Economic Evaluation of Severe Anaemia: Review-Based Recommendations and a Conceptual Framework

Abstract

Objective: Novel anaemia treatments have greatly improved patient outcomes in the last decade and have also undergone economic evaluations in various settings using heterogenous model structures, costs, and inputs. The objectives were to review published economic evaluation studies in major red blood cell disorders, identify limitations in the applied methodology, provide a set of recommendations, and produce a conceptual framework for future economic research in this disease area. Methods: A targeted search was conducted for economic literature evaluating treatments in major red blood cell disorders related to anaemia. Disorders included autoimmune haemolytic anaemia, β-thalassaemia, chemotherapy-induced anaemia, anaemia in chronic kidney disease, and severe aplastic anaemia. Budget impact models and cost-effectiveness and cost-utility analyses were considered. Modelling assumptions regarding the model structure, time horizon, perspective, and type of costs were reviewed and recommendations and a conceptual framework for future economic analyses were created. Results: A total of four budget impact models, nine cost-utility analyses, and four cost-effectiveness analyses were investigated. A major limitation was that the included costs varied significantly across studies. Costs which were rarely included, and generally should be considered, were related to adverse events, mortality, and productivity. Additionally, relationships between levels of serum ferritin, hepatic or total body iron, and haemoglobin with long-term complications and mortality were rarely included. Conclusion: Published economic analyses evaluating treatments for major red blood cell disorders frequently exclude vital costs. A set of recommendations and a conceptual framework will aid researchers in applying a more comprehensive approach for economic evaluations in major red blood cell disorders.