Cost-Utility of Acromegaly Pharmacological Treatments in a French Context

Front Endocrinol (Lausanne). 2021 Oct 7:12:745843. doi: 10.3389/fendo.2021.745843. eCollection 2021.

Abstract

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 € 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.

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.

Keywords: QALY; acromegaly; cost-utility; growth hormone; insulin-like growth factor-1; pasireotide; pegvisomant; somatostatin.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acromegaly / drug therapy*
  • Acromegaly / economics
  • Acromegaly / epidemiology
  • Aged
  • Aged, 80 and over
  • Cohort Studies
  • Cost-Benefit Analysis
  • Drug Costs* / statistics & numerical data
  • Drug Therapy, Combination / adverse effects
  • Drug Therapy, Combination / economics
  • Female
  • France / epidemiology
  • Human Growth Hormone / administration & dosage
  • Human Growth Hormone / adverse effects
  • Human Growth Hormone / analogs & derivatives
  • Human Growth Hormone / economics
  • Humans
  • Male
  • Markov Chains
  • Middle Aged
  • Network Meta-Analysis
  • Octreotide / administration & dosage
  • Octreotide / adverse effects
  • Octreotide / economics
  • Quality-Adjusted Life Years
  • Somatostatin / administration & dosage
  • Somatostatin / adverse effects
  • Somatostatin / analogs & derivatives
  • Somatostatin / economics

Substances

  • Human Growth Hormone
  • Somatostatin
  • pasireotide
  • pegvisomant
  • Octreotide