Abstract Background: Adults with special needs have dental problems requiring treatment; however, patient management could be extremely difficult under local anesthesia. This review aimed to compare the needs of pharmacological approaches versus non-pharmacological approach for dental treatment to adults with special needs. Methods: This systematic review was registered in PROSPERO (CRD42024528488). The systematic search was conducted in databases: PUBMED; EBSCO; SCOPUS, April 10, 2024. Clinical studies published in English from 2000 to June 2024, demonstrating the needs for pharmacological as compared with non-pharmacological approach in older adults over 18 years old were included. The primary outcome was the completion of dental treatment. Results: Titles and abstracts were screened after the initial search, then forty studies were identified for potential inclusion. After retrieving full-text studies, Information relevant to objectives and outcome measures was recorded by using a data extraction form and analyzed again by three reviewers. Only 2 articles were eligible and included. Conclusions: The best patient management approach could not be conclusive. Pharmacological approach remains necessary in many situations. Preparation of health care setting and multidisciplinary team is important to ensure patient safety. Further studies focusing on older adults with special needs are needed.

目的：对地舒单抗治疗骨质疏松的有效性、安全性和经济性进行快速卫生技术评估(HTA),为临床治疗提供循证证据。方法：计算机检索PubMed、Embase、the Cochrane Library、中国知网、万方数据库、维普数据库、中国生物医学文献服务系统及相关HTA网站、数据库，搜集地舒单抗治疗骨质疏松的高质量临床证据、药物经济学评价文献(干预组患者采用地舒单抗治疗；对照组患者采用唑来膦酸等药物及支持治疗),检索时限均为建库至2024年8月30日。由2名研究者独立筛选文献、提取资料和评价纳入研究的质量后，对结果进行定性描述与分析。结果：共纳入27篇文献，其中HTA报告2篇，系统评价/Meta分析12篇，药物经济学研究13篇。有效性方面，地舒单抗能够显著提高腰椎、髋部、股骨颈的骨密度，降低骨标志物水平，联合用药效果较单一用药效果更佳。安全性方面，地舒单抗主要导致耳鼻喉和胃肠道感染，总体发生率较低，在老年患者中心血管方面更为获益。经济学研究结果显示，地舒单抗治疗原发性骨质疏松具有成本-效益优势。结论：地舒单抗治疗骨质疏松具有一定的有效性和安全性，仍需高质量、大样本、多中心研究更进一步揭示其有...

The combination of immunosuppressive drugs is part of the treatment regimen of patients undergoing kidney transplantation (RT). Thymoglobulin(R), a rabbit immunoglobulin directed against human thymocytes, is the most commonly agent used for induction therapy in RT in the US. In Brazil, Thymoglobulin(R) is approved by ANVISA for the use in patients who underwent kidney transplantation and despite being widely used, there are controversies regarding the drug administration. We prepared a systematic review of the literature, evaluating studies that used Thymoglobulin(R) for induction and for acute rejection treatment in patients undergoing RT. The review used the computadorized databases of EMBASE, LILACS and MedLine. Data were extracted from the studies concerning general features, methodological characteristics and variables analyzed in each study. From the results, a practical guide was prepared analyzing various aspects on the use of Thymoglobulin(R) in patients submitted to RT

Hepatocellular carcinoma is one of the leading causes of cancer deaths globally and a key hindrance to extending life expectancy. Celastrol (CEL) demonstrates excellent antitumor activity, but faces challenges like low solubility and a narrow therapeutic window, limiting its clinical application. To address these limitations, drug combinations and nano-delivery systems have emerged as effective solutions. Curcumin (CUR), known for its antitumor and hepatoprotective effects, also exhibits good biocompatibility and the ability to mitigate drug-induced liver injury. Considering the complementary properties of CEL and CUR, including CEL's potent antitumor activity and CUR's hepatoprotective effects, we developed a novel self-assembling nanodrug delivery system (CCPN) for the co-loading of both compounds. CCPN nanoparticles were constructed through non-covalent interactions, including hydrogen bonding, π-π stacking, and electrostatic forces, which confer good stability and significantly enhance the solubility and bioavailability of CEL and CUR. Extensive in vitro and in vivo experiments demonstrated that CCPN effectively reduced CEL-induced hepatotoxicity in zebrafish and mouse models, exhibiting good biosafety. Additionally, CUR's fluorescence provides a unique advantage for real-time monitoring of drug distribution and release, facilitating the tracking of therapeutic progress. Furthermore, CCPN nanoparticles enhanced delivery efficiency in HepG2 cells, exhibiting superior anti-liver tumor outcomes, which are associated with the promotion of apoptosis in tumor cells. This study presents CCPN as a promising therapeutic strategy for hepatocellular carcinoma, integrating reduced hepatotoxicity, self-monitoring capabilities, and superior therapeutic efficacy.

