The Importance of Clinical Context and Consistency in Methodology When Using Matching-Adjusted Indirect Comparisons (MAICs) to Compare Outcomes

Katharine Batt,1,* Robert Klamroth,2,* Maria Elisa Mancuso,3,4,* Andreas Tiede,5,* Lorenzo G Mantovani6,7,* 1Department of Internal Medicine, Section of Hematology/Medical Oncology, Wake Forest University Baptist Medical Center, Winston-Salem, NC, USA; 2Department for Internal Medicine, Vascular Medicine and Coagulation Disorders, Vivantes Hospital, Friedrichshain, Berlin, Germany; 3Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Italy; 4Humanitas University, Pieve Emanuele, Milan, Italy; 5Hematology, Hemostaseology, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany; 6Center for Public Health Research, University of Milano-Bicocca, Monza, Italy; 7Value-Based Healthcare Unit, IRCCS MultiMedica Research Hospital, Sesto San Giovanni, Italy*These authors contributed equally to this workCorrespondence: Katharine Batt, Wake Forest University Baptist Medical Center, 1 Medical Center Blvd, Winston-Salem, NC, 27157, USA, Tel +1 919-593-6188, Email [email protected]: Hemophilia A is rare, which makes large, randomized, controlled, statistically driven, head-to-head comparison trials difficult. Matching-adjusted indirect comparisons (MAICs) are validated statistical tools designed to help make the results of non-comparative trials more comparable. The purpose of this commentary is to provide an insight into the MAIC method, in order to assist the hemophilia community with interpretation of MAIC data. It includes a comparison of the findings from previously published MAICs comparing recombinant factor replacement options and their methodologies. As MAICs are being used more often to compare treatment options for patients with hemophilia A, it is paramount that robust and consistent methodologies for cross-trial comparisons are used and that all efficacy analysis findings are linked to factor utilization.Keywords: hemophilia, MAIC, methodology