Lifetime Economic Burden of Hemophilia Using Phase-Based Costing and Survival Estimation Through Rolling Extrapolation: An Analysis of South Korean Nationwide Administrative Data

OBJECTIVES: Hemophilia treatment is initiated at an early age and continued for a lifetime, but there is not enough evidence about the lifetime economic burden of hemophilia. we aim to identify the real-world lifetime cost of hemophilia, considering cost changes by disease phase and time.

METHODS: Male patients with hemophilia A(Ha) or B(Hb) in the 2007-2022 HIRA database of South Korea were included. Life expectancy was obtained through the rolling extrapolation method. Phase-specific costs(before hemophilic arthropathy [BH], after hemophilic arthropathy [AH], 1 year before death [BD]) were analyzed for phase-based costing. Transition probability from BH to AH was calculated from the incidence rate of hemophilic arthropathy. The observed phase-specific annual cost of each year was used to fit generalized estimating equations. Lifetime cost was estimated by summing up the predicted phase-specific annual costs multiplied by the probability of the corresponding phase throughout the estimated life expectancy.

RESULTS: Life expectancy of Ha(n=2,624) and Hb(n=664) was 76.13 and 77.54 years. With an incidence rate of hemophilic arthropathy (Ha: 0.090 case/person-year [95%CI, 0.084-0.097]; Hb: 0.080 [0.070-0.092]), the annual transition probability from BH to AH was estimated at 0.086(Ha) and 0.077(Hb). Mean phase-specific annual cost was highest in BD followed by AH and BH in both Ha(BH: $11,331; AH: $27,271; BD: $27,985) and Hb(BH: $15,567; AH: $38,659; BD: $55,985). Ha had significantly higher mean phase-specific annual cost than Hb with 1.37 and 1.42 times higher AH and BH costs, respectively(p<0.05). Lifetime cost was 1.76 times higher in Hb($22,250,382) than Ha($12,624,218) in hypothetical patients born in 2000.

CONCLUSIONS: Lifetime cost was higher in Hb than in Ha, which was 240 and 136 times the average lifetime healthcare expenditure per capita in South Korea. The estimated lifetime cost of hemophilia in our study would aid developers, manufacturers, and regulatory bodies in identifying the value of new treatments for hemophilia.