Cost-effectiveness analysis

成本效益分析
  • 文章类型: Systematic Review
    我们比较了相对收益,术中腹膜热化疗+细胞减灭术±全身化疗与细胞减灭术±全身化疗或单纯全身化疗对结肠直肠腹膜转移患者的危害和成本效益,通过系统评价胃癌或卵巢癌,元分析和基于模型的成本效用分析。
    我们搜索了MEDLINE,EMBASE,科克伦图书馆和科学引文索引,ClinicalTrials.gov和WHOICTRP试验登记至2022年4月14日。我们仅包括解决研究目标的随机对照试验。我们使用Cochrane偏倚风险工具版本2来评估随机对照试验中的偏倚风险。在适用时,我们使用随机效应模型进行数据合成。对于成本效益分析,我们使用美国国家健康与护理卓越研究所推荐的方法进行了基于模型的成本-效用分析.
    系统评价包括总共8项随机对照试验(7项随机对照试验,955名参与者纳入定量分析)。除III期或更高的上皮性卵巢癌以外的所有比较仅包含一项试验,表明缺乏提供数据的随机对照试验。对于结直肠癌,术中腹腔热化疗+细胞减灭术+全身化疗可能导致全因死亡率几乎没有差异(60.6%vs.60.6%;风险比1.00,95%置信区间0.63至1.58),与细胞减灭术±全身化疗相比,可能会增加严重不良事件的比例(25.6%vs.15.2%;风险比1.69,95%置信区间1.03~2.77)。与单纯以氟尿嘧啶为基础的全身化疗相比,术中腹腔热化疗+细胞减灭术+全身化疗可能会降低全因死亡率(40.8%vs.60.8%;风险比0.55,95%置信区间0.32至0.95)。对于胃癌,术中腹腔热化疗+细胞减灭术+全身化疗与细胞减灭术+全身化疗或单纯全身化疗对全因死亡率的影响存在高度不确定性.对于接受间隔细胞减灭术的III期或更高的上皮性卵巢癌,与细胞减灭术+全身化疗相比,术中腹腔热化疗+细胞减灭术+全身化疗可能降低全因死亡率(46.3%vs.57.4%;风险比0.73,95%置信区间0.57~0.93)。术中腹腔热化疗+细胞减灭术+全身化疗可能与细胞减灭术+全身化疗治疗结直肠癌的成本效益不同,但对于其余的比较可能是成本效益。
    我们无法按计划获取个体参与者数据。每次比较的随机对照试验数量有限,以及与健康相关的生活质量数据匮乏,这意味着随着新证据(来自偏倚风险较低的试验)的出现,建议可能会发生变化。
    在患有结肠直肠癌腹膜转移的人中,腹膜转移有限,并且可能承受大手术,在常规临床实践中不宜使用术中腹腔热化疗+细胞减灭术+全身化疗(强烈推荐)。对于胃癌和腹膜转移患者,是否应提供术中高温腹膜化疗+细胞减灭术+全身化疗或细胞减灭术+全身化疗存在相当大的不确定性(无推荐)。术中腹腔热化疗+细胞减灭术+全身化疗应常规用于III期或更高级别上皮性卵巢癌和局限于腹部的转移患者,需要并可能在化疗后经受间期细胞减灭术(强烈推荐)。
    需要更多的随机对照试验。
    本研究注册为PROSPEROCRD42019130504。
    该奖项由美国国家卫生与护理研究所(NIHR)卫生技术评估计划(NIHR奖编号:17/135/02)资助,并在《卫生技术评估》中全文发布。28号51.有关更多奖项信息,请参阅NIHR资助和奖励网站。
    肠癌,卵巢或胃可以扩散到腹部(“腹膜转移”)。通过注射或片剂(“全身化疗”)给予的化疗(使用旨在杀死癌细胞的药物)是主要的治疗选择之一。对于增加细胞减灭术(细胞减灭术;切除癌症的手术)和“术中腹膜热化疗”(在细胞减灭术中进入腹部衬里的热化疗)是否有益,存在不确定性。我们回顾了截至2022年4月14日发表的所有医学文献信息,以回答上述不确定性。我们从八项试验中发现了以下内容,包括约1000名参与者。在患有肠癌腹膜转移的人中,与细胞减灭术+全身化疗相比,术中腹腔热化疗+细胞减灭术+全身化疗可能不会带来任何益处,也会增加伤害。与单纯全身化疗相比,细胞减灭术+全身化疗似乎能提高生存率。对于胃癌腹膜转移患者的最佳治疗方法存在不确定性。在患有卵巢癌腹膜转移的女性中,在进行细胞减灭术之前需要进行全身化疗以缩小癌症以进行手术(“晚期卵巢癌”),与细胞减灭术+全身化疗相比,术中腹腔热化疗+细胞减灭术+全身化疗可能会增加生存率。在能够承受大手术并且可以切除癌症的人中,肿瘤细胞减灭术+全身化疗应提供给患有肠癌腹膜转移的人,对于“晚期卵巢癌”腹膜转移的女性,应提供术中高温腹膜化疗+细胞减灭术+全身化疗。胃癌治疗的不确定性仍在继续。该奖项由美国国家卫生与护理研究所(NIHR)卫生技术评估计划(NIHR奖ref:17/135/02)资助,并在《卫生技术评估》中全文发表;28号51.有关更多奖项信息,请参阅NIHR资助和奖励网站。
    UNASSIGNED: We compared the relative benefits, harms and cost-effectiveness of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery ± systemic chemotherapy versus cytoreductive surgery ± systemic chemotherapy or systemic chemotherapy alone in people with peritoneal metastases from colorectal, gastric or ovarian cancers by a systematic review, meta-analysis and model-based cost-utility analysis.