Purpose: In order to scientifically evaluate the clinical value of the comprehensive attributes of Calcitonin gene-related peptide (CGRP) inhibitor drugs, a comprehensive literature-based clinical evaluation of CGRP-targeted therapy drugs was conducted using the drug evaluation method modified by expert discussion in the Rapid Guide for Drug Evaluation and Selection in Chinese Medical Institutions (Second Edition). Methods: Based on evidence-based data and the relevant elements and weighting in the "Selection Guidelines" quantification record form for drug evaluation and selection in medical institutions, adjustments were made according to the characteristics of CGRP-targeted therapy drugs. We systematically evaluated erenumab, galcanezumab, fremanezumab, eptinezumab, rimegepant, ubrogepant, atogepant, zavegepant for safety, efficacy, economy, and pharmacological properties. Results: The final assessment result scores from highest to lowest were rimegepant (84.5 points), erenumab (75.78 points), galcanezumab (74.02 points), fremanezumab (73.93 points), atogepant (72.64 points), eptinezumab (71.69 points), ubrogepant (70.37 points), zavegepant (56.44 points). Conclusion: Rimegepant, erenumab, fremanezumab, atogepant, galcanezumab, eptinezumab, ubrogepant can be entered into the medication list of medical institutions as strongly recommended drugs.

目的 为临床合理、安全应用度普利尤单抗治疗中重度特应性皮炎（AD）提供循证依据。方法 采用计算机检索PubMed,Embase,The Cochrane Library及中国知网（CNKI）、万方（WanFang）数据库和各国卫生技术评估（HTA）官方网站中自建库起至2023年12月1日关于度普利尤单抗治疗中重度AD的HTA报告、系统评价/Meta分析、药物经济学研究。采用系统性评价方法学质量工具AMSTAR2量表评价系统评价/Meta分析的质量，采用卫生经济学评价报告标准共识（CHEERS）评价药物经济学文献的质量。采用快速HTA法对结果进行描述性分析。结果 共纳入11篇文献，其中系统评价/Meta分析9篇、药物经济学研究2篇。有效性方面，度普利尤单抗较安慰剂/外用糖皮质激素（TCS）可显著改善湿疹面积和严重程度指数（EASI）评分、研究者整体评估（IGA）评分、瘙痒指数（NRS）评分、体表受累面积（BSA）较基线下降的百分比、皮肤病生活质量指数（DLQI）评分、湿疹测量（POEM）评分较基线下降的百分比、AD严重程度积分量表（SCORAD）评分较基线下降的百分比（P <0.0...

目的：评估转移性结直肠癌（metastatic colorectal cancer,mCRC）三线治疗口服药瑞戈非尼（regorafenib,Rego）、呋喹替尼（fruquintinib,Fruq）与曲氟尿苷替匹嘧啶（trifluridine/tipiracil,TAS102）的安全性、有效性与经济性，为临床决策提供循证依据。方法：系统检索HTA机构官方网站、PubMed、Cochrane Library、Embase、Web of Science、中国知网、万方、维普、中国生物医学文献数据库，纳入Rego、Fruq与TAS102用于mCRC三线治疗的系统评价/Meta分析、HTA报告和药物经济学研究，以描述性分析方法研究数据。结果：纳入14篇系统评价/Meta分析和8篇经济学研究。在mCRC三线治疗中，Fruq相比TAS102在无进展生存期和疾病控制率方面的获益更大。三药间的总生存期和客观缓解率相似。对于KRAS野生型患者，Fruq相比其余两药在无进展生存期方面的表现更优，但尚需更丰富的证据进一步验证。三药相比，Rego更易导致肝功能异常，TAS102更易发生白细胞、中性粒细胞减少...

