Comparison of Methods of Clinical Trial Emulation Utilizing Data From the Comparison of AMD Treatment Trial (CATT) and the IRIS® Registry

Abstract

PurposeWe used exact matching and inverse propensity score weighting (IPSW) using real-world data (RWD) from the American Academy of Ophthalmology IRIS® Registry (Intelligent Research in Sight) to emulate the two pro re nata (prn) treatment arms from the Comparison of AMD Treatment Trial (CATT) and to compare the outcomes of the RWD arms to the two monthly treatment arms from the clinical trial. DesignRetrospective cohort study utilizing deidentified electronic health record registry data and patient-level deidentified clinical trial data SubjectsAll treatment-naive patient eyes with neovascular age-related macular degeneration treated with ranibizumab or bevacizumab only for one year from either the CATT or the IRIS Registry MethodsPatients were identified in the IRIS Registry between October 1, 2015 and December 31, 2019. After all non-imaging based inclusion and exclusion criteria from the CATT were applied, patient eyes receiving bevacizumab or ranibizumab only on a prn basis were identified as the eligible cohort. Exact matching and ISPW was applied based on age, gender, and baseline visual acuity. ResultsWe identified 427 eyes treated with ranibizumab prn and 771 eyes treated with bevacizumab prn. Using exact matching, 98% (n= 281) of CATT patient eyes in the bevacizumab monthly treatment arm and 87% (n=261) of CATT patient eyes in the ranibizumab monthly treatment arm were matched to a patient eye in the IRIS Registry. For the ranibizumab prn treatment arm, patient eyes generated using exact matching gained 1.9 letters and those generated using IPSW gained 2.8 letters (Exact Matching: 1.9 letters +/- 14.0 vs. IPSW: 2.8 letters +/- 15.0 letters, p=0.43). For the bevacizumab prn treatment arm, patient eyes generated using exact matching gained 2.4 letters and those generated using IPSW gained 2.1 letters (Exact Matching: 2.4 letters +/- 15.4 vs. IPSW: 2.1 letters +/- 16.0 letters, p=0.79). ConclusionsBoth exact matching and IPSW produced similar results in emulating the prn treatment arms of the CATT using IRIS Registry data and patient-level clinical trial data. Similar to prior real-world studies, the clinical outcomes were significantly worse in the IRIS Registry treatment arms compared to the clinical trial.