Matching-adjusted indirect treatment comparisons in chronic inflammatory demyelinating polyneuropathy: interpreting current evidence and enhancing future studies

Aim: In the absence of direct evidence, indirect treatment comparisons (ITCs) serve as a tool for comparing treatment efficacy to guide evidence-based healthcare decisions. As an example, given the growing number of treatments available for chronic inflammatory demyelinating polyneuropathy (CIDP), it is important to accurately estimate relative treatment effects to ensure the best patient outcomes. Using CIDP as an example, the aim of this study was to review published best practices and guidelines to determine key methodological considerations for conducting robust ITCs. Materials & methods: ITCs in CIDP were identified and evaluated against best practices and guidelines, including the National Institute for Health and Care Excellence Technical Support Document 18, the Health Technology Assessment guidelines for assessment of direct and indirect comparisons, and the Preferred Reporting Items for Systematic Reviews and Meta-analyses extension statement for the reporting of systematic reviews and incorporating network meta-analyses. Results: Recently published ITCs in CIDP were limited, with two conference materials identified. Both studies utilized matching-adjusted indirect treatment comparisons to assess the comparative efficacy of CIDP therapies. As per best practices and guidelines reviewed, key methodological considerations associated with comparing CIDP pivotal trials to conduct ITCs were identified. Differences in trial target populations and baseline characteristics, heterogeneity in study designs and outcome definitions, and inappropriate pooling of therapies may limit comparability between trials if not carefully controlled for using available frameworks. Conclusion: In the absence of direct comparative evidence, ITCs are necessary and useful tools to compare treatment efficacy. Available frameworks can guide ITC feasibility and limit heterogeneity, to ensure robust and reliable ITC outcomes.