Effectiveness，Safety and Economy of Treprostinil in the Treatment of Pulmonary Arterial Hypertension ：A Rapid Health Technology Assessment / 中国药房

ABSTRACT

OBJECTIVE：To provide evidence-based reference for c linical drug selection and decision by rapidly evaluating the effectiveness，safety and economy of treprostinil in the treatment of pulmonary arterial hypertension （PAH）. METHODS ：Retrieved from Chinese and English database such as PubMed ，Embase，Web of Science ，the Cochrane Library ，Epistemonikos，HTA database（University of York ），CNKI and Wanfang databases ，included the health technology assessment （HTA）report，systematic/ Meta-analysis and pharmacoeconomic evaluation of treprostinil compared with placebo or other drugs in the treatment of PAH. The search time limit is from the construction of the database to May 1st，2020. HTA checklist ，AMSTA and CHEERS were applied to evaluate the quali ty of the literatures about HTA ，systematic review/Meta-analysis analysis a nd pharmacoecono mic evaluation and the inclusion studies was analyazed by descriptive summary. RESULTS：A total of 18 literatures were included ，involving 1 HTA report ，12 systematic review/Meta-analysis ，5 pharma- coeconomic studies. The analysis results of effectiven- ess mail： showed that compared with placebo ，treprostinil could signifi- cantly increase 6-MWD while decrease Borg dyspnea score of PAH p atients（P＜0.05），but had no significant effect on mortality，the rate of clinical deterioration ，WHO functional grading ，the rate of hospitalization ，mPAP，PVR，cardiac index and mRAP（P＞0.05）. In addition ，compared with placebo combined with endothelin receptor antagonist and/or phosphodiesterase inhibitors，oral administration of treprostinil combined with endothelin receptor antagonist and/or phosphodiesterase inhibitors could extend 6-MWD significantly. Compared with riociguat ，treprostinil could significantly reduce Borg dyspnea score of patients. The analysis results of safety displayed that ，although the incidence of drug withdrawal due to can ’t tolerate ADR increased in patients receiving treprostinil ，there was no significant difference in the incidence of serious adverse events compared with placebo or other treatments，and it was better tolerated when administered by inhalation or intravenous injection. The analysis results of pharmacoeconomic studies showed that ICER of treprostinil was higher than the willingness payment threshold ，although the willingness payment threshold was different in different countries and different payers. CONCLUSIONS ：Treprostinil treatment is effective for PAH. Patients may stop taking it due to can ’t tolerate ADR ，but the risk of serious adverse events are not increase. Although the price is high ，it is still an important alternative for PAH patients with clinical progress or poor prognosis.