Objective We aimed to evaluate the comparative efficacy and cost-effectiveness of the ECMO versus mechanical ventilation through a rapid health technology assessment. Methods PubMed, EMbase, The Cochrane Library, CNKI, WanFang Data, and CBM databases were electronically searched to collect randomized controlled trials (RCTs), systematic reviews, meta-analyses, complete economic evaluations, and CRD database for HTA reports from inception to December 2020. Two reviewers independently screened literature, extracted data, and assessed the risk of bias of the included studies. Descriptive analysis and summary were then performed. Results A total of 21 references were involved, including 2 HTA reports, 5 RCTs, 11 systematic reviews or meta-analyses, and 3 economic evaluations. The quality of the literature evidence was heterogenous, and only 2 RCTs of high quality were included for meta-analysis. The results showed that the difference of 60-day mortality between ECMO and mechanical ventilation was statistically significant (RR=0.73, 95%CI 0.57 to 0.92, P=0.007). The majority of recent meta-analysis literature showed that short-term mortality of ECMO treatment was lower than that of mechanically ventilated patients. The cost-effective literature from different countries all showed that it was cost-effective in their respective health system, however, the quality of the literature varied. Conclusions Current evidence shows that ECMO has better safety, effectiveness and cost-effectiveness for ARDS compared with mechanical ventilation. However, it still required to be verified by high-quality studies with a long-term follow-up. Validate conclusions are needed through rigorous health technology assessments.
ObjectiveTo systematically review the health economic evaluation of nasopharyngeal carcinoma (NPC). MethodsThe PubMed, Web of Science, EMbase, CNKI, WanFang Data and CBM databases were electronically searched to collect the health economic evaluations on NPC from inception to December 18, 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of the included studies. A descriptive analysis was performed. ResultsA total of 20 studies were included, which contained 14 about different drug combinations, 6 about chemotherapy and the comparison among intensity modulated radiotherapy, conventional radiotherapy and surgery. The results showed that for patients with recurrent, metastatic, or advanced NPC, compared with docetaxel plus cisplatin, fluorouracil plus cisplatin or docetaxel and cisplatin plus fluorouracil, gemcitabine plus cisplatin (GP) was the most economical, and compared with GP or camrelizumab plus GP, toripalimab plus GP (TGP) was more economical. For early-stage NPC, intensity modulated radiotherapy was not economical compared with conventional radiotherapy and surgery. ConclusionCurrent evidence shows that GP and TGP are economical and can be popularized clinically.
ObjectiveTo explore how to determine the attributes of stated preference research more scientifically and reasonably. MethodsBased on the best-worst scaling object case (BWS-1) method, a BWS-1 questionnaire was generated using a balanced incomplete block design. Data collection was conducted among type 2 diabetes mellitus (T2DM) patients in Hainan and Jiangsu provinces. Data analysis was performed using counting analysis and conditional logit model to obtain the priority order of each attribute. ResultsThe results of BWS-1 using the counting and modelling approach showed high consistency. Among the 11 attributes, the top three attributes influencing the preference for second-line antihyperglycemic medications selection in T2DM patients were blood glucose control effectiveness, cardiovascular protection capability, and risk of hypoglycemic events, while the last three factors were dosing frequency, mode of administration and bone fracture. Based on literature review, qualitative research, and BWS-1 results, the seven attributes of discrete choice experiment and best-worst scaling profile case (BWS-2) were determined as follows: treatment efficacy, weight change, hypoglycemic events, gastrointestinal side effects, cardiovascular health, mode of administration and out-of-pocket cost. ConclusionBWS-1 can serve as an effective tool for determining the attributes of stated preference research. However, it is not recommended to solely rely on the priority ranking of BWS-1 results to determine the scope of attributes for stated preference research. It is necessary to conduct a specific analysis in conjunction with the research's policy objectives and real-world circumstances.