    UNASSIGNED: We searched MEDLINE, EMBASE, Cochrane Library and the Science Citation Index, ClinicalTrials.gov and WHO ICTRP trial registers until 14 April 2022. We included only randomised controlled trials addressing the research objectives. We used the Cochrane risk of bias tool version 2 to assess the risk of bias in randomised controlled trials. We used the random-effects model for data synthesis when applicable. For the cost-effectiveness analysis, we performed a model-based cost-utility analysis using methods recommended by The National Institute for Health and Care Excellence.
    UNASSIGNED: The systematic review included a total of eight randomised controlled trials (seven randomised controlled trials, 955 participants included in the quantitative analysis). All comparisons other than those for stage III or greater epithelial ovarian cancer contained only one trial, indicating the paucity of randomised controlled trials that provided data. For colorectal cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably results in little to no difference in all-cause mortality (60.6% vs. 60.6%; hazard ratio 1.00, 95% confidence interval 0.63 to 1.58) and may increase the serious adverse event proportions compared to cytoreductive surgery ± systemic chemotherapy (25.6% vs. 15.2%; risk ratio 1.69, 95% confidence interval 1.03 to 2.77). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to fluorouracil-based systemic chemotherapy alone (40.8% vs. 60.8%; hazard ratio 0.55, 95% confidence interval 0.32 to 0.95). For gastric cancer, there is high uncertainty about the effects of hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy versus cytoreductive surgery + systemic chemotherapy or systemic chemotherapy alone on all-cause mortality. For stage III or greater epithelial ovarian cancer undergoing interval cytoreductive surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably decreases all-cause mortality compared to cytoreductive surgery + systemic chemotherapy (46.3% vs. 57.4%; hazard ratio 0.73, 95% confidence interval 0.57 to 0.93). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy may not be cost-effective versus cytoreductive surgery + systemic chemotherapy for colorectal cancer but may be cost-effective for the remaining comparisons.
    UNASSIGNED: We were unable to obtain individual participant data as planned. The limited number of randomised controlled trials for each comparison and the paucity of data on health-related quality of life mean that the recommendations may change as new evidence (from trials with a low risk of bias) emerges.
    UNASSIGNED: In people with peritoneal metastases from colorectal cancer with limited peritoneal metastases and who are likely to withstand major surgery, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should not be used in routine clinical practice (strong recommendation). There is considerable uncertainty as to whether hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy or cytoreductive surgery + systemic chemotherapy should be offered to patients with gastric cancer and peritoneal metastases (no recommendation). Hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered routinely to women with stage III or greater epithelial ovarian cancer and metastases confined to the abdomen requiring and likely to withstand interval cytoreductive surgery after chemotherapy (strong recommendation).
    UNASSIGNED: More randomised controlled trials are necessary.
    UNASSIGNED: This study is registered as PROSPERO CRD42019130504.
    UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.
    Cancers of the bowel, ovary or stomach can spread to the lining of the abdomen (‘peritoneal metastases’). Chemotherapy (the use of drugs that aim to kill cancer cells) given by injection or tablets (‘systemic chemotherapy’) is one of the main treatment options. There is uncertainty about whether adding cytoreductive surgery (cytoreductive surgery; an operation to remove the cancer) and ‘hyperthermic intraoperative peritoneal chemotherapy’ (warm chemotherapy delivered into the lining of the abdomen during cytoreductive surgery) are beneficial. We reviewed all the information from medical literature published until 14 April 2022, to answer the above uncertainty. We found the following from eight trials, including about 1000 participants. In people with peritoneal metastases from bowel cancer, hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably does not provide any benefits and increases harm compared to cytoreductive surgery + systemic chemotherapy, while cytoreductive surgery + systemic chemotherapy appears to increase survival compared to systemic chemotherapy alone. There is uncertainty about the best treatment for people with peritoneal metastases from stomach cancer. In women with peritoneal metastases from ovarian cancer who require systemic chemotherapy before cytoreductive surgery to shrink the cancer to allow surgery (‘advanced ovarian cancer’), hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy probably increases survival compared to cytoreductive surgery + systemic chemotherapy. In people who can withstand a major operation and in whom cancer can be removed, cytoreductive surgery + systemic chemotherapy should be offered to people with peritoneal metastases from bowel cancer, while hyperthermic intraoperative peritoneal chemotherapy + cytoreductive surgery + systemic chemotherapy should be offered to women with peritoneal metastases from ‘advanced ovarian cancer’. Uncertainty in treatment continues for gastric cancer. This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 17/135/02) and is published in full in Health Technology Assessment; Vol. 28, No. 51. See the NIHR Funding and Awards website for further award information.
