Joint Assessment of Intended and Unintended Effects of Medications: An Example Using Vascular Endothelial Growth Factor Inhibitors for Neovascular Age-Related Macular Degeneration

Table 1

Model inputs, intended and unintended effects.

Intended Effects	Pegaptanib (𝑛=294)^1𝑛(%)	Ranibizumab (𝑛=379)^2𝑛(%)	UC Combined (𝑛=677)^3𝑛(%)
Loss of ≥6 lines ETDRS VA	28 (9.5)	3 (0.8)	118 (39.9)
Loss of ≥3 but <6 lines ETDRS VA	60 (20.4)	15 (4.0)	153 (51.7)
Loss of >0 but <3 lines ETDRS VA	108 (36.7)	83 (21.9)	227 (76.7)
Gain of ≥0 but <3 lines ETDRS VA	80 (27.2)	140 (36.9)	153 (51.7)
Gain of ≥3 lines ETDRS VA	18 (6.1)	138 (36.4)	26 (8.8)
Unintended Effects
Nonfatal MI, % (SE)	0.3 (0.378)	1.1 (0.525)	0.6 (0.295)
RR (95% CI) Nonfatal MI, versus UC	0.5 (0.0–5.5)	2.0 (0.4–10.9)	—
Nonfatal CVA, % (SE)	1.0 (0.586)	1.1 (0.525)	0.4 (0.255)
RR (95% CI) Nonfatal CVA, versus UC	3.0 (0.3–28.9)	2.0 (0.4–10.9)	—
Nonfatal severe NOH, % (SE)	1.0 (0.653)	1.5 (0.614)	0.6 (0.295)
RR (95% CI) Nonfatal severe NOH, versus UC	1.0 (0.2–4.9)	5.5 (0.7–46.3)	—

¹From VISION Study [6] of 0.3 mg pegaptanib, and VISION-study safety and efficacy summaries provided by Pfizer Inc.
²Pooled estimates from MARINA and ANCHOR [7, 8] studies of 0.5 mg ranibizumab.
³Pooled estimates from the control arms of the VISION [6], ANCHOR and MARINA [7, 8] studies.
ETDRS VA: Early Treatment of Diabetic Retinopathy Study visual acuity; MI: myocardial infarction; SE-standard error; 95%CI: 95% confidence interval; UC: Usual care; NOH: nonocular hemorrhage.