Multiple myeloma (MM) remains a complex and incurable hematologic malignancy, despite the rapid evolution of therapeutic options over the past decade. With the emergence of novel drug classes—ranging from monoclonal antibody-drug conjugates to CAR-T cell therapies—the treatment landscape has become increasingly intricate, making direct comparisons between regimens challenging. In the absence of head-to-head randomized controlled trials (RCTs), indirect treatment comparisons (ITCs) and population-adjusted indirect comparisons (PAICs) have become essential tools for evaluating relative effectiveness. However, the validity of these comparisons hinges on the accurate identification and adjustment of treatment effect modifiers (TEMs) and prognostic factors (PFs).
To address this need, the Value & Evidence team at EVERSANA (Karen Chiang and Imtiaz A. Samjoo), in collaboration with the team at Johnson & Johnson Innovative Medicine (Jianming He, Xiwu Lin, Winghan Jacqueline Kwong, John Maringwa, Lingfeng Yang, Sandhya Nair, Mahmoud Hashim, Mi Jun Keng), conducted a literature review of RCTs and PAICs across newly diagnosed and relapsed/refractory MM populations. The goal was to identify key baseline covariates that influence treatment outcomes—specifically progression-free survival and overall survival—and to determine which variables should be prioritized in future clinical trial designs and comparative analyses.
This research not only provides a statistically grounded framework for identifying TEMs and PFs but also offers a practical roadmap for improving the transparency and consistency of future ITCs and PAICs. The insights from this study can inform preplanned subgroup and stratification analyses in RCTs, guide covariate selection in PAICs, and ultimately support more personalized and evidence-based treatment strategies for patients living with MM.
For a deeper dive into the methodology and findings, access the full article in Journal of Comparative Effectiveness Research.
Author
Karen is a Manager on the Value & Evidence team at EVERSANA and has over five years of experience on evidence synthesis projects. At EVERSANA, Karen has contributed to the development of health economic…
As Vice President of the Value & Evidence team at EVERSANA®, Imtiaz leads evidence synthesis projects that support global HEOR initiatives involving systematic literature reviews, indirect treatment comparisons, and health economic modelling, to support reimbursement and market access for pharmaceuticals. Imtiaz…