Utility Values for a Cost-Effectiveness Model in France: A Literature Review on Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

OBJECTIVES: When submitting an efficiency dossier of a treatment to the national health authorities (HAS), the quality of the data incorporated into the modelling is crucial. Although efficacy and tolerance data are systematically collected in a clinical trial used as a pivotal trial for the model, this is not always the case for utility data. Particular utility values are therefore necessary in order to be aligned with the structure of the model, but above all with the recommendations of the HAS.

METHODS: An exhaustive literature review of quality of life data collected from patients with haematological malignancies eligible for allo-HSCT was conducted for the last 10 years. The results were then discussed by a committee of experts.

RESULTS: The research focusing on France identified 10 studies and has been complemented by a European review of the EQ-5D utilities (3 additional studies). The estimated EQ-5D utility in allo-HSCT identified in the literature is derived from a mapping of European QLQ-C30 data. It varies between 0.5 and 0.75 depending on the patient's health state following transplantation (Tremblay, 2020). This utility can be considered similar for all haematological malignancies. However, utility of allograft and autograft patients is not comparable; disutility associated with graft-versus-host disease is not negligible and varies between -0.08 and -0.2.

CONCLUSIONS: The primary point to be considered when estimating utility values is the homogeneity of sources, in order to guarantee consistency in utility variations according to the health state and/or the treatment considered. This makes the data more acceptable to the HAS and prevents the incremental cost-effectiveness ratio calculated for the treatment from being invalidated.