Abstract

Sacubitril/valsartan is an innovative treatment for patients with symptomatic chronic heart failure with reduced ejection fraction (HFrEF). In Portugal, as of May 2019, a total of 9,896 patients were estimated to have already started treatment with it. This study aims at estimating the projected short term societal impact to the Portuguese society of sacubitril/valsartan treatment in a real-world population of patients already being treated. A Markov model was used to describe HFrEF progression and associated lifetime outcomes. To estimate treatment effects and utilities, the model was calibrated with data from the PARADIGM-HF trial. Baseline population characteristics were adapted to match a real-world population of patients already being treated with sacubitril/valsartan (patients from an observational study conducted in Portugal, the PRiMe study). Results are expressed as hospitalizations and deaths estimated to be avoided, as well as life-years (LY) and quality adjusted life-years (QALY) estimated to be gained with sacubitril/valsartan versus enalapril. A 5-year time horizon and a 5% discount rate were considered. Assuming that the total number of patients treated with sacubitril/valsartan are persistent and that they have the same baseline characteristics as the patients recruited in the PRiMe study, after 5-years of treatment, we estimate to have avoided 2,553 hospitalizations (647 hospitalizations due to HF, 946 due to other cardiovascular events and 960 non-cardiovascular). Similarly, a total of 494 deaths are estimated to be avoided after 5-years versus a scenario with enalapril. A benefit of 1,161 LY and 1,173 QALY gained over a period of 5-years is also estimated. Sacubitril/valsartan is estimated to have a high impact on morbidity and mortality in the Portuguese population which is shown by the number of hospitalizations and deaths estimated to be avoided and LY and QALY estimated to be gained in the currently treated population.

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