| Background:In light of the escalating total health expenditure and the proportionately higher drug expenditure in comparison to developed countries in Europe and the United States,it is imperative that we address this issue with urgency.China proposed the objective of constructing a value-oriented medical service system in 2016,with the aim of effectively allocating scarce healthcare resources.Pulmonary arterial hypertension(PAH)is a progressive disease with a multifactorial and incompletely understood pathogenesis,which can ultimately lead to right heart failure and even mortality in severe cases.After treatment with targeted agents,the survival rate and quality of life of patients with PAH have been significantly improved.Targeted agents exert therapeutic effects through the pathways of endothelin-1,Nitric Oxide,and prostacyclin(PGI2),respectively.For the treatment of PAH,guidelines recommend an initial combination therapy with targeted agents that target different signaling pathways to effectively manage disease progression.The preferred combination includes endothelin receptor antagonists(ERA)and phosphodiesterase type 5 inhibitors(PDE5i).For sequential treatment,the guidelines recommend adding selexipag sequentially based on ERA combined with PDE5 i to delay disease progression.As the sole commercially available oral prostacyclin receptor agonist,there is currently a dearth of comprehensive evaluation regarding the clinical and economic disparities between selexipag and other targeted drugs,as well as the cost-effectiveness of treatment regimens involving selexipag+ERA+PDE5i in comparison to ERA+PDE5i.Objective:To facilitate clinical decision-making and inform health policy development,this study aims to compare the clinical and economic value of selexipag with other targeted agents,as well as evaluate the cost-effectiveness of sequential addition of selexipag to the basis of ERA in combination with PDE5 i.Methods:Rapid health technology assessment was utilized to assess the clinical and economic differences among targeted drugs.The health technology assessment reports,systematic reviews,and meta-analyses,as well as pharmacoeconomic evaluations of selexipag for the treatment of PAH,were identified through searches of Chinese and English databases and international health technology assessment websites.The quality of the literature included was assessed using various rating scales,and the study characteristics of the literature included were extracted.Descriptive qualitative analyses were performed to summarize the discrepancies in clinical and economic value between selexipag and other targeted agents for treating PAH,based on findings from various literature studies.The cost-utility analysis is utilized in pharmacoeconomic evaluation to assess the cost-effectiveness of selexipag.This study conducted an economic evaluation from the perspective of the Chinese healthcare system.A Markov model was developed to simulate the natural progression of PAH based on the World Health Organization functional class(WHO FC)of PAH patients.The time horizon of the study was 30 years,the cycle length was 26 weeks,and the discount rate was set at 5% for health outcome and cost.The transition probability was derived from clinical trials,while the cost parameter was obtained from publicly available data in China.The evaluation measures were cumulative costs,quality-adjusted life years(QALYs),and incremental cost-effectiveness ratios(ICER).The threshold for willingness to pay(WTP)has been established at three times the per capita GDP of 2022.The ICER obtained from basic analysis was compared against the WTP threshold to assess the cost-effectiveness of various treatment regimens.Univariate sensitivity analysis,probabilistic sensitivity analysis,and scenario analysis were employed to examine the robustness of the findings.Results:The results of the r HTA indicated that selexipag exhibited an increased incidence of adverse events and discontinuations due to intolerable adverse events in terms of safety.In comparison with targeted drugs acting on the NO pathway,selexipag demonstrated a higher rate of discontinuation caused by intolerable adverse events.In terms of efficacy,selexipag has demonstrated improvements in exercise tolerance and hemodynamic parameters,as well as reductions in the incidence of clinical worsening events and hospitalization rates.However,prostacyclin analogues exhibit superior performance in improving WHO FC and reducing all-cause mortality when compared to selexipag.The results of the CUA indicated that,after 30 years of simulation calculations,the treatment regimen consisting of selexipag + ERA + PDE5 i for patients with PAH in WHO FC II and FC III has an ICER of 3045386.790 RMB per QALY compared to ERA + PDE5 i alone.This represents a significant increase of 11.85 times the WTP threshold.In the univariate sensitivity analysis,the cost of selexipag and transition probability were found to have significant impacts on the ICER.Varying each parameter within the preset range consistently resulted in an ICER exceeding WTP.In the probabilistic sensitivity analysis,if the WTP threshold exceeded 3031343.2 RMB per quality-adjusted life year(QALY),the probability of selexipag + ERA + PDE5 i being cost-effective was greater than 50%.In the scenario analysis,we conducted parameter adjustments for selexipag dosage,initial cohort status distribution,and study time horizon.After simulation calculations,all ICERs exceeded the WTP threshold.Conclusion:Selexipag is appropriate for sequential therapy in low-risk patients with PAH;however,meticulous pharmaceutical monitoring should be implemented throughout the treatment process.In the current medical context,sequential addition of selexipag is not a cost-effective option for PAH patients who have already received treatment with ERA combined with PDE5 i.It is recommended that government departments should give more attention to the current state of PAH treatment and alleviate the economic burden of this disease through price negotiations. |
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