Pro30 Economic Burden of Pompe Disease in the Russian Federation

Abstract

Objectives: To assess the burden of cystic fibrosis (CF), a rare, progressive genetic disease, in infants and children aged ,6 years in the US by comparing their health care resource utilization (HCRU) with that of matched individuals without CF. Methods: In a retrospective study using US commercial and Medicaid claims databases (2010)(2011)(2012)(2013)(2014)(2015)(2016)(2017), patients aged ,6 years with CF were matched 1:3 to individuals without CF by age, gender, geographic region, race, enrollment year, and insurance plan type. 1-year HCRU outcomes, including hospitalizations and outpatient medication use, were compared between patients with CF and controls using bivariate statistics for all patients and by age group (infants [aged ,2 years] and children aged 2-,6 years). P values ,0.05 were considered significant; no multiplicity adjustment was conducted. Results: 835 patients with CF were matched to 2505 controls in the commercial population; 735 patients with CF were matched to 2205 controls in the Medicaid population. Significantly higher HCRU was observed among patients with CF, and more were hospitalized annually versus controls (commercially insured: 15.4% vs. 5.4%; Medicaid-insured: 24.6% vs. 3.1%). Mean annual hospitalization rates were up to 4-fold higher with CF versus controls in the commercially insured population (0.25 vs. 0.06) and 10-fold higher with CF versus controls in the Medicaidinsured population (0.41 vs. 0.04). Similar patterns were observed within age subgroups, where infants and children aged ,6 years with CF had significantly higher hospitalization rates versus controls. On average, commercially insured patients aged ,6 years with CF had 31.4 total prescription fills/year versus 3.5 for controls; Medicaid-insured patients aged ,6 years with CF had 40.9 total prescription fills/ year versus 7.0 for controls. P values across all comparisons were ,0.05. Conclusions: Infants and children aged ,6 years with CF have substantial disease burden with significantly greater hospitalizations and outpatient medication use versus matched controls.

Evaluation of orphan diseases global burden

“…The cost varies depending on the drug, weight, type of disease and the presence of complications. 8 Kolbin A.S. et al (2018) 9 provides figures for the cost of treating Pompe disease. The cost of providing patients with the specific drug alglucosidase alfa as enzyme replacement therapy for a child is 10131952 rubles or 131264 USD per year per patient, and for an adult 34596 912 rubles or 448219 USD per year per patient.…”

“…On the contrary, the increase in the costs of diagnostic and rehabilitation measures in patients on symptomatic therapy determines a significant burden on the budget of medical institutions, given the severity of the course and the deterioration of patients' condition. 9 Moreover, this is just a small fraction of the numbers and needs of patients with rare diseases. According to various estimates, from 15000 to 1.5 million people in Russia suffer from orphan pathology.…”