PURPOSE:

With an ever-increasing number of treatment options, assessing the impact of changing treatment sequences/pathways has become crucial in health technology assessment (HTA). A major challenge is the scarcity of clinical effectiveness evidence, as trials typically do not compare treatment sequences. Real-world data (RWD) offers an alternative for capturing evidence on treatment sequences but analyses require appropriate statistical methods/assumptions to address time-varying confounding and immortal time bias. Descriptive treatment pattern analyses (e.g. Sankey diagrams) are useful but do not answer the counterfactual (i.e., what-if) questions required for HTA, particularly what would have happened had patients received alternative treatment sequences and the attributable effect of different lines of treatment (LOTs).

This workshop provides an in-depth discussion on recent advances in treatment sequence evaluation in HTA, with tutorials on advanced methods for comparing treatment sequences using RWD.

DESCRIPTION:

Workshop attendees will review the latest developments in treatment sequence evaluation and be introduced to newly proposed methods using RWD. The audience will participate in real-time polling to consider the broader use of these methods in HTA. Prof. Latimer will chair the session, providing an overview (9 mins). Mr. Brooke will summarize NICE’s Pathways Project, which aimed to develop multi-use economic models that included treatment sequencing to appraise multiple LOTs within a single disease area and discuss requirements of RWD for sequencing (12 mins). Ms. Chang will provide a tutorial on using Target Trial Emulation and causal inference methods to adjust for time-varying confounding, to derive the comparative effectiveness of treatment sequences using RWD (12 mins). Dr. Adamson will address the challenges of long-term survival estimation in transportability studies due to treatment sequences and the utility of the Flatiron database for treatment sequence evaluation (12 mins). The workshop will conclude with an audience discussion on the priorities for treatment sequence research using RWD (15 mins).