Review and comparison of methodologies for indirect comparison of clinical trial results: an illustration with ranibizumab and aflibercept

Expert Rev Pharmacoecon Outcomes Res. 2016 Dec;16(6):793-801. doi: 10.1586/14737167.2016.1165609. Epub 2016 Mar 25.

Abstract

Aim: To review and compare methods for indirect comparison of aflibercept and ranibizumab in patients with diabetic macular edema.

Methods: Post-stratification, inverse probability weighting based on simulated data, weight optimization, and regression model techniques were used to compare pooled individual patient-level data from the RESTORE and RESPOND (ranibizumab 0.5 mg as needed after 3 initial monthly doses) studies with summary-level data from the VIVID and VISTA (aflibercept 2.0 mg every 8 weeks after 5 initial monthly doses, 2q8) studies. The impact of adjusting for up to two baseline characteristics was assessed.

Results: All methods provided similar results. After adjustment for baseline best-corrected visual acuity and central retinal thickness, no statistically significant difference in average gain in baseline best-corrected visual acuity from baseline at month 12 was found between ranibizumab 0.5 mg and aflibercept 2q8.

Conclusions: Weight optimization and regression methods are useful options to adjust for more than one baseline characteristic.

Keywords: Confounders; indirect treatment comparison; inverse probability weighting; regression; stratified sampling.

Publication types

  • Comparative Study
  • Meta-Analysis

MeSH terms

  • Angiogenesis Inhibitors / therapeutic use
  • Clinical Trials as Topic / methods
  • Diabetic Retinopathy / complications
  • Diabetic Retinopathy / drug therapy*
  • Humans
  • Macular Edema / drug therapy*
  • Macular Edema / etiology
  • Models, Statistical
  • Ranibizumab / therapeutic use*
  • Receptors, Vascular Endothelial Growth Factor / therapeutic use*
  • Recombinant Fusion Proteins / therapeutic use*
  • Regression Analysis
  • Treatment Outcome
  • Visual Acuity / drug effects

Substances

  • Angiogenesis Inhibitors
  • Recombinant Fusion Proteins
  • aflibercept
  • Receptors, Vascular Endothelial Growth Factor
  • Ranibizumab