目的：评价注射用乌司他丁(UTI)治疗急性循环衰竭(ACF)的有效性、安全性及经济性，为临床合理用药提供循证依据。方法：在PubMed、the Cochrane Library、Embase、万方数据库、中国知网、中国生物医学文献数据库以及卫生技术评估(HTA)机构官方网站中进行检索，收集关于UTI治疗ACF的系统评价/Meta分析、经济学研究及HTA报告，检索时间为建库至2024年6月。由2名研究者根据纳入与排除标准独立进行文献筛选、质量评价和数据提取。结果：共纳入5篇文献，均为Meta分析，未检索到经济学研究及HTA报告。目前纳入的研究在有效性方面选择炎症介质、多器官功能障碍综合征发生率、肝肾功能指标、心功能及死亡率等指标进行分析，联合应用UTI治疗ACF较单纯使用常规治疗具有明显优势，能显著降低炎症介质水平、改善器官损伤。结论：UTI用于ACF的治疗效果确切，安全性良好。

Background: Medication-related harm, including adverse drug events (ADEs) and medication errors, represents a significant iatrogenic burden in clinical care. Digital health technology (DHT) interventions can significantly enhance medication safety outcomes. Although the clinical effectiveness of DHT for medication safety has been relatively well studied, much less is known about the cost-effectiveness of these interventions. Objective: This study aimed to systematically review the economic impact of DHT interventions on medication safety and examine methodological challenges to inform future research directions. Methods: A systematic search was conducted across 3 major electronic databases (ie, PubMed, Scopus, and EBSCOhost). The PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines were followed for this systematic review. Two independent investigators conducted a full-text review after screening preliminary titles and abstracts. We adopted recommendations from the Panel on Cost-Effectiveness in Health and Medicine for data extraction. A narrative analysis was conducted to synthesize clinical and economic outcomes. The quality of reporting for the included studies was assessed using the CHEERS (Consolidated Health Economic Evaluation Reporting Standards) guidelines. Results: We included 13 studies that assessed the cost-effectiveness (n=9, 69.2%), cost-benefit (n=3, 23.1%), and cost-utility (n=1, 7.7%) of DHT for medication safety. Of the included studies, more than half (n=7, 53.9%) evaluated a clinical decision support system (CDSS)/computerized provider order entry (CPOE), 4 (30.8%) examined automated medication-dispensing systems, and 2 (15.4%) focused on pharmacist-led outreach programs targeting health care professionals. In 12 (92.3% ) studies, DHT was either cost-effective or cost beneficial compared to standard care. On average, DHT interventions reduced ADEs by 37.12% (range 8.2%-66.5%) and medication errors by 54.38% (range 24%-83%). The key drivers of cost-effectiveness included reductions in outcomes, the proportion of errors resulting in ADEs, and implementation costs. Despite a significant upfront cost, DHT showed a return on investment within 3-4.25 years due to lower cost related with ADE treatment and improved workflow efficiency. In terms of reporting quality, the studies were classified as good (n=10, 76.9%) and moderate (n=3, 23.1%). Key methodological challenges included short follow-up periods, the absence of alert compliance tracking, the lack of ADE and error severity categorization, and omission of indirect costs. Conclusions: DHT interventions are economically viable to improve medication safety, with a substantial reduction in ADEs and medication errors. Future studies should prioritize incorporating alert compliance tracking, ADE and error severity classification, and evaluation of indirect costs, thereby increasing clinical benefits and economic viability.

Background: Non-proliferative and proliferative diabetic retinopathy are common complications of diabetes and a major cause of sight loss. Anti-vascular endothelial growth factor drugs represent a treatment option for people with diabetic retinopathy and are routinely used to treat various other eye conditions. However, anti-vascular endothelial growth factor drugs are expensive relative to current care options, and it is unclear whether this additional cost is justified when the immediate risk of vision loss is lower compared to patients with more aggressive ophthalmological conditions. Objective: To systematically review the evidence supporting the cost-effectiveness of alternative treatments for diabetic retinopathy. Methods: A systematic review of all comparative cost-effectiveness studies evaluating any treatment for diabetic retinopathy was conducted. Bibliographic searches were carried out to identify studies reporting on the cost-effectiveness of treatments for diabetic retinopathy; the latest searches were conducted on 28 April 2023. Included studies were synthesised narratively and evaluated with reference to UK decision-making. Studies were grouped by population into non-proliferative diabetic retinopathy and proliferative diabetic retinopathy. Results: The review identified five studies in the proliferative diabetic retinopathy population, all of which examined the cost-effectiveness of anti-vascular endothelial growth factor treatments compared to pan-retinal photocoagulation. Results of these studies suggest that anti-vascular endothelial growth factor treatments offer some additional benefits in terms of preserved visual acuity but also incur substantial additional costs relative to pan-retinal photocoagulation. Most authors agreed that the additional costs outweigh the limited benefits, especially in certain patient subgroups without pre-existing oedema. As most of the identified evidence considered a US perspective, it is unclear how these results would translate to a UK setting. Two studies were identified in the non-proliferative diabetic retinopathy population. There was limited evidence to support the early use of anti-vascular endothelial growth factor treatment. However, one UK study suggested that early treatment of non-proliferative diabetic retinopathy with pan-retinal photocoagulation is cost-effective compared to delayed pan-retinal photocoagulation. Conclusions: Overall, there is a dearth of cost-effectiveness evidence considering the UK context. The identified studies raised doubts about the cost-effectiveness of anti-vascular endothelial growth factor treatments for proliferative diabetic retinopathy. No conclusions can be made regarding the cost-effectiveness of anti-vascular endothelial growth factor treatments for non-proliferative diabetic retinopathy. Future research should focus on developing rigorous model-based cost-effectiveness analyses integrating all available evidence. Funding: This article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number NIHR132948.

