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Elizabeth A Sugar, Janet T Holbrook, John H Kempen, Alyce E Burke, Lea T Drye, Jennifer E Thorne, Thomas A Louis, Douglas A Jabs, Michael M Altaweel, Kevin Frick, Multicenter Uveitis Steroid Treatment (MUST) Trial Research Group; Incremental Cost-Effectiveness of Fluocinolone Acetonide Implant vs Systemic Therapy for Non-infectious Intermediate, Posterior, and Pan-Uveitis. Invest. Ophthalmol. Vis. Sci. 2014;55(13):5796.
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The goal of a cost-effectiveness analysis is to simultaneously compare the cost and health-utility of multiple therapies to determine which will have the best value. Such analyses are particularly important when the more expensive therapy is also more effective, as occurred in the Multicenter Uveitis Steroid Treatment (MUST) Trial comparing the fluocinolone acetonide implant to systemic therapy for patients with non-infectious intermediate, posterior, or panuveitis. We evaluated the 3-year incremental cost-effectiveness for these two treatments.
Based upon the expected lifetime of the implant, data from the first 3 years of follow-up in the MUST Trial were evaluated. Analyses were stratified by uveitis laterality (31 unilateral and 224 bilateral). Costs include medications, surgeries, hospitalizations, and standard procedures. The EQ-5D was used to evaluate health utility. The primary outcome was the incremental cost-effectiveness ration (ICER): a ratio of the difference in total costs divided by the difference in change in quality adjusted life years (QALYs). Generalized estimating equations were used to account for repeated measures when estimating the total costs and the change in QALYs. A bootstrap was used to assess the variability of the paired outcomes. The probability of being cost-effect for standard thresholds ($50,000 and $100,000/QALY) was also computed.
The ICER for bilateral disease was $301,700/QALY and the probability of being cost-effective at the $100,000/QALY threshold was 0.11. This result was driven by the high relative cost of implant therapy (Difference: $17,000, p < 0.001) and the moderate gain in QALYs (0.057, p = 0.22). In contrast, the ICER for unilateral disease was $41,200/QALY and the probability of being cost-effective at the $100,000/QALY level was 0.79. Both the cost difference ($5,300, p = 0.44) and the QALY benefit (0.130, p = 0.12) were more favorable than in the bilateral case. However, the limited sample size made the estimates more variable.
Fluocinolone acetonide implant therapy may be cost-effective compared to systemic therapy for individuals with unilateral disease but not for those with bilateral disease. Additional investigation of cost effectiveness is warranted for cases in which systemic therapy has failed or if large changes in therapy prices occur.
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