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  • 文章类型: Journal Article
    肾细胞癌是最常见的肾癌类型,占所有肾脏恶性肿瘤的约85%。晚期肾细胞癌患者是国家健康与护理卓越研究所多项技术评估的重点。患者疾病进展的风险取决于许多预后风险因素;患者被分类为具有中等/低风险或有利的疾病进展风险。
    这项多技术评估的目标是评估lenvatinib和pembrolizumab的临床有效性和成本效益,以及美国国家卫生与护理卓越研究所发布的最终范围中列出的相关比较剂:舒尼替尼,帕唑帕尼,tivozanib,卡博替尼和纳武单抗联合伊匹单抗。
    评估小组进行了临床和经济系统评价,并评估了卫材提交的临床和成本效益证据,哈特菲尔德,赫特福德郡,英国(lenvatinib的制造商)和默克夏普和多姆,怀特豪斯车站,NJ,美国(pembrolizumab的制造商)。评估小组使用贝叶斯框架进行固定效应网络荟萃分析,以产生临床有效性的证据。由于数据稀疏,出现了收敛问题,随机效应网络荟萃分析结果不可用.评估小组没有建立从头的经济模型,而是修改了MerckSharp&Dohme提供的分区生存模型。
    评估组临床系统评价确定了一项相关的随机对照试验(CLEAR试验)。清晰的审判是一个很好的质量,第三阶段,多中心,开放标签试验提供了lenvatinib联合pembrolizumab与舒尼替尼相比的有效性和安全性的证据.所有三个风险组的评估组无进展生存网络荟萃分析结果不应用于推断任何治疗比较的统计学显著差异(或缺乏统计学显著差异),这是由于试验内比例风险违反或比例风险假设有效性的不确定性。中/低风险亚组的评估组总体生存网络荟萃分析结果表明,但没有统计学意义,与接受卡博替尼或纳武单抗联合ipilimumab治疗的患者相比,接受乐伐替尼联合派博利珠单抗治疗的患者的总生存期有所改善.由于审判内比例风险违反或比例风险假设有效性的不确定性,对于有利风险亚组和所有风险人群,评估组总体生存网络荟萃分析结果不应用于推断任何治疗比较的统计学显著性差异(或无统计学显著性差异).仅一项成本效益研究被纳入评估小组对成本效益证据的审查。这项研究仅限于所有风险人群,从美国医疗保健系统的角度出发,包括美国国家健康与护理卓越研究所不推荐的未经治疗的晚期肾细胞癌患者的比较。因此,资源使用和结果可推广到NHS的程度尚不清楚.评估组的成本效益来自修改后的分区生存模型,该模型侧重于中/低风险和有利风险子组。评估小组的成本效益结果,使用所有药物的标价生成,表明,对于有利风险亚组的所有比较,与NHS患者可用的所有其他治疗方法相比,乐伐替尼联合派博利珠单抗治疗的成本更高,获益更少.对于中/低风险亚组,与卡博替尼和纳武单抗联合ipilimumab治疗相比,乐伐替尼联合派博利珠单抗治疗成本更高,获益更多.
    Lenvatinib+pembrolizumab与舒尼替尼比较的高质量临床有效性证据可从CLEAR试验中获得。对于大多数评估组贝叶斯风险比网络荟萃分析比较,由于试验中违反比例风险或不确定比例风险假设的有效性,因此很难得出结论.然而,用于填充经济模型的数据(临床效果和成本效果)与NHS临床实践相关,可用于告知美国国家健康与护理卓越研究所的决策.评估小组的成本效益结果,使用所有药物的标价生成,显示lenvatinib联合pembrolizumab的成本效益低于所有其他治疗方案。
    本研究注册为PROSPEROCRD4202128587。
    该奖项由美国国家卫生与护理研究所(NIHR)证据综合计划(NIHR奖项参考:NIHR134985)资助,并在《卫生技术评估》中全文发表;卷。28号49.有关更多奖项信息,请参阅NIHR资助和奖励网站。
    肾细胞癌是最常见的肾癌类型。有几种药物治疗选择可用于患有晚期或转移性疾病的NHS患者,治疗的选择取决于患者疾病进展的风险。一种新的药物组合,lenvatinib加pembrolizumab,可能很快就可以用于治疗NHS患者。这篇综述探讨了lenvatinib联合pembrolizumab治疗是否为NHS提供了物有所值。我们回顾了乐伐替尼联合派姆单抗治疗与其他NHS治疗方案的有效性。我们还估计了lenvatinib联合pembrolizumab治疗与目前的NHS治疗对疾病进展风险较高和较低的患者的成本和收益。与目前的NHS治疗相比,乐伐替尼联合派博利珠单抗治疗可能会增加疾病进展风险较高(即疾病恶化)的患者存活时间.然而,对于疾病进展风险较低的患者,现有证据有限,仅表明乐伐替尼联合派姆单抗治疗可延长患者病情稳定的时间.对于所有患者来说,与目前所有的NHS治疗方法相比,lenvatinib联合pembrolizumab治疗非常昂贵.与当前NHS治疗未经治疗的肾细胞癌相比,使用公布的价格(不包括向NHS提供的任何折扣),lenvatinib联合pembrolizumab治疗可能无法为NHS提供良好的物有所值.
    UNASSIGNED: Renal cell carcinoma is the most common type of kidney cancer, comprising approximately 85% of all renal malignancies. Patients with advanced renal cell carcinoma are the focus of this National Institute for Health and Care Excellence multiple technology appraisal. A patient\'s risk of disease progression depends on a number of prognostic risk factors; patients are categorised as having intermediate/poor risk or favourable risk of disease progression.
    UNASSIGNED: The objectives of this multiple technology appraisal were to appraise the clinical effectiveness and cost-effectiveness of lenvatinib plus pembrolizumab versus relevant comparators listed in the final scope issued by the National Institute for Health and Care Excellence: sunitinib, pazopanib, tivozanib, cabozantinib and nivolumab plus ipilimumab.
