OBJECTIVES: The objective of this study is to present results of a Quebec/Canadian adaptation of a cost-effectiveness analysis of tolvaptan, the first Health Canada approved treatment for autosomal dominant polycystic kidney disease (ADPKD), versus best supportive care (BSC).

METHODS: The model uses patient level data from the TEMPO 3:4 trial placebo arm to estimate annual rates of ADPKD progression based on association between total kidney volume and renal function. The model was validated against observed progression in the REPRISE study. Quality of life was based on a disutility relative to general population approach. Costs included direct medical costs and societal costs from lost productivity; taken from Quebec/Canadian sources. A discount rate of 1.5% (costs and utilities) was used, analyses were undertaken from a societal perspective. The population reflected both TEMPO 3:4 and REPRISE populations, with treatment effect derived from TEMPO 3:4.

RESULTS: Over a lifetime horizon (80 years), patients in the tolvaptan arm progressed through ADPKD slower compared to BSC, the tolvaptan arm experienced delayed ESRD onset by 3 years, with a cost savings of $70,058 per patient. The incremental costs and QALYs gained with tolvaptan treatment per patient were estimated at $204,810 and 1.12 respectively, with an incremental cost utility ratio of $182,868/QALY. In the BSC arm, 78.7% of patients reach ESRD in 10 years compared to 17.4% in the tolvaptan arm. Results from this model indicate that a 31.6% reduction using 1/serum creatinine in rate of renal function decline, as observed both the model and the TEMPO 3:4 trial, would delay the time to ESRD and death by 3.03 and 1.58 years, respectively.

CONCLUSIONS: The results of this analysis suggest that tolvaptan represents a cost-effective treatment for delaying the progression of ADPKD, with significant healthcare system cost savings potential by delaying and reducing the need for costly interventions involved with ESRD.