Objectives: Immune checkpoint inhibitor (ICI)-containing treatment is currently prescribed as first-line treatment for all patients with advanced non-small cell lung cancer (NSCLC) without targetable driver mutations. However, only 30-45% of patients show no progression within 12 months after treatment start. Various biomarkers are being studied to save costly and potentially harmful treatment in non-responders. We evaluated the cost-effectiveness of implementing a hypothetical predictive biomarker for ICI-containing treatment response compared with standard of care (e.g., no implemented biomarker) for pembrolizumab-containing treatment in patients with advanced NSCLC in the Netherlands. Materials and methods: Standard-of-care-based and predictive-biomarker-based strategies were compared using Markov models for three first-line pembrolizumab-containing treatments depending on a patient's tumor programmed cell death ligand-1 (PD-L1) expression and histology. A Dutch healthcare system perspective was adopted. Assuming a receiver operating characteristic-area under the curve of 1.0 in identifying responders, alternative treatments were offered for non-responders in the predictive-biomarker-based strategy. Parameters and assumptions were based on real-world data from surveys, literature using a targeted search, expert opinion, and registries. Outcomes included differences in costs, survival (life years (LYs)), and survival corrected for health-related quality of life (QoL) quality-adjusted life-years (QALYs) between the predictive-biomarker- and standard-of-care-based strategy. Results: Implementing a predictive biomarker in pembrolizumab-carboplatin-paclitaxel treatment led to a mean survival reduction of 24 days (- 0.067 LYs) (18 days corrected for QoL (- 0.049 QALYs)), with cost savings of €22,606 compared with standard of care. Pembrolizumab monotherapy and pembrolizumab-pemetrexed-platinum treatments showed survival reductions of 4.5 and 3.9 months, respectively (3.6 and 2.8 months corrected for QoL), with cost savings of €24,345 and €28,456. Sensitivity analyses confirmed consistent cost savings and survival reductions. Survival losses were mainly observed due to the lower survival rates associated with the alternative first-line treatment options available for non-responders in the predictive-biomarker-based strategy within each pembrolizumab-containing treatment regimen. Pembrolizumab-carboplatin-paclitaxel treatment also showed survival gains under certain conditions related to QoL and survival estimates. Conclusions: Our study highlights the importance of careful de-implementation of ICI-treatments in advanced NSCLC, balancing costs reductions and side effects without comprising survival. In the pembrolizumab-carboplatin-paclitaxel treatment regimen, the survival loss could be considered negligible. Future research should define acceptable tradeoffs and thresholds for de-implementation, considering factors such as survival of alternative treatments and responder classification to guide predictive biomarker implementation and optimize health resource allocation.