Objective To systematically review the health economic evaluations of using long-term rhythm-control antiarrhythmic drugs (AAD) for patients with paroxysmal or persistent atrial fibrillation (AF). Methods Databases including PubMed, EMbase, Scopus, CNKI, SinoMed, WanFang Data, and official websites of well-established health technology assessment (HTA) institutions were electronically searched to present the economic evaluations of AAD and the recommendations of HTA institutions based on drug economy from inception to April 23rd, 2022. Two reviewers independently screened the literature, extracted data and systematic review was then performed. Results A total of 19 studies were included, including 11 cost-effectiveness or cost-utility analysis studies and 8 official documents from HTA institutions. Only 5 (45.5%) economic evaluations were of relatively high quality, and English language studies were of higher quality than Chinese language studies ones. The included studies lacked elements that CHEERS 2022 concerns, such as health economics analysis plans, equity and distributional effects, engagement with patients and other stakeholders and the impact on the study. Dronedarone and amiodarone were the main focus of the evaluation, and the study results showed that dronedarone was cost-effective compared with other drugs in different study designs and national settings. However, there were differences between the recommendations of HTA agencies and the results of economic evaluation studies. Conclusion The completeness of health economics evaluations needs to be improved, along with the quality of clinical evidence in the field of AF-AAD for Chinese patients. Additionally, the informational value of drugs should be thoroughly investigated through budget impact analysis and distributional cost-effectiveness analysis to provide evidence of high-quality studies for decision-makers in China.
ObjectiveTo utilize a rapid health technology assessment to evaluate the efficacy, safety and cost-effectiveness of the MitraClip device for patients with severe mitral regurgitation (MR). MethodsPubMed, EMbase, The Cochrane Library, CNKI, WanFang Data, CBM and the CRD databases were electronically searched to collect clinical evidence and economic evaluations on the efficacy, safety and cost-effectiveness of the MitraClip device for patients with severe MR from inception to May 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies; then, descriptive analyses and data summaries were performed. ResultsA total of 33 studies, involving 4 HTA reports, 3 RCTs, 16 systematic reviews or meta-analyses, and 10 economic evaluations were included. In the evidence comparing MitraClip and surgery, most of the literature showed that the MitraClip group had higher postoperative residual MR, fewer blood transfusion events, and fewer hospital days. We found no significant treatment effects on 30-day adverse events and mortality, and the 1-year and above survival rate. In the evidence of MitraClip versus medical therapy alone, all included studies showed that MitraClip benefited mid-term and long-term survival and reduced the incidence of subsequent cardiac hospitalizations. Economic evaluations showed that the clinical benefits were cost-effective in the setting of their health service systems. ConclusionThe available high-grade clinical evidence shows that MitraClip is effective and safe to some extent, and has cost-effectiveness compared with traditional treatment in other countries. However, the real-world effectiveness and cost-effectiveness of the MitraClip need to be tested in the Chinese population and health-care setting.
Objective To systematically review the survival outcome and safety of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for β-thalassemia. Methods The PubMed, EMbase, CNKI, WanFang Data and CBM databases were electronically searched to collect studies on haplo-HSCT for β-thalassemia from January 1, 2017 to December 31, 2021. Two reviewers independently screened the literature, extracted data and assessed the risk of bias of the included studies. Meta-analysis was then performed by using RevMan 5.4.1 software and Stata 16.0 software. Results A total of 6 case-series studies involving 286 patients were included. The results of meta-analysis indicated that overall survival (OS) and thalassemia-free survival (TFS) for β-thalassemia patients undergoing haplo-HSCT were 92.5% (95%CI 86.1% to 96.1%) and 88.5% (95%CI 74.6% to 95.3%), the incidence of Ⅲ-Ⅳ degree acute graft versus host disease (Ⅲ-Ⅳ aGvHD) and chronic graft versus host disease (cGvHD) were 11.5% (95%CI 6.5% to 20.0%) and 23.1% (95%CI 12.3% to 39.8%), and the transplantation related mortality was 6.5% (95%CI 3.8% to 10.7%). Conclusion Relevant clinical studies published in the past 5 years provide the latest information and progress of haplo-HSCT for β-thalassemia. At present, great efficacy has been shown in NF-14-TM therapeutic regimen, but the long-term efficacy remains unclear. Due to the limited quality and quantity of the included studies, more high-quality evidence from long-term comparative studies is still needed.
Objective This study aimed to quantitatively investigate the preferences and willingness of patients with breast cancer to pay for central venous access and to provide implications for the clinical selection of appropriate chemotherapy pathways. Methods A discrete-choice experiment survey was conducted to elicit the preferences for central venous access in three hospitals in east, middle and west China. The conditional logit model was used to analyse the relative importance of six central venous access-related attributes: risk of thrombosis, risk of infections, restriction of daily activities, maintenance interval, catheter incision size and out-of-pocket costs. Results The valid data for a total of 103 patients was collected from three hospitals. All six attributes significantly influenced patients’ preferences for central venous access. The risk of thrombosis (RIS=26.0%) and risk of infections (RIS=24.3%) were the top two attributes influencing patients’ preferences for central venous access. To reduce the risk of thrombosis and infection from 12% and 8% to 1%, patients were willing to pay 14 861.2 yuan and 13 907.2 yuan, respectively. The catheter incision size was of least concern (RIS=4.6%); the patients were only willing to pay 2 653.6 yuan for smaller catheter incisions. Conclusion Thrombosis and infection are the primary factors that affect the choice of central venous access for patients with breast cancer. Patients have a sensitive trade-off between safety and out-of-pocket costs; with the change in thrombosis and infection risk, patients’ willingness to pay changes accordingly.
ObjectiveTo systematically review the economic evaluation research of anti-novel coronavirus infection drugs at home and abroad, so as to promote clinical rational drug use. MethodsThe PubMed, Cochrane Library, EMbase, Web of Science, INAHTA, SinoMed, WanFang Data, and CNKI databases were systematically searched from January 1, 2020 to March 25, 2023, to collect economic evaluation studies related to anti-novel coronavirus infection drugs. ResultsA total of 22 articles were included, among which 11 studies were conducted from the perspective of health system, and most of the studies performed cost estimation on direct medical costs. The overall compliance rate of the included studies ranged from 42% to 70%, with deficiencies in model setting, incomplete uncertainty analysis, and lack of stakeholder participation. The results showed that immunotherapy drugs (Dexamethasone, Tocilizumab), neutralizing antibody (REGEN-COV antibody), small molecule drugs (Baricitinib, Nirmatrelvir/Ritonavir, Molnupiravir, Favipiravir) and statin were cost-effective. There was some variation in the results of the economic evaluation of Remdesivir. ConclusionAt present, there are few studies on the economic evaluation of drug interventions in COVID-19. Existing studies have pointed out that most drug interventions are cost-effective. It is suggested that more standardized pharmacoeconomic evaluation studies based on the actual situation of China epidemic should be carried out in the future.
ObjectiveTo systematically review the economy of non-pharmaceutical interventions (NPIs) for COVID-19. MethodsThe Web of Science, PubMed, EMbase, Cochrane Library, INAHTA, CNKI, WanFang Data and SinoMed databases were electronically searched to collect studies on health economic evaluations from 1 January 2020 to 20 August 2022. Then the included materials were reviewed, extracted and data integration analysis were conducted based on inclusion and exclusion criteria. ResultsSeventy-one academic publications were finally included, which contained 25 papers about nucleic acid testing, antigen testing and screening, 5 papers about personal protection, 12 papers about social distancing, quarantine and isolation, 11 papers about regional or national lockdown and 18 papers about multiple NPIs. The results showed that compared with no intervention, nucleic acid testing, antigen testing, screening and personal protection measures were economical. Social distancing, quarantine and isolation were also economical compared with no intervention. However, in low-income countries, movement restriction and factory shutdown may exact a heavy toll on the poorest and most vulnerable. Moreover, compared with a single long-term lockdown, multiple short-term lockdowns could be more economical, but the cost was still huge overall. ConclusionNPIs such as nucleic acid testing, antigen testing, personal protection, social distancing, quarantine, isolation and factory shutdown are economical. Although regional or national lockdown can save lives, it is not suitable for wide use. The researches on specific populations, specific variants (especially Omicron) and in the context of China need to be carried out.
With the acceleration of global innovative drug development, selecting safe, effective, and cost-effective products from numerous drugs has posed new challenges for the decision-making process of medical insurance drug access and dynamic updating of insurance directory. Real-world data (RWD) provides a new perspective for evaluation of clinical and economic value of drugs, but there are still uncertainties regarding the scope, quality standards, and evidence categories of RWD that can be used. Based on the current status of domestic and international RWD supporting the assessment of the clinical and economic value of drugs, this paper, in collaboration with national RWD and healthcare experts, has developed the key considerations for using real-world data to evaluate the clinical and economic value of drugs. This paper first clarifies the scope of RWD that can be used to evaluate the clinical and economic value of drugs evaluate; secondly, provides specific requirements and guidance on data attribution, data governance, and quality standards for RWD; finally, summarizes the evidence categories of RWD supporting evaluate the clinical and economic value of drugs evaluate.