    UNASSIGNED: The assessment group carried out clinical and economic systematic reviews and assessed the clinical and cost-effectiveness evidence submitted by Eisai, Hatfield, Hertfordshire, UK (the manufacturer of lenvatinib) and Merck Sharp & Dohme, Whitehouse Station, NJ, USA (the manufacturer of pembrolizumab). The assessment group carried out fixed-effects network meta-analyses using a Bayesian framework to generate evidence for clinical effectiveness. As convergence issues occurred due to sparse data, random-effects network meta-analysis results were unusable. The assessment group did not develop a de novo economic model, but instead modified the partitioned survival model provided by Merck Sharp & Dohme.
    UNASSIGNED: The assessment group clinical systematic review identified one relevant randomised controlled trial (CLEAR trial). The CLEAR trial is a good-quality, phase III, multicentre, open-label trial that provided evidence for the efficacy and safety of lenvatinib plus pembrolizumab compared with sunitinib. The assessment group progression-free survival network meta-analysis results for all three risk groups should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons owing to within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption. The assessment group overall survival network meta-analysis results for the intermediate-/poor-risk subgroup suggested that there was a numerical, but not statistically significant, improvement in the overall survival for patients treated with lenvatinib plus pembrolizumab compared with patients treated with cabozantinib or nivolumab plus ipilimumab. Because of within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption, the assessment group overall survival network meta-analysis results for the favourable-risk subgroup and the all-risk population should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons. Only one cost-effectiveness study was included in the assessment group review of cost-effectiveness evidence. The study was limited to the all-risk population, undertaken from the perspective of the US healthcare system and included comparators that are not recommended by the National Institute for Health and Care Excellence for patients with untreated advanced renal cell carcinoma. Therefore, the extent to which resource use and results are generalisable to the NHS is unclear. The assessment group cost-effectiveness results from the modified partitioned survival model focused on the intermediate-/poor-risk and favourable-risk subgroups. The assessment group cost-effectiveness results, generated using list prices for all drugs, showed that, for all comparisons in the favourable-risk subgroup, treatment with lenvatinib plus pembrolizumab costs more and generated fewer benefits than all other treatments available to NHS patients. For the intermediate-/poor-risk subgroup, treatment with lenvatinib plus pembrolizumab costs more and generated more benefits than treatment with cabozantinib and nivolumab plus ipilimumab.
    UNASSIGNED: Good-quality clinical effectiveness evidence for the comparison of lenvatinib plus pembrolizumab with sunitinib is available from the CLEAR trial. For most of the assessment group Bayesian hazard ratio network meta-analysis comparisons, it is difficult to reach conclusions due to within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption. However, the data (clinical effectiveness and cost-effectiveness) used to populate the economic model are relevant to NHS clinical practice and can be used to inform National Institute for Health and Care Excellence decision-making. The assessment group cost-effectiveness results, generated using list prices for all drugs, show that lenvatinib plus pembrolizumab is less cost-effective than all other treatment options.
    UNASSIGNED: This study is registered as PROSPERO CRD4202128587.
    UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis Programme (NIHR award ref: NIHR134985) and is published in full in Health Technology Assessment; Vol. 28, No. 49. See the NIHR Funding and Awards website for further award information.
    Renal cell carcinoma is the most common type of kidney cancer. Several drug treatment options are available for NHS patients with advanced or metastatic disease, and the choice of treatment varies depending on a patient’s risk of disease progression. A new drug combination, lenvatinib plus pembrolizumab, may soon become available to treat NHS patients. This review explored whether treatment with lenvatinib plus pembrolizumab offered value for money to the NHS. We reviewed the effectiveness of treatment with lenvatinib plus pembrolizumab versus other NHS treatment options. We also estimated the costs and benefits of treatment with lenvatinib plus pembrolizumab versus current NHS treatments for patients with higher and lower risks of disease progression. Compared with current NHS treatments, treatment with lenvatinib plus pembrolizumab may increase the time that people with a higher risk of disease progression (i.e. worsening disease) were alive. However, for patients with a lower risk of disease progression, the available evidence is limited and only shows that treatment with lenvatinib plus pembrolizumab may prolong the time that patients have a stable level of disease. For all patients, compared to all current NHS treatments, treatment with lenvatinib plus pembrolizumab is very expensive. Compared with current NHS treatments for untreated renal cell carcinoma, using published prices (which do not include any discounts that are offered to the NHS), treatment with lenvatinib plus pembrolizumab may not provide good value for money to the NHS.
