Análogos de insulina humana versus insulina NPH para o tratamento do diabetes mellitus tipo 1: uma avaliação econômica na perspectiva do Sistema Único de Saúde.

Abstract

Type 1 diabetes mellitus (T1DM) is a chronic and costly disease both for people who live with it and for governments and society. The economic burden of T1DM is largely due to drug costs and diabetes complications. After the diagnosis of T1DM, treatment must be started immediately, as the lack of endogenous insulin can cause diabetic ketoacidosis. Currently, the Unified Health System (SUS) provides neutral protamine Hagedorn (NPH) as a first-line treatment for patients with T1DM and, as a second-line treatment, the human insulin analogues glargine (IGla U100 and IGla U300), detemir (IDet) and degludec (IDeg). Although some studies show favorable results for insulin analogues, uncertainties remain in terms of health-related quality of life (HRQoL) and cost-utility in relation to NPH insulin, especially in the context of Brazil and SUS. In this sense, the main goal of this thesis was to economically evaluate, from the perspective of SUS, the treatment with human insulin analogues (IGla U100, IGla U300, IDet and IDeg) in comparison to NPH insulin for the treatment of people living with T1DM. In the first stage of the thesis, two cross-sectional studies were carried out that analyzed and measured which factors were associated with the HRQoL of patients treated with IGla U100 (N = 401) and with NPH insulin (N = 179). The results for the two multiple linear regressions of the two cross-sectional studies demonstrated that a higher HRQoL was associated with being young; have higher education; having self-rated health as good or very good; not having been bedridden and having practiced physical activity in the last 15 days; having performed up to three medical consultations in the last year; not having been hospitalized in the last year; have zero to three comorbidities; not having angina, diabetic neuropathy, hearing loss, kidney disease, systemic arterial hypertension or chronic obstructive pulmonary disease; have few, non-severe episodes of hypoglycemia. Additionally, the results of the two studies suggested a barrier to accessing drugs from the Specialized Component of Pharmaceutical Assistance in the SUS, since patients treated with IGla U100 had a higher socioeconomic level when compared to patients treated with NPH insulin. In the second stage of the thesis, two other studies were carried out, one being a systematic review of economic evaluations and a cost-utility analysis (CUA) of the insulins available on SUS (i.e., NPH, IGla U100, IGla U300, IDet and IDeg) for patients with T1DM. The findings of the systematic review demonstrated a low standardization in studies of economic evaluations, lack of transparency of the parameters used to feed the models and the non-use of checklists for the description of the works. Furthermore, incremental cost-effectiveness ratio results generally favored trial sponsors. From the CUA point of view, the effectiveness results, in terms of quality-adjusted life years, pointed to a similarity between the analogues and NPH insulin, with the incremental cost being the main difference between the treatments. This thesis provided theoretical support to deepen knowledge about HRQoL and the cost-utility of insulin therapy for people living with T1DM in Brazil, especially SUS users. Finally, the two cross-sectional studies made it possible to understand which variables had the greatest impact on the HRQoL of patients with T1DM treated with IGla U100 or with NPH insulin in the SUS. In addition, the CUA results pointed to a similarity between the analogues (i.e., IGla U100, IGla U300, IDeg, and IDet) and NPH insulin, with incremental cost being the main difference between the technologies.