OBJECTIVES: To estimate the monetized socioeconomic burden of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with C5 inhibitors (C5i) in Germany and Japan.

METHODS: An open cohort, dynamic, three-state transition model was developed in a lifetime horizon. Model outcomes linked to productivity loss were a) number of red blood cell transfusions, b) proportion of patients with moderate-to-severe fatigue, c) hospitalization days and d) time spent for C5i infusion. Data were obtained from the International PNH Registry, the most recent “Adelphi PNH Disease Specific Programme 2022 dataset”, the “German Federal Statistical Office” and the “Statistics Bureau of Japan”. Epidemiological data, patient demographics, clinical inputs, labor market and unpaid work information were also collected. The Human Capital Approach was employed to evaluate total productivity loss. All monetary inputs referred to 2022 and results were presented undiscounted according to the international literature. Scenario analysis, one-way and probabilistic sensitivity analysis were also performed to assess the robustness of results and to produce 95% Confidence Intervals (95%CI).

RESULTS: The total socioeconomic burden of PNH was estimated at €813.9m. (95%CI: €750-€930m.) and ¥242.6b. (95%CI: ¥201-¥280b.) for both countries, respectively. The average per patient-year burden was estimated at €3,890 (95%CI: €3,430-€4,365) and ¥210,178 (95%CI: ¥182,959-¥238,251), respectively. “Proportion of patients with moderate-to-severe fatigue” and “time spent for C5i infusion” were the major socioeconomic burden drivers, accounting for 52% (20%) and 38% (56%) of the total burden in Germany (Japan). Unpaid work loss represented 49.8% (42.1%) of the total productivity costs in Germany (Japan). The probabilistic analysis results varied from -20.1% (-18.7%) to 9.8% (9.9%) compared to the base case results.

CONCLUSIONS: The burden of PNH despite current treatment with C5i results in substantial productivity impact in patients in Germany and Japan. Management of patients with this rare disease should consider both PNH-disease and treatment burdens.