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  • 文章类型: Journal Article
    林奇综合征是一种遗传性疾病,导致结直肠的风险增加,子宫内膜癌和卵巢癌。一旦完成生育,通常建议进行降低风险的手术来控制妇科癌症的风险。妇科结肠镜检查作为一种临时措施或代替降低风险的手术的价值尚不确定。我们旨在确定妇科监测在林奇综合征中是否有效和具有成本效益。
    我们对Lynch综合征妇科癌症监测的有效性和成本效益进行了系统评价,以及与癌症和妇科风险降低相关的健康效用值的系统评价。研究识别包括书目数据库搜索和引文追踪(搜索于2021年8月3日更新)。纳入资格的筛选和评估由独立研究人员进行。预后是预先指定的,并由临床专家和患者参与告知。进行了数据提取和质量评估,并对结果进行了叙述综合。我们还使用离散事件模拟方法开发了Lynch综合征的全病经济模型,包括结直肠的自然史成分,子宫内膜癌和卵巢癌,我们使用该模型对妇科风险管理策略进行了成本效用分析,包括监视,降低手术风险,无所事事。
    我们发现30项临床有效性研究,其中20项为非比较(单臂)研究。没有高质量的研究提供低偏倚风险的精确结果估计。有证据表明,监测的死亡率高于降低风险的手术,但没有监测的死亡率也高于监测的死亡率。通过监测发现了一些无症状的癌症,但也错过了一些癌症。有各种各样的疼痛经历,包括一些人感觉不到疼痛,一些人感觉剧烈疼痛。使用止痛药(例如布洛芬)很常见,一些妇女接受了全身麻醉监测。现有的经济评估清楚地发现,降低风险的手术可带来最佳的终生健康(使用质量调整的寿命年衡量),并且具有成本效益,而相比之下,监测并不划算。我们的经济评估发现,单独监测或提供监测和降低风险的手术策略具有成本效益,除了path_PMS2林奇综合征。仅提供降低风险的手术不如提供有或没有手术的监视有效。
    由于缺乏高质量的研究,无法得出关于临床有效性的确切结论。我们没有假设女性会立即接受降低风险的手术,如果手术在提供时接受,降低风险的手术可能会更有效和更具成本效益。
    根据临床理由,没有足够的证据推荐或反对林奇综合征的妇科癌症监测,但是建模表明,监控可能具有成本效益。需要进一步的研究,但它必须严格设计和良好的报告是有益的。
    本研究注册为PROSPEROCRD42020171098。
    该奖项由美国国家卫生与护理研究所(NIHR)卫生技术评估计划(NIHR奖参考:NIHR129713)资助,并在《卫生技术评估》中全文发表;卷。28号41.有关更多奖项信息,请参阅NIHR资助和奖励网站。
    林奇综合征是一种遗传性疾病,它使人们患肠癌的风险更高,子宫癌和卵巢癌。尽管患有林奇综合症的人更容易患上这些癌症,如果他们得了癌症,他们更有可能存活下来。被诊断患有林奇综合征的人使用相机进行定期测试(监视),以检查肠癌或息肉。对于子宫和卵巢癌,监视也可能是一种选择,但在这些癌症中研究较少。这意味着许多妇女没有被监视。患有Lynch综合征的女性被建议在风险开始上升时进行降低风险的手术,如果他们不想要更多的孩子。我们想知道子宫和卵巢癌的监测是否有效,是否物有所值。医生和患者说,这些都是重要的研究问题。我们搜索了关于这个主题的已发表的研究,发现了很多研究,但是这些研究通常很小或设计得不好,所以他们只能告诉我们有限的数量。研究并不总是衡量患者想知道的事情。有一些证据表明,有监视的人可能比没有监视的人寿命更长,但也有一些证据表明,降低风险的手术比监测更好。监测发现了一些没有症状的癌症,但是在一次监视访问后不久,也没有发现任何癌症。人们经常觉得监视很痛苦,但经验各不相同。我们的工作表明,对于许多患有林奇综合征的女性来说,监测和手术可能是物有所值的。我们需要更好的研究来帮助患者和医生确定监视是否适合他们。
    UNASSIGNED: Lynch syndrome is an inherited condition which leads to an increased risk of colorectal, endometrial and ovarian cancer. Risk-reducing surgery is generally recommended to manage the risk of gynaecological cancer once childbearing is completed. The value of gynaecological colonoscopic surveillance as an interim measure or instead of risk-reducing surgery is uncertain. We aimed to determine whether gynaecological surveillance was effective and cost-effective in Lynch syndrome.
    UNASSIGNED: We conducted systematic reviews of the effectiveness and cost-effectiveness of gynaecological cancer surveillance in Lynch syndrome, as well as a systematic review of health utility values relating to cancer and gynaecological risk reduction. Study identification included bibliographic database searching and citation chasing (searches updated 3 August 2021). Screening and assessment of eligibility for inclusion were conducted by independent researchers. Outcomes were prespecified and were informed by clinical experts and patient involvement. Data extraction and quality appraisal were conducted and results were synthesised narratively. We also developed a whole-disease economic model for Lynch syndrome using discrete event simulation methodology, including natural history components for colorectal, endometrial and ovarian cancer, and we used this model to conduct a cost-utility analysis of gynaecological risk management strategies, including surveillance, risk-reducing surgery and doing nothing.
    UNASSIGNED: We found 30 studies in the review of clinical effectiveness, of which 20 were non-comparative (single-arm) studies. There were no high-quality studies providing precise outcome estimates at low risk of bias. There is some evidence that mortality rate is higher for surveillance than for risk-reducing surgery but mortality is also higher for no surveillance than for surveillance. Some asymptomatic cancers were detected through surveillance but some cancers were also missed. There was a wide range of pain experiences, including some individuals feeling no pain and some feeling severe pain. The use of pain relief (e.g. ibuprofen) was common, and some women underwent general anaesthetic for surveillance. Existing economic evaluations clearly found that risk-reducing surgery leads to the best lifetime health (measured using quality-adjusted life-years) and is cost-effective, while surveillance is not cost-effective in comparison. Our economic evaluation found that a strategy of surveillance alone or offering surveillance and risk-reducing surgery was cost-effective, except for path_PMS2 Lynch syndrome. Offering only risk-reducing surgery was less effective than offering surveillance with or without surgery.
    UNASSIGNED: Firm conclusions about clinical effectiveness could not be reached because of the lack of high-quality research. We did not assume that women would immediately take up risk-reducing surgery if offered, and it is possible that risk-reducing surgery would be more effective and cost-effective if it was taken up when offered.
