RESUMO
CONTEXT: Autoimmune polyendocrinopathy (AP), a chronic complex orphan disease, encompasses at least two autoimmune-induced endocrine diseases. OBJECTIVE: To estimate for the first time total, indirect and direct costs for patients with AP, as well as cost drivers. DESIGN: Cross-sectional cost of illness study. SETTING: Academic tertiary referral center for AP. PATIENTS: 146 consecutive, unselected AP patients. INTERVENTION: Interviews pertaining to patients' socioeconomic situation covered a recall period of 12 months. Both the human capital (HCA) and the friction cost approaches (FCAs) were applied as estimation methods. MAIN OUTCOME MEASURES: Direct and indirect annual costs, and sick leave and medication costs. RESULTS: AP markedly impacts healthcare expenses. Mean overall costs of AP in Germany ranged from 5 971 090 to 29 848 187 per year (HCA). Mean indirect costs ranged from 3 388 284 to 16 937 298 per year (HCA) while mean direct costs ranged from 2 582 247 to 12 908 095/year. Mean direct costs per year were 1851 in AP patients with type 1 diabetes (T1D, 76%) and 671 without T1D, which amounts to additional direct costs of 1209 for T1D when adjusting for concomitant autoimmune disease (95% CI = 1026-1393, P < 0.0001). Sick leave cost estimates for AP patients with T1D exceeded those without T1D by 70% (FCA) and 43% (HCA), respectively. In multiple regression analyses, T1D predicted total and direct costs, medication costs and costs for diabetic devices (all P < 0.001). Overall, AP patients with T1D were 54% (FCA) more expensive than those without T1D. CONCLUSIONS: Public health socioeconomic relevance of AP was demonstrated, with T1D as main cost driver.
