Comparison of Alternative Methods to Assess the Cost-Effectiveness of Tumor-Agnostic Therapies: A Triangulation Approach Using Larotrectinib as a Case Study.

OBJECTIVES: The study objective was to investigate the economic value of tumor-agnostic therapies when only single-arm effectiveness data are available at launch by applying multiple methodologies to establish comparative effectiveness. METHODS: In the absence of direct comparative data, 3 methods were used to estimate the counterfactual: (1) a historical control based on a systematic literature review for each tumor site from the larotrectinib trials, (2) an intracohort comparison using the previous line of therapy time to progression from larotrectinib trials, and (3) a nonresponder control that applied outcomes for larotrectinib nonresponders. Cost-effectiveness was modeled using the partitioned survival approach. Stochastic parameter uncertainty was assessed in a probabilistic sensitivity analysis (PSA). A triangulated estimate of the mean cost-effectiveness result was generated combining all 3 counterfactual estimates. RESULTS: Incremental cost-effectiveness ratios were similar across the 3 methodologies in the deterministic analysis ranging from £83 868 (95% uncertainty interval [UI] £65 698-£107 668) to £104 922 per quality-adjusted life-year (95% UI £80 132-£139 658). PSA results for each method substantially overlapped when plotted on the cost-effectiveness plane. Weighting PSA results for each method equally in the triangulation method produced an incremental cost-effectiveness ratios of £95 587 per quality-adjusted life-year gained (95% UI £70 449-£137 431). CONCLUSIONS: In the absence of direct comparative data, different methods of estimating a counterfactual are possible, each with strengths and limitations. Triangulating results across the methods provides a composite view of the total uncertainty and a more consistent estimation of the cost-effectiveness of the tumor-agnostic intervention compared with choosing a single method.

Cost-effectiveness Analysis of a Flash Glucose Monitoring System for Patients with Type 1 Diabetes Receiving Intensive Insulin Treatment in Sweden.

Flash glucose monitoring - an alternative to traditional self-monitoring of blood glucose (SMBG) - prevents hypoglycaemic events without impacting glycated haemoglobin (HbA1c).21 Given the potential benefits, this study assessed the cost-effectiveness of using flash monitoring versus SMBG alone in patients with type 1 diabetes (T1D) receiving intensive insulin treatment in Sweden. Methods: This study used the IQVIA CORE Diabetes Model (IQVIA CDM, v9.0) to simulate the impact of flash monitoring versus SMBG over 50 years from the Swedish societal perspective. Trial data informed cohort data, intervention effects, and resource utilisation; literature and Tåndvards-Läkemedelförmånsverket (TLV) sources informed utilities and costs. Scenario analyses explored the effect of key base case assumptions. Results: In base case analysis, direct medical costs for flash monitor use were SEK1,222,333 versus SEK989,051 for SMBG use. Flash monitoring led to 0.80 additional quality-adjusted life years (QALYs; 13.26 versus 12.46 SMBG) for an incremental cost effectiveness ratio (ICER) of SEK291,130/QALY. ICERs for all scenarios remained under SEK400,000/QALY. Conclusion: Hypoglycaemia and health utility benefits due to flash glucose monitoring may translate into economic value compared to SMBG. With robust results across scenario analyses, flash monitoring may be considered cost-effective in a Swedish population of T1D intensive insulin users.