OBJECTIVES: Intravitreal (IVT) pegcetacoplan is the first FDA-approved treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). GA is one of the leading causes of blindness. We developed a de novo cost-utility model for evaluating the treatment of GA and the impact of delaying vision loss.

METHODS: This is a health-state transition model with health states defined by best corrected visual acuity (BCVA) bands measured by Early Treatment for Diabetic Retinopathy Study (ETDRS) letters. Data from the untreated (sham) arms of 2 phase III trials of pegcetacoplan, OAKS and DERBY, were used to construct transition matrices for progression of GA disease using 3-month cycles. The primary outcome measured in the clinical trials evaluating pegcetacoplan was lesion growth. Numerous GA natural history studies provide a linear association between the aggregate rate of lesion growth and vision loss. Utility values, caregiver costs, and utilities were taken from peer-reviewed literature.

RESULTS: When incorporating the costs and utility loss of informal caregiving for those with severe or total vision loss, the incremental cost per quality-adjusted life year (QALY) gained following treatment with pegcetacoplan is $77,735 assuming a lifetime model with costs and benefits discounted at 3.5%. Similarly, treating GA before the fovea is encroached, defined as GA with lesions at least 250 μm from the foveal center, carries an incremental cost of just $55,806 per QALY gained. Thus, in both populations, estimated incremental costs per QALY gained lie below the widely accepted $100,000 cost-effectiveness threshold used in the US.

CONCLUSIONS: Pegcetacoplan is a cost-effective treatment for all patients with GA secondary to AMD in the US, but most cost effective when delivered in early stages of disease after incorporating the significant burden of reduced independence due to vision loss in old age.