Economic evaluation of type 2 diabetes mellitus T2DM pharmaceutical treatment compared to an alternative, conducted alongside randomized controlled trials: A scoping review

Diabetes mellitus is recognized as one of the most challenging health conditions facing all healthcare systems around the world and poses a high burden on individuals and society. Prevention of type 2 diabetes via screening programs and accessible, safe, and effective treatments would benefit people who might otherwise suffer decades of drug therapy and disease-related complications, leading to premature mortality, preventable morbidity, and significant economic burden. This study aimed to systematically map existing research on the cost-effectiveness of T2DM pharmaceutical treatment conducted alongside randomized controlled trials (RCT).

This scoping review was carried out according to the "Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews" and "Consolidated Health Economic Evaluation Reporting Standards 2022 (CHEERS 2022) " checklists. Medline, Health & Medical Complete (ProQuest), Cochrane Central Register of Controlled Trials (CENTRAL), and Tufts Global Health Cost Effectiveness Analysis (GH CEA) Registries were searched from January 2010 to January 2023.

205 records were initially identified, and 21 records were selected for full-text review, resulting in the extraction and analysis of data from 13 articles, including 15 studies. Of the included studies, 53% of economic evaluations have been carried out in Europe (Spain, United Kingdom, Sweden and the Netherlands), 27% United States and 20% in China. Two-thirds of included studies used the payer’s perspective, three studies adopted a healthcare system perspective, one study used a societal perspective and one did not provide information on the perspective chosen. With the exception of three studies, the remaining investigations were predicated upon a lifetime model.13 studies identify the relevant pharmaceutical treatments are cost-effective. In contrast, one study’s result showed that the insulin degludec/liraglutide versus its monotherapy is not cost-effective, and one study deduced that the short-term cost-effectiveness of one-daily liraglutide vs once-weekly exenatide highly depends on the selected source of the clinical data. Among the 12 studies that conducted a sensitivity analysis, three studies employed probabilistic sensitivity analysis, three studies utilized univariate sensitivity analysis, and six studies employed both approaches. The medication cost emerged as the most frequently identified key driver of the outcome.

This review may help inform decisions about investment, research, and development of type 2 diabetes pharmaceutical treatment. However, it also clearly demonstrates significant gaps in local evidence in this area and could be used to support ongoing research on this topic.