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Management of Neovascular Age-related Macular Degeneration: Systematic Drug Class Review and Economic Evaluation

Technologies

Single and combination therapy with photodynamic therapy using verteporfin (V-PDT); anti-vascular endothelial growth factor (anti-VEGF) therapies, including pegaptanib, ranibizumab, and bevacizumab; and steroids or analogues, anecortave acetate and triamcinolone.

Condition

Adults 40 years of age or older with neovascular age-related macular degeneration (AMD).

Issue

The recent emergence of several pharmacologic therapies for AMD has led to uncertainty regarding the impact of single and combination therapies as well as the optimal timing of therapy and the impact of re-treatment.

Methods and Results

Eighteen articles describing nine unique randomized trials, one controlled trial, and five case series were identified through a systematic literature review. Two cost-utility analyses in adults 40 years of age and older from the perspective of the Canadian public health care system and a patient lifetime time horizon were conducted. Pegaptanib, ranibizumab, and V-PDT were compared for predominantly classic lesions, and pegaptanib and ranibizumab were compared for all neovascular lesions. An analysis of budget impact and ethical and psychosocial issues was also conducted.

Implications for Decision Making

· Uncertainty still remains. No direct evidence demonstrating the effect of timing or re-treatment on health was found. There was insufficient evidence to suggest whether combination therapy (or which combinations) is better than monotherapy. Evidence for bevacizumab’s effectiveness is less compelling than other anti-VEGF agents.

· Pegaptanib or ranibizumab represent optimal treatment strategies. Pegaptanib is the least costly strategy, and ranibizumab would be likely to be the most cost-effective strategy for those willing to pay more than an additional $59,000/quality-adjusted life year. These results are most sensitive to the cost of ranibizumab therapy and change in visual acuity. At its current price, bevacizumab is likely to be the most cost-effective strategy if it is more effective than V-PDT.

· Access, equity, and legal issues remain. Access is of issue in Canada and the UK as existing systems are over extended in meeting resource needs to achieve early referral, diagnosis, and treatment within an effective therapeutic window. Equity issues are encountered as patients incur the costs of treatment at private clinics. Continued off-label use of bevacizumab raises ethical, legal, equity, and policy implications.

This summary is based on a comprehensive health technology assessment available from CADTH’s web site (www.cadth.ca): Brown A, Hodge W, Cruess A, Blackhouse G, Hopkins R, McGahan L, Sharma S, Pan I, Blair J, Vollman D, Morrison A. Management of Neovascular Age-related Macular Degeneration: Systematic Drug Class Review and Economic Evaluation.