    UNASSIGNED: There is insufficient evidence to recommend for or against gynaecological cancer surveillance in Lynch syndrome on clinical grounds, but modelling suggests that surveillance could be cost-effective. Further research is needed but it must be rigorously designed and well reported to be of benefit.
    UNASSIGNED: This study is registered as PROSPERO CRD42020171098.
    UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: NIHR129713) and is published in full in Health Technology Assessment; Vol. 28, No. 41. See the NIHR Funding and Awards website for further award information.
    Lynch syndrome is an inherited condition which puts people at a higher risk of getting bowel cancer, womb cancer and ovarian cancer. Although people with Lynch syndrome are more likely to get these cancers, they are more likely to survive cancer if they get it. People diagnosed with Lynch syndrome get regular testing (surveillance) using a camera to check for bowel cancer or polyps. For womb and ovarian cancer, surveillance may also be an option, but it is less well studied in these cancers. This means that many women are not offered surveillance. Women with Lynch syndrome are recommended to have risk-reducing surgery when their risk starts rising, if they do not want any more children. We wanted to find out whether surveillance for womb and ovarian cancer would work and would be good value for money. Doctors and patients have said that these are important research questions. We searched for published research on this subject and found a lot of studies, but these studies were often small or not well designed, so they could only tell us a limited amount. Studies did not always measure the things that patients want to know. There was some evidence that people having surveillance might live longer than people not having surveillance, but there was also some evidence that risk-reducing surgery is better than surveillance. Surveillance has detected some cancers which had no symptoms, but there are also cancers diagnosed soon after a surveillance visit where nothing was found. People often find surveillance painful, but experiences vary. Our work shows that surveillance and surgery could be good value for money for many women with Lynch syndrome. We need better research to help patients and doctors decide whether surveillance is right for them.
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  • 文章类型: Journal Article
    成本效益分析(CEA)在医疗保健决策和资源分配中很重要;然而,近年来,将CEA的范围扩大到传统的临床经济学概念之外,还包括健康公平等价值要素,这引起了人们的极大兴趣。这项总括性审查旨在综合有关在修改后的CEA类型中如何考虑公平概念的证据。2024年1月25日搜索了MEDLINE中的公开文章,以确定自2013年以来以英文发布的系统评价(SLR),其中纳入了CEA中的健康公平考虑。标题/摘要,全文文章筛选和数据提取由一名评审员进行,并由另一名评审员进行验证.定性合成结果以确定共同主题。包括八个SLR。分布式CEA(DCEA),基于股权的权重,扩展CEA(ECEA),数学规划和多准则决策分析(MCDA)是讨论最多的方法。有人强调,在将卫生公平纳入CEA的最佳方法上缺乏共识,因为这些方法目前在决策中并不一致。重要的限制包括缺乏可靠的数据来告知健康股票指数,与常用的健康结果指标相关的偏见,以及考虑其他环境因素(如公平性和机会成本)的挑战。由于数据不可用,扩大CEA以解决公平问题的建议带来了挑战,方法复杂性,决策者对这些方法并不熟悉。我们的审查表明,扩展和分布的CEA可以通过捕获不公平对治疗的临床和成本效益评估的影响来支持决策。尽管未来的建模应考虑其他背景因素,如公平性和机会成本。关于今后采取行动的建议包括对与公平有关的结果的数据收集进行标准化,并熟悉方法,以解决将健康公平考虑因素纳入CEA的复杂性。
    Cost-effectiveness analyses (CEA) are important in healthcare decision-making and resource allocation; however, expanding the scope of CEAs beyond the traditional clinicoeconomic concepts to also include value elements such as health equity has attracted much interest in recent years. This umbrella review aimed to synthesize evidence on how equity concepts have been considered in modified types of CEAs. Publicly available articles in MEDLINE were searched on January 25, 2024, to identify systematic reviews (SLRs) published in English since 2013 that incorporate health equity considerations in CEAs. Title/abstract, full-text article screening and data extraction were conducted by a single reviewer and validated by a second reviewer. Results were qualitatively synthesized to identify common themes. Eight SLRs were included. Distributional CEAs (DCEA), equity-based weighting, extended CEA (ECEA), mathematical programming and multi-criteria decision analysis (MCDA) were the most discussed approaches. A lack of consensus on the best approach for incorporating health equity into CEAs was highlighted, as these approaches are not currently consistently used in decision-making. Important limitations included scarcity of robust data to inform health equity indices, bias associated with commonly used health outcome metrics and the challenge of accounting for additional contextual factors such as fairness and opportunity costs. Proposals to expand CEAs to address equity issues come with challenges due to data unavailability, methods complexity, and decision-makers unfamiliarity with these approaches. Our review indicates that extended and distributional CEAs can support decision-making by capturing the impact of inequity on the clinical and cost-effectiveness assessment of treatments, although future modeling should account for additional contextual factors such as fairness and opportunity costs. Recommendations for actions moving forward include standardization of data collection for outcomes related to equity and familiarity with methodologies to account for the complexities of integrating health equity considerations in CEAs.
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  • 文章类型: Journal Article
    用药错误,可能造成伤害和造成伤害,在重症监护环境中照顾的新生儿显着增加。在这个意义上,这项工作进行了系统的审查,以分析与新生儿重症监护中的用药错误有关的最新证据,讨论涉及智能泵健康技术的主题,药物的成本效益,护理专业人员对用药过程和质量改进模式的实践。这样,它可以被认为是提高新生儿重症监护质量和安全性的有用工具。
    Medication errors, potentially causing harm and causing harm, increase significantly in newborns cared for in intensive care settings. In this sense, this work carries out a systematic review to analyze the most current evidence in relation to medication errors in neonatal intensive care, discussing the topics that refer to health technology from smart pumps, cost-effectiveness of medications, the practice of nursing professionals on the medication administration process and quality improvement models. In this way, it could be considered a useful tool to promote quality and safety in neonatal intensive care.
