Cost-Utility of Acromegaly Pharmacological Treatments in a French Context.

Thierry Brue,Lucile Marié,Laurène Juban,Brigitte Delemer,Patrice Rodien,Gérald Raverot,Robin Henocque,Delphine Drui,Lara Salvi,Philippe Chanson

doi:10.3389/fendo.2021.745843

Abstract

ObjectiveEfficacy 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.MethodsA 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).ResultsThe incremental cost-utility ratios (ICUR) of treatments compared to FGSA were estimated to be 562,463 € per QALY gained for pasireotide, 171,332 € per QALY gained for pegvisomant, and 186,242 € per QALY gained for pegvisomant + FGSA. Pasireotide seems to be the least cost-efficient treatment. Sensitivity analyses showed the robustness of the results.ConclusionFGSA, 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.

Highlights

Is a rare disease characterized by progressive somatic disfigurement, mainly involving the face and extremities, together with systemic manifestations related to organ overgrowth [1]
Recent studies suggest that the prevalence of acromegaly would be around 94 cases per 100,000 inhabitants from a study performed in Belgium and 1,034 per million from a systematic biochemical study performed in Germany [9, 10]
Pasireotide is recommended in second-line treatment if the residual tumor is still present and the resection is unsuitable

Summary

Introduction

Is a rare disease characterized by progressive somatic disfigurement, mainly involving the face and extremities, together with systemic manifestations related to organ overgrowth [1]. The most common cause of the disease is the presence of a benign tumor or adenoma originating from pituitary somatotroph cells and secreting excess growth hormone (GH) [2, 3]. This excessive secretion of GH leads to a persistent elevation of IGF-1, which facilitates the growth-promoting effects of GH [4]. For patients for whom surgery is not possible or with a persistent disease, first-generation somatostatin analogs (FGSA) are recommended as first-line treatment. Adding pegvisomant to FGSA is recommended in case of clinically relevant residual tumor and pre-existing impaired glucose metabolism [11]

Methods

Discussion

Conclusion