Cost-Utility of Acromegaly Pharmacological Treatments in a French Context

Objective Efficacy of pharmacological treatments for acromegaly has been assessed in many clinical or real-world studies but no study was interested in economics evaluation of these treatments in France. Therefore, the objective of this study was to estimate the cost-utility of second-line pharmacological treatments in acromegaly patients. Methods A Markov model was developed to follow a cohort of 1,000 patients for a lifetime horizon. First-generation somatostatin analogues (FGSA), pegvisomant, pasireotide and pegvisomant combined with FGSA (off label) were compared. Efficacy was defined as the normalization of insulin-like growth factor-1 (IGF-1) concentration and was obtained from pivotal trials and adjusted by a network meta-analysis. Costs data were obtained from French databases and literature. Utilities from the literature were used to estimate quality-adjusted life year (QALY). Results The incremental cost-utility ratios (ICUR) of treatments compared to FGSA were estimated to be 562,463 euro per QALY gained for pasireotide, 171,332 euro per QALY gained for pegvisomant, and 186,242 euro per QALY gained for pegvisomant + FGSA. Pasireotide seems to be the least cost-efficient treatment. Sensitivity analyses showed the robustness of the results. Conclusion FGSA, pegvisomant and pegvisomant + FGSA were on the cost-effective frontier, therefore, depending on the willingness-to-pay for an additional QALY, they are the most cost-effective treatments. This medico-economic analysis highlighted the consistency of the efficiency results with the efficacy results assessed in the pivotal trials. However, most recent treatment guidelines recommend an individualized treatment strategy based on the patient and disease profile.

Automated summary

The study aimed to estimate the cost-utility of second-line pharmacological treatments in acromegaly patients. The incremental cost-utility ratios for pasireotide, pegvisomant, and pegvisomant + FGSA were estimated, showing pasireotide to be the least cost-efficient treatment option.