No one would dispute that off-label use of bevacizumab to treat neovascular age-related macular degeneration (nAMD) is more cost effective than either of the branded agents, Lucentis (ranibizumab) and Eylea (aflibercept). Indeed, that is its sole advantage and impetus for use. But good data is harder to come by for more sophisticated comparisons; for instance, those involving dosing regimens or the relative performance of the two branded drugs. A recent study found the majority of published cost-effectiveness analyses (CEAs) comparing the various treatments are of insufficient quality and validity. Consequently, they wrongly inform decision-makers of cost comparisons and negatively influence overall patient outcomes and societal costs.¹

Researchers reviewed 48 CEAs, only four of which met quality and validity standards. The remaining analyses didn’t meet the four basic criteria specific to studies of this type: both eyes evaluated instead of one, a timeframe beyond four years, visual acuity extrapolation and treatment intervals beyond trial data, and the costs of low vision.¹

In half of the CEAs that aligned with quality and validity criteria, bevacizumab PRN was more cost-effective than all other regimens (i.e., bevacizumab monthly, aflibercept and ranibizumab monthly or PRN). In two of the analyses—which centered on the branded agents—ranibizumab outperformed aflibercept both monthly and PRN. In two others, however, aflibercept was either more cost-effective or dominant, indicating “either treatment can be more cost-effective depending on the assumptions used for drug prices and treatment frequencies.”¹

Given the dearth of adequate CEAs, researchers suggest “for future ophthalmic treatments, CEAs need to be improved and only published when they are of sufficient quality and validity.”¹ 

“A valid and useful CEA is important because the government or the third-party insurer may decide to discontinue reimbursement if CEA shows that the treatment is not cost-effective,” said Weiye Li, MD, in a Practice Update commentary on the study. “Because CEA is so critical for whether any new medications for nAMD will be supported or denied, much higher-quality and truer-validity studies are imperative.”²