OBJECTIVES : Anemia treatments have greatly improved patient life expectancy in the past decade. However, few economic studies have evaluated the budgetary impact and cost effectiveness of these new therapies. The purpose of this study is to review published economic studies in severe red blood cell disorders, including autoimmune hemolytic anemia, beta-thalassemia, chemotherapy-induced anemia, chronic kidney disease anemia, and severe aplastic anemia to inform the conceptual framework for future economic research in severe anemia.

METHODS : Economic literature for the various types of anemia was reviewed by a targeted search. The review considered cost-effectiveness analyses (CEA), cost-utility analyses (CUA), and budget impact models (BIM) and evaluated the main assumptions impacting their validity. Primarily, the assumptions assessed included the model structure (decision tree, Markov model), the time horizon, the perspective, and the included costs. Based on these assessments, an optimal conceptual framework was proposed for future economic research, accounting for the criteria used previously.

RESULTS

: A total of eleven studies were analyzed: five CEAs, four BIMs, and two CUA. The studies were predominantly from G8 countries. The time horizon varied significantly from 15 weeks to lifetime, which is important as smaller timeframes do not take into consideration the long-term impact of treatment outcomes. Additionally, the types of costs included varied among studies, and mortality costs were rarely included. Furthermore, the association between serum ferritin and mortality was neglected in most of the studies considering adverse events. Lastly, uncertainty was not always assessed properly, particularly for lifetime models.

CONCLUSIONS : The economic analyses found in red blood cell disorders seem to overlook core elements, particularly the included costs. Importantly, adverse events, administration, and mortality costs were not calculated in most of the studies. The conceptual framework proposed will help identify and reduce gaps related to economic model conceptualization in red blood cell disorders.