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  • 文章类型: Journal Article
    背景:心血管疾病(CVDs)仍然是全球死亡的主要原因,在印度也是如此。印度心血管疾病的流行迅速激增,迫切需要促进当代,可持续,和具有成本效益的干预措施,以解决心血管疾病负担。本系统综述整合了基于研究的证据,证明了可用于控制印度CVD的各种干预措施的成本效益。
    方法:数据库,即,PubMed,科克伦图书馆,Embase,和谷歌学者,搜索了30年(1991-2021年)基于印度人口的针对CVD的干预措施的经济评估数据。两名审稿人评估了这些文章的资格,并根据预定义的模板从入围文章中提取数据,包括方法论方面的量化。
    结果:总计,检查了1249项研究,其中23项完全符合全文审查的纳入标准。共有16项研究仅基于印度人口,其余(7)包括南亚/亚洲进行干预,印度是一个参与国。大多数经济评估都针对CVD的基于治疗或药物干预措施(14)。评估基于基于决策的模型(10),随机对照试验(RCT)(9),和观察性研究(4)。由于方法学方法的差异,纳入研究的成本效益比表现出不同的范围,例如学习设置的差异,人口,和研究设计的不一致。原始和初级预防措施的平均ICER(增量成本效益比)为3073.8(2022美元)和17489.9(2022美元),分别。此外,二级和三级预防的合并平均值为2029.6(2022美元).
    结论:公共卫生干预措施的经济证据正在扩大,但他们的重点仅限于药物干预。迫切需要强调原始和一级预防,以在卫生经济学决策中取得更好的结果。基于技术的干预途径需要更多的探索,以迎合像印度这样的庞大人口。
    BACKGROUND: Cardiovascular diseases (CVDs) continue to be the primary cause of mortality globally and invariably in India as well. The rapid upsurge in the prevalence of CVDs in India has created a pressing need to promote contemporary, sustainable, and cost-effective interventions to tackle the CVD burden. This systematic review integrates the research-based evidence of the cost-effectiveness of various interventions that can be adapted to control CVDs in India.
    METHODS: Databases, namely, PubMed, Cochrane Library, Embase, and Google Scholar, were searched for data on the economic evaluation of interventions targeting CVD based on the Indian population for a period of 30 years (1991-2021). Two reviewers assessed the articles for eligibility, and data were extracted from the shortlisted articles as per a predefined template, including the quantification of methodological aspects.
    RESULTS: In total, 1249 studies were examined, out of which 23 completely met the inclusion criteria for full-text review. A total of 16 studies were based solely on the Indian population, while the rest (7) included South Asia/Asia for the intervention, of which India was a participant nation. Most of the economic evaluations targeted treatment-based or pharmacological interventions (14) for CVDs. The evaluations were based on Decision-based models (10), Randomized controlled Trials (RCTs) (9), and Observational studies (4). The cost-effectiveness ratio for the included studies exhibited a diverse range due to variations in methodological approaches, such as differences in study settings, populations, and inconsistencies in study design. The mean ICER (Incremental Cost-effectiveness ratio) for primordial and primary preventions was found to be 3073.8 (US $2022) and 17489.9 (US $2022), respectively. Moreover, the combined mean value for secondary and tertiary prevention was 2029.6 (US$2022).
    CONCLUSIONS: The economic evidence of public health interventions are expanding, but their focus is restricted towards pharmacological interventions. There is an urgency to emphasize primordial and primary prevention for better outcomes in health economics decision-making. Technology- based avenues for intervention need more exploration in order to cater to a large population like India.
