tentative list price for Spain), the incremental cost-utility ratio was 25,000€/QALY. The sensitivity analysis (DSA) confirmed the robustness of the results. The probabilistic sensitivity analysis (PSA), undertaken with 1000 iterations, indicated a 50% and 83% probability of difelikefalin being cost-effective at a willingness-to-pay (WTP) thresholds of 25,000 €/QALY and 30,000 €/QALY, respectively.

Conclusions: Difelikefalin could be a cost-effective option compared to BSC for the management of CKD-aP in adult haemodialysis patients within the Spanish NHS setting. Considering the unmet needs, these results support the convenience of incorporating difelikefalin in routine clinical practice in Spain.