目的:评价德曲妥珠单抗(trastuzumab deruxtecan, T-DXd)在晚期或转移性乳腺癌治疗中的有效性、安全性和经济性。方法:系统检索中英文数据库及卫生技术评估(health technology assessment, HTA)相关网站，遴选相关研究并提取数据，进行描述性统计分析。结果:共纳入5篇HTA报告、19篇Meta分析/系统综述和14篇经济学研究。在人表皮生长因子受体2(human epidermal growth factor receptor 2,HER2)阳性晚期或转移性乳腺癌的二线治疗中，T-DXd对比恩美曲妥珠单抗(trastuzumab emtansine, T-DM1)有效性更佳。T-DXd常见不良事件包括胃肠道反应、血液毒性、疲劳、脱发等，特殊不良事件主要为间质性肺病。由于药品价格限制，对于中国支付者，T-DXd相比T-DM1和化疗方案均不具成本效益。结论:T-DXd有效性良好、安全性可控，暂不具备经济性。

目的 对托法替布治疗溃疡性结肠炎的有效性、安全性和经济性进行快速卫生技术评估，为临床合理用药及决策提供循证依据。方法 计算机检索PubMed、Embase、Cochrane Library、CNKI、WanFang Data和VIP数据库，以及相关卫生技术评估网站及数据库，搜集托法替布治疗溃疡性结肠炎的高质量临床证据、经济学评价文献，检索时限均从建库至2024年7月31日。由2位研究者独立筛选文献、提取资料和评价质量后，对结果进行定性分析。结果 共纳入文献17篇，其中卫生技术评估报告1篇、系统评价/Meta分析9篇、药物经济学研究8篇。有效性方面，托法替布治疗能够显著改善溃疡性结肠炎患者的临床反应率、临床缓解率、黏膜愈合率、内镜缓解率和短期结肠切除率（P 0.05），仅感染发生率略有增加（P <0.05）；经济性方面，在包括中国在内的多个国家，与传统治疗或生物制剂相比，托法替布治疗中重度溃疡性结肠炎具有成本-效果优势。结论 托法...

Background: Obinutuzumab was approved in China in June 2021 used in combination with chemotherapy (followed by obinutuzumab maintenance) for the treatment of adult patients with previously untreated stage II bulky, III, or IV follicular lymphoma (FL). The clinical application of obinutuzumab has recently begun in China, but there is a lack of evidence to determine under which circumstances it should be considered the treatment of choice. A comprehensive assessment is necessary to evaluate the efficacy, safety, and cost-effectiveness of obinutuzumab in adult patients with FL. Objective: To summarize the evidence on the efficacy, safety, and cost-effectiveness of obinutuzumab in adult patients with FL, aiming to provide medical professionals with evidence for informed choices in clinical practice. Methods: The approach to this evidence synthesis was a rapid review of systematic reviews/meta-analyses (SR/meta-analyses), health technology assessment (HTA) reports, and pharmacoeconomic studies that brings together and summarizes the efficacy, safety, and cost-effectiveness of obinutuzumab in adult patients with FL. A literature search was conducted across multiple databases, including PubMed, Embase, Wanfang, CNKI, Weipu database, the Cochrane Library, the Centre for Reviews and Dissemination (CRD) database, International Network of Agencies for Health Technology Assessment (INAHTA) and Canada's Drug Agency (CDA-AMC), International Society for Pharmacoeconomics and Outcomes Research (ISPOR), National Institute For Health and Care Excellence (NICE), Institute For Clinical And Economic Review (ICER), Grey Literature Database and Grey Net International. The studies on obinutuzumab for FL were searched in full text with obinutuzumab, systematic review, meta-analysis, economics, cost, and health technology assessment as keywords, with a search time frame from the date of database creation to 29 November 2024. The literature was screened based on predefined inclusion and exclusion criteria, and data were meticulously extracted and synthesized by two authors. Simultaneously, the quality of the literature was thoroughly assessed. Results: Obinutuzumab based chemotherapy (the chemotherapy regimen-cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP); cyclophosphamide, vincristine, and prednisone (CVP); or bendamustine) significantly prolonged progression free survival (PFS) compared to other chemotherapy regimen at primary and updated analyses. The incidence of grade 3-5 AEs, infusion-related reactions (IRRs), and infection were higher in the obinutuzumab based chemotherapy group compared to other chemotherapies. The economic researches conducted in China, United States, Japan, Italy and Norway had demonstrated that obinutuzumab-based chemothrepy was cost-effective compared to other chemothrepies. Although obinutuzumab significantly prolonged PFS and was cost-effective, its safety profile was considered lower. Conclusion: Compared with other chemothrapy regimen, obinutuzumab based chemotherapy significantly prolonged PFS and was cost-effective, while its safety profile was considered lower. Therefore, medical professionals should be caution when using or introducing obinutuzumab treatment for FL patients.