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  • 文章类型: Journal Article
    帕金森氏病是一种导致逐渐失去协调性和运动问题的脑部疾病。在英国,大约有145,500人患有帕金森病。左旋多巴是在早期阶段管理运动症状的最处方治疗。应每6-12个月由专科医生监测患者的疾病进展和不良反应的治疗。可穿戴设备可以通过直接监测患者的运动迟缓,提供一种新的管理方法。运动障碍,震颤等症状。它们旨在与临床判断一起使用。
    确定用于监测帕金森病的五种设备的临床和成本效益:个人动力学图,Kinesia360,KinesiaU,PDMonitor和STAT-ON。
    我们对五种设备的所有证据进行了系统评价,结果包括:诊断准确性,对决策的影响,临床结果,患者和临床医生的意见和经济结果。我们检索了截至2022年2月的MEDLINE和其他12个数据库/试验登记处。评估偏倚风险。叙事综合被用来总结所有确定的证据,因为证据不足以进行荟萃分析.其中一项试验提供了个人水平的数据,重新分析。开发了一个从头决策分析模型,以估算个人运动图和Kinesia360在5年时间范围内与英国NHS护理标准相比的成本效益。基本情况分析考虑了两种替代监测策略:一次性使用和设备的常规使用。
    57项个人动力学图谱研究,15个STAT-ON,包括Kinesia360的3个,KinesiaU的1个和PDMonitor的1个。有一些证据表明,个人运动图可以准确地测量运动迟缓和运动障碍,导致一些患者的治疗改变,使用统一帕金森病评定量表测量时,临床结果可能有所改善。STAT-ON的证据表明它可能对诊断症状有价值,但目前尚无证据表明其临床影响。Kinesia360,KinesiaU和PDMonitor的证据不足以就其在临床实践中的价值得出任何结论。与一次性和常规使用的护理标准相比,PersonalKinetiGraph的基本案例结果导致每质量调整生命年增加67,856英镑和57,877英镑的成本效益比,分别,个人动力学图对统一帕金森病评定量表III和IV域的有益影响。与一次性和常规使用的护理标准相比,Kinesia360的增量成本效益比结果为每获得质量调整生命年38,828英镑和67,203英镑,分别。
    证据的范围有限,质量通常很低。对于所有设备,除了个人动力学图,该技术的临床影响几乎没有证据。
    个人动力学图可以合理地在实践中用于监测患者症状并在需要时修改治疗。关于STAT-ON的证据太少了,Kinesia360,KinesiaU或PDMonitor确信它们在临床上有用。远程监测的成本效益似乎在很大程度上不利,在一系列替代假设中,每质量调整生命年的增量成本效益比超过30,000英镑。成本效益的主要驱动因素是患者症状改善的持久性。
    本研究注册为PROSPEROCRD42022308597。
    该奖项由美国国家卫生与护理研究所(NIHR)证据综合计划(NIHR奖项参考:NIHR135437)资助,并在《卫生技术评估》中全文发表;卷。28号30.有关更多奖项信息,请参阅NIHR资助和奖励网站。
    帕金森氏病是一种导致协调性丧失和运动问题的脑部疾病。左旋多巴是治疗早期疾病的最佳处方。患者应每6-12个月由专科医生就诊,以评估其治疗需求。可穿戴设备(如智能手表)可以通过直接监测患者的疾病症状,包括震颤和运动迟缓(运动迟缓)来帮助管理。或治疗的副作用,如不自主运动(运动障碍)。这项评估考虑了五种可穿戴设备的临床和经济价值:PersonalKinetiGraph,STAT-ON,Kinesia360,KinesiaU和PDMonitor。我们搜索了医学数据库,找到了这五种设备的所有研究。我们评估了这些研究的质量并回顾了它们的结果。我们发现了77项有关该设备的研究。有一些证据表明,个人运动图可以准确地测量运动迟缓和运动障碍,导致一些患者的治疗改变,和症状的可能改善。STAT-ON的证据表明它可能对诊断症状有价值,但目前尚无证据证明其临床价值。Kinesia360,KinesiaU和PDMonitor没有足够的证据得出任何结论。进行了经济分析,以调查使用这些技术中的任何一种是否在经济上可行。经济分析发现,相对于在NHS中实施远程监控设备的额外成本,远程监控设备产生的生活质量收益很小。因此,与目前的护理标准相比,没有一种远程监护设备物有所值.
    UNASSIGNED: Parkinson\'s disease is a brain condition causing a progressive loss of co ordination and movement problems. Around 145,500 people have Parkinson\'s disease in the United Kingdom. Levodopa is the most prescribed treatment for managing motor symptoms in the early stages. Patients should be monitored by a specialist every 6-12 months for disease progression and treatment of adverse effects. Wearable devices may provide a novel approach to management by directly monitoring patients for bradykinesia, dyskinesia, tremor and other symptoms. They are intended to be used alongside clinical judgement.
    UNASSIGNED: To determine the clinical and cost-effectiveness of five devices for monitoring Parkinson\'s disease: Personal KinetiGraph, Kinesia 360, KinesiaU, PDMonitor and STAT-ON.
    UNASSIGNED: We performed systematic reviews of all evidence on the five devices, outcomes included: diagnostic accuracy, impact on decision-making, clinical outcomes, patient and clinician opinions and economic outcomes. We searched MEDLINE and 12 other databases/trial registries to February 2022. Risk of bias was assessed. Narrative synthesis was used to summarise all identified evidence, as the evidence was insufficient for meta-analysis. One included trial provided individual-level data, which was re-analysed. A de novo decision-analytic model was developed to estimate the cost-effectiveness of Personal KinetiGraph and Kinesia 360 compared to standard of care in the UK NHS over a 5-year time horizon. The base-case analysis considered two alternative monitoring strategies: one-time use and routine use of the device.
    UNASSIGNED: Fifty-seven studies of Personal KinetiGraph, 15 of STAT-ON, 3 of Kinesia 360, 1 of KinesiaU and 1 of PDMonitor were included. There was some evidence to suggest that Personal KinetiGraph can accurately measure bradykinesia and dyskinesia, leading to treatment modification in some patients, and a possible improvement in clinical outcomes when measured using the Unified Parkinson\'s Disease Rating Scale. The evidence for STAT-ON suggested it may be of value for diagnosing symptoms, but there is currently no evidence on its clinical impact. The evidence for Kinesia 360, KinesiaU and PDMonitor is insufficient to draw any conclusions on their value in clinical practice. The base-case results for Personal KinetiGraph compared to standard of care for one-time and routine use resulted in incremental cost-effectiveness ratios of £67,856 and £57,877 per quality-adjusted life-year gained, respectively, with a beneficial impact of the Personal KinetiGraph on Unified Parkinson\'s Disease Rating Scale domains III and IV. The incremental cost-effectiveness ratio results for Kinesia 360 compared to standard of care for one-time and routine use were £38,828 and £67,203 per quality-adjusted life-year gained, respectively.
    UNASSIGNED: The evidence was limited