🤖 AI Summary
In the absence of head-to-head trials, indirect treatment comparisons play a critical role in health technology assessment (HTA). This work proposes a methodological framework tailored to the French HTA context, developed by a working group of French experts in market access and methodology, informed by guidance from the Transparency Committee of the French National Authority for Health and systematic reviews. The framework emphasizes early planning, rigorous control of confounding factors, and explicit evaluation of similarity and transitivity assumptions. It also establishes clear criteria for the appropriate use of single-arm trials with external comparators and integrates advanced methods such as network meta-analysis, population-adjusted indirect comparisons, and non-randomized study designs. The resulting practical guidance aims to enhance the quality, robustness, and validity of indirect comparisons, thereby supporting reliable decision-making in complex healthcare settings where direct evidence is unavailable.
📝 Abstract
Context: Indirect treatment comparisons (ITC) are essential when direct head-to-head evidence is unavailable. Their reliability depends on rigorous methodological choices and careful assessment of underlying assumptions. Appropriate methodological choices can help address challenges such as cross-country variations in treatment practices, ethical constraints, and evolving treatment landscapes during trial conduct. This opinion and perspective paper provides practical guidance to strengthen the quality, robustness and accuracy of ITCs in the context of health technology assessment (HTA) in France. Methods: A panel of experts in ITCs and French market access environment developed the present strategic guidance, informed by previous work reviewing HTA methodological guidelines and complemented by a systematic review of Transparency Committee opinions from the French National Authority for Health (HAS). Results: Key considerations include early anticipation of ITCs, justification of potential confounding factors, and rigorous assessment of similarity and transitivity in randomized trial-based comparisons. In network meta-analysis, the structure of the evidence network should be adapted to the specific decision context. Population-Adjusted Indirect Comparisons require careful reporting and interpretation of the effective sample size. When evidence relies on non-randomized clinical trials, comparisons between single-arm studies and external control arms may be appropriate under different scenarios, depending on the feasibility of conducting subsequent randomized studies. Conclusions: Robust and reliable ITCs require methods consistent with the validity of their assumptions and the strength of the available evidence. This practical guidance supports the development of rigorous ITCs to inform decision-making in complex medical contexts where direct comparisons are not feasible.