US Health Resource Utilization and Cost Burden Associated with Choroideremia

PURPOSE: Choroideremia is a progressive, inherited retinal dystrophy that leads to blindness. This study of choroideremia addresses health resource utilization (HRU) and costs from a US payor perspective using insurance claims data. The retrospective analysis used data between January 2013 and December 2018 from the IBM MarketScan Commercial, Medicare Supplemental, and Multi-State Medicaid Databases. PATIENTS AND METHODS: Patients having ≥1 claim with an International Classification of Diseases, Ninth or Tenth Edition, diagnostic code for choroideremia (363.55/H31.21) were included; a control group was matched 3:1 to the choroideremia group. Patients were followed for ≥6 months. All-cause HRU and costs were compared between cohorts using generalized linear models adjusted for Charlson Comorbidity Index. RESULTS: There were 199 and 597 patients in the choroideremia and control groups, respectively; the choroideremia group had a higher mean baseline Charlson Comorbidity Index (0.47 vs 0.26). The choroideremia group had a significantly greater mean number of hospital admissions (0.09 vs 0.06), outpatient visits (22.33 vs 11.22), and emergency department visits (0.41 vs 0.26) per patient per year than the control group. The choroideremia cohort had higher all-cause total annualized costs than the control cohort ($15,372 vs $9285), primarily driven by outpatient visits ($8306 vs $4702). This trend was observed across age categories, particularly among patients aged 20 to 44 years (choroideremia, $14,544 vs control, $5953). CONCLUSION: The choroideremia group had higher all-cause HRU and total costs versus the control group. These findings provide economic context around HRU associated with choroideremia and help assess the potential impact of novel treatments.