Presentation (CTI)

OBJECTIVES: Many therapies have recently launched in late-line hemato-oncology indications. Barring one-off therapies, most are used “treat-to-progression”; however, some use a different treatment approach, stipulating maximum cycle number. This research sought to understand the impact of this difference for HTA.
METHODS: Two bispecific antibody therapies with maximum cycle number, approved since 2022, were identified and assessments compared with five examples of “treat-to-progression” therapies in the same indications and/or drug class. Published HTA documents from agencies in Europe and Canada were reviewed to identify value assessment and critique relevant to treatment approach.
RESULTS: Patient benefit associated with a fixed duration was suggested in UK and Spanish assessments, with reference to limiting treatment burden and toxicity; no clinical or economic value of a fixed duration was otherwise evidenced as recognized. The impact of treatment approach on total costs of these therapies, as considered in economic analyses, appears limited: calculated costs reflect poor response rates and early discontinuation in many patients in late-line indications regardless of treatment approach, and commonly higher-dose, and hence higher-cost, initial cycles versus lower dosing in later cycles. Trials of fixed duration therapies used trial data directly for treatment continuation and hence therapy cost in economic analyses. In contrast, HTA agency critique on model inputs for time-to-discontinuation was common for agents used as treat-to-progression: this included extrapolation of trial data and relationship to PFS. Further, stopping rules were considered in evaluations of three therapies, with uncertainties raised around plausibility in clinical practice, post-stopping treatment benefit, and relationship to functional cure assumptions.
CONCLUSIONS: Treatment approach appears to have limited impact in HTA, although suggesting issues (e.g., value of time off-treatment, stopping rules) that may be more prominent if therapies enter earlier lines. Multiple factors influence choice of treatment approach: this research highlights some considerations in anticipation of future HTA.