目的 评价替尔泊肽用于2型糖尿病和长期体重管理的有效性、安全性和经济性，为临床药物治疗及医保政策制定提供循证依据。方法检索Embase、PubMed、theCochraneLibrary、中国知网和国际卫生技术评估（HTA）官方网站，收集替尔泊肽用于T2DM和长期体重管理的HTA报告、系统评价/Meta分析和药物经济学研究，检索时限均为建库至2024年10月1日。经资料提取、质量评价后，对纳入研究的结果进行描述性分析。结果共纳入18篇文献，包括14篇系统评价/Meta分析和4篇药物经济学研究，未检索到HTA报告。在有效性方面，绝大多数研究表明，替尔泊肽10、15mg在降低HbA1c、体重和腰围方面均显著优于其他胰高血糖素样肽1（GLP-1）受体激动剂（P＜0.05）。在安全性方面，与其他GLP-1受体激动剂比较，替尔泊肽未增加胃肠道相关不良事件（AE）发生率、≥3级AE发生率和严重低血糖发生率（P＞0.05），但替尔泊肽15mg可能会显著升高低血糖发生率和因不良反应退出率（P＜0.05）。在经济性方面，基于国外药物经济学研究的结果显示，替尔泊肽相比于司美格鲁肽和利拉鲁肽具有成本-效益优势。结论替尔泊肽10、15mg用于T2DM和长期体重管理的疗效和安全性均较好，但使用替尔泊肽15mg时，需密切关注其可能导致的低血糖风险和因不良反应退出风险；基于国外药物经济学研究结果，替尔泊肽具有经济学优势。

目的 采用快速卫生技术评估（HTA）的方法，评价度普利尤单抗治疗重度哮喘的有效性、安全性和经济性，为临床治疗提供循证依据。方法检索PubMed、CochraneLibrary、中国知网、万方和维普网等数据库及HTA机构官网，收集度普利尤单抗治疗重度哮喘的HTA报告、系统评价/Meta分析和药物经济学研究。筛选文献、提取数据并评估文献质量后，对文献结果进行描述性分析。结果共纳入15篇文献，包括9篇系统评价/Meta分析和6篇药物经济学研究。有效性方面，度普利尤单抗显著优于安慰剂。与其他生物制剂比较，在12岁及以上的重度哮喘患者中，对于嗜酸性粒细胞（EOS）≥300个/μL的人群，度普利尤单抗改善第1秒用力呼气容积（FEV1）的作用排第1位，优于特泽鲁单抗、贝那利珠单抗和美泊利珠单抗；减少哮喘急性发作的作用排第2位，仅次于特泽鲁单抗；而改善哮喘控制问卷评分的作用排名比较靠后，仅优于贝那利珠单抗。对于150个/μL≤EOS＜300个/μL的人群，度普利尤单抗对哮喘急性发作的改善情况优于美泊利珠单抗，而对FEV1的改善作用弱于贝那利珠单抗和美泊利珠单抗。安全性方面，度普利尤单抗与安慰剂及其他生物制剂的不良事件和严重不良事件发生率的差异无统计学意义，但其注射部位反应的发生率显著高于安慰剂。经济性方面，各国研究结果不一致，尚缺乏我国的研究数据。结论度普利尤单抗用于重度哮喘具有良好的有效性和安全性，经济性有待基于我国医疗环境进一步研究。

目的 采用快速卫生技术评估方法，评价伏诺拉生（VPZ）治疗胃食管反流病的有效性、安全性和经济性，为临床决策提供依据。方法计算机检索PubMed、Medline、CochraneLibrary、中国知网、维普、万方等中英文数据库，以及国内外卫生技术评估机构官网，检索时限从建库起至2024年8月，由2位研究者独立筛选文献、提取资料并评价纳入研究的质量后，对结果进行定性描述与分析。结果共纳入21篇文献，包括系统评价/Meta分析15篇和药物经济学研究6篇。有效性方面，与对照方案（不同剂量VPZ、安慰剂、其他阳性对照药或联合治疗）相比，VPZ（主要为20mg/dVPZ）显著提高了治疗总有效率、第2周的黏膜愈合率、症状缓解率和愈合后第12、24周的维持率（P＜0.05）；当内镜下洛杉矶分级为C/D时，VPZ有效率显著高于对照方案（P＜0.05）。安全性方面，VPZ与对照方案治疗GERD的不良事件发生率无显著差异（P＞0.05），但长期使用VPZ导致血清胃泌素升高及肝功能异常的风险较对照方案更高（P＜0.05）。经济性方面，与雷贝拉唑、兰索拉唑、艾司奥美拉唑相比，VPZ更具有成本-效用/成本-效果优势。结论VPZ治疗胃食管反流病具有良好的有效性、安全性、经济性。

Spinal muscular atrophy (SMA) is a rare inherited autosomal recessive progressive disease of a varying phenotype, with varying clinical symptoms, and as a result the patients suffering from it require multiple types of care. It was deemed useful to conduct a systematic literature review on the pharmacoeconomic evaluations of all currently registered disease-modifying therapies in order to inform policy and highlight research gaps. Pharmacoeconomic analyses written in English and published after 2016 were considered for inclusion. PubMed/Medline, Global Health and Embase were systematically and separately searched between 16 October and 23 October 2023. Hand-searching was also conducted on PubMed based on reference lists of published literature. After the exclusion criteria were applied, 14 studies were included. BMJ checklist was used for quality assessment and the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist was used to assess the quality of reporting of all included studies. Data extraction was performed manually. Regarding evidence synthesis, data were heterogeneous and are thus presented based on comparison. This study confirms the need for pharmacoeconomic analyses (cost-effectiveness or cost-utility) also in cases when the cost of treatment is very high and the incremental cost-effectiveness ratio values exceed the usual, acceptable values for standard therapy. Specific willingness to pay thresholds for orphan medicines are of the utmost importance, to allow patients with SMA to have access to safe and effective treatments. With such economic evaluations, it is possible to compare the value of medications with the same indication, but it should be emphasized that in the interpretation of data and in making decisions about the use of medicines, the impact of new knowledge should be considered.

Background: Heart failure, a complex clinical syndrome with high morbidity and mortality, has become a significant burden on public health. Recently, a new class of antidiabetic agents-the sodium-glucose cotransporter 2 (SGLT2) inhibitors-was associated with a significant reduction on mortality and hospitalization in HF with reduced ejection fraction (HFrEF) when added to standard pharmacological treatment. Considering the lack of data on its cost-effectiveness, the present study aims to estimate the incremental cost-effectiveness ratio of add-on dapagliflozin treatment for HFrEF from the Brazilian public healthcare system perspective. Methods: We built a Markov model to estimate the clinical outcomes and costs of 1,000 hypothetical subjects with established HFrEF in a lifetime horizon. The model inputs were based on the Dapagliflozin and Prevention of Adverse Outcomes in Heart Failure (DAPA-HF) trial and local data. The main outcome was the incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained. Deterministic and probabilistic sensitivity analyses, as well as scenario analyses, were performed. Findings: The addition of dapagliflozin to standard care treatment in 1,000 HFrEF patients yielded an expected value of 366.99 additional QALYs at an incremental cost of US$ 1,517,878.49, resulting in an ICER of US$ 4,136.08 per QALY gained, being a cost-effective strategy considering the Brazilian official cost-effectiveness threshold (US$ 8,000/QALY). In probabilistic sensitivity analyses, 96.60% of the simulations were also cost-effective. In the scenario analyses, results were similar for individuals with and without diabetes. Interpretation: Dapagliflozin is likely to be cost-effective when added to standard HFrEF therapy in Brazil. Funding: This study was supported by the National Institute of Science and Technology for Health Technology Assessment (Instituto de Avaliação de Tecnologias em Saúde-IATS).

Objective: This study aims to support the selection of PCSK9 inhibitors for patients requiring lipid management within medical institutions. By quantitatively evaluating four PCSK9 inhibitors, we provide evidence-based guidance for optimal selection in this patient population. Methods: According to the Rapid Guide for Drug Evaluation and Selection in Chinese Medical Institutions (Second Edition) released in 2023, relevant databases such as PubMed, Cochrane, Embase, drug labels, and clinical guidelines were searched for drug information. Using a percentage scoring method, we systematically evaluated 4 PCSK9 inhibitors marketed in China for safety, efficacy, economy, pharmacological properties, and other attributes. Results: The final assessment result scores from highest to lowest were evolocumab (78.00 points), alirocumab (77.24 points), inclisiran (72.89 points), and tafolecimab (65.33 points). Evolocumab was the best in the economy, alirocumab scored the highest in terms of efficacy and other attributes, and inclisiran had the strongest performance in terms of pharmacological properties. Conclusion: For lipid management in medical institutions, evolocumab, alirocumab, inclisiran, and tafolecimab may be prioritized accordingly based on evaluation results.