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Table 1 MARINA study clinical and cost-utility analysis parameters

From: The comparative effectiveness and cost-effectiveness of ranibizumab for neovascular macular degeneration revisited

Clinical features [1]

 Each participant had minimally classic or occult, subfoveal choroidal neovascularization

 Best corrected ETDRS entrance vision in the affected eye: 20/40–20/320

 Choroidal neovascular lesions <12 disc areas at baseline

 Baseline vision: mean 20/80 − 1 in both the ranibizumab treatment and sham treatment cohorts

 Mean baseline age: 77 years

 Treatment protocol: Participants were randomized equally to: (1) a 0.5 mg intravitreal ranibizumab dose cohort (n = 240), (2) a 0.3 mg intravitreal ranibizumab dose cohort (n = 238) or (3) a sham injection treatment cohort (n = 238)

 Only data from the 0.5 mg ranibizumab cohort (0.5 mg was the dose eventually approved by the Food & Drug Administration [5]) and the sham treatment control cohort were utilized in the cost-utility analysis herein

 The average participant received 22 × 0.05 cc intravitreal injections, given approximately monthly, over 2 years

Cost-utility analysis assumptions

 Mean life expectancy: 12 years for the control and ranibizumab study cohorts [9]

 12-year time span for model utilizing 2-year MARINA data from the sham treatment control group and the 0.05 mg ranibizumab treatment group

 Combined-eye model [14, 15]

 Societal and 3rd party insurer cost perspectives

 Cost basis: average 2015, national, Medicare Fee Schedule

 Vision utilities (based upon visual acuity in the better-seeing eye) [24,25,26,27,28,29,30,31]

 Vision

Utility

  20/20 OU

0.97

  20/40

0.80

  20/80

0.701

  20/200

0.62

  20/640

0.538

 Sham treatment, control cohort data utilized: mean vision in MARINA Study for years 1 and 2; Lineweaver-Burke plot meta-analysis [20] control cohort for years 3–12

 Treatment cohort (0.5 mg ranibizumab) mean vision: MARINA Study for years 1 and 2, HORIZON open-label extension trial for 25–49 months, LOCF (last observation carried forward) for months 49–144 [23]

 Net Present Value (NPV) analysis discounts value outcomes and costs at a 3% annual rate, as per the Panel on Cost-Effectiveness in Health and Medicine [13, 18]

 Adverse events as previously listed [9]

 Patient utilities as previously listed [9]

  1. MARINA minimally classic/occult trial of the Anti-VEGF antibody ranibizumab in the treatment of neovascular age-related macular degeneration (AMD), ETDRS early treatment diabetic retinopathy study