THE COST-EFFECTIVENESS OF TREATMENT OF NEOVASCULAR MACULAR DEGENERATION

Purpose: Neovascular age-related macular degeneration (AMD) secondary to choroidal neovascularization (CNV) is the leading cause of blindness in the United States. Photodynamic therapy (PDT), has been shown to slow disease progression; as has the anti-vascular endothelial growth factor (VEGF) therapy, pegaptanib (Macugen^®). A second VEGF therapy, ranibizumab (Lucentis^®) has been shown to improve visual acuity. Treatment of all people in the U.S. with neovascular AMD using ranibizumab would cost U.S. payors in excess of $2 billion/annually. We examined the cost-effectiveness of these treatments for AMD.
Methods: We constructed a Markov decision analytic model and evaluated it using a microsimulation approach. The model was populated with data major randomized clinical trials of treatment of neovascular AMD. As PDT has been show to only be effective in treatment of predominantly classic (PC) lesions, we examined treatment of people with PC lesions in one model, and treatment of those with any lesion separately. The model for treatment of PC lesions compared treatment with PDT, pegaptanib, and ranibizumab; and the second considered only pegaptanib and ranibizumab. We report the incremental cost-effectiveness ratios (ICER) for both models. The influence of changes in model parameters was evaluated using one-way sensitivity analysis and probabilistic sensitivity analysis. Results: In the model for treatment of PC lesions only, PDT was dominated by pegaptanib. However, only ranibizumab resulted in improved visual acuity from treatment. In the PC model, treatment with ranibizumab resulted in an average gain of 0.42 quality-adjusted life years (QALYs) over the lifetime, with an ICER of $106,674/QALY. In the any CNV lesion model, the treatment with ranibizumab resulted in a gain 0.52 QALYs over the patient's lifetime with an ICER of $106,683/QALY. In one-way sensitivity analysis, the cost of ranibizumab and the change in visual acuity following treatment were found to result in a change in the cost-effectiveness decision. In probabilistic sensitivity analyses, ranibizumab was the most cost-effective strategy more than 70% of the time if the societal willingness to pay was in excess of $125,000/QALY.
Conclusions: Of the three treatments for neovascular macular degeneration examined (i.e., PDT, pegaptanib, and ranibizumab), only ranibizumab results in improvement of visual acuity and quality of life. However, our analyses indicate that the premium required for this medication exceeds most commonly accepted standards of cost-effectiveness.