OBJECTIVES: Primary IgA nephropathy in adults (IgAN) is a rare autoimmune disease that can lead to chronic kidney disease (CKD), end-stage renal disease (ESRD), and early death. Until recently, there were no approved therapies available. Nefecon is the development project name for a novel oral formulation of budesonide which is or will be marketed under other trade names. Nefecon is being investigated for IgAN in a two-part pivotal study (NefIgArd). The objective of this analysis was to evaluate the cost-effectiveness of Nefecon in addition to standard of care (SoC), compared to SoC alone, in primary IgAN in the United States (US) based on Part A results.

METHODS: A semi-Markov model with a lifetime horizon was constructed to estimate the cost-effectiveness of Nefecon in addition to SoC, compared to standard of care alone, from a US healthcare and societal perspective. The model structure consisted of nine health states of varying CKD severity, ESRD, and death. Transition probabilities between heath states were estimated from regression analysis of individual patient level data from Part A of the NefIgArd trial, along with literature-based sources. Costs included treatment, routine medical care, adverse events, dialysis, transplant, long-term complications, mortality, and indirect costs.

RESULTS: In the base case analysis, overall lifetime costs after Nefecon plus SoC and SoC alone were $1,209,075 and $1,205,265, respectively, generating incremental costs of $3,810. Overall quality-adjusted life years (QALYs) after Nefecon plus SoC and SoC alone were 13.43 and 13.18, respectively, generating incremental QALYs of 0.25. These results led to an incremental cost effectiveness ratio of $15,427 per QALY gained. Sensitivity analysis showed consistency with base case findings contributing to the robustness of the economic case.

CONCLUSIONS: At a willingness to pay threshold of $150,000 per QALY gained, Nefecon is likely to be a cost-effective treatment in primary IgAN in the US.