The disease burden of paroxysmal nocturnal hemoglobinuria in Denmark: Epidemiology, survival, healthcare resource utilization, costs, treatment gaps, and labor market attachment

Objectives To establish epidemiology, healthcare costs, and labor market attachment in patients with paroxysmal nocturnal hemoglobinuria (Pt‐PNH) in Denmark. Methods Data were from Statistics Denmark and the Danish Health Data Authority national population registers (2005–2021). Descriptive baseline statistics characterized the Pt‐PNH analytic population; ordinary least squares and adjusted Cox proportional hazards regressions measured outcomes in the Pt‐PNH versus Danish general population matched comparators. Results Overall PNH incidence in Denmark was n = 11 during 2007–2009, n = 25 during 2016–2018 and n = 7 during 2019–2020; prevalence increased from n = 13 in 2006 to n = 62 in 2021. Of the overall n = 85 Pt‐PNH; n = 24 were treated with complement‐5 inhibitors (Pt‐C5i) and n = 61 not treated with C5i (Pt‐nC5i). Versus respective comparators, all patients had significantly greater annual per‐patient costs (from inpatient hospital admissions, outpatient contacts, PNH treatments; indirect costs from lost earnings + transfer payments; post‐diagnosis for Pt‐PNH and Pt‐nC5i, post‐treatment initiation for Pt‐C5i). The Pt‐C5i incurred the greatest healthcare and indirect cost differences (€709 119; €152 832, respectively) followed by the Pt‐PNH (€189 323; €29 159, respectively) and Pt‐nC5i (€95 548; €4713, respectively). The Pt‐PNH versus comparators also had an increased hazard of death (2.71 [95% CI, 1.63 − 4.51]). Conclusion Although a rare disease, PNH is associated with significant patient, healthcare system, and societal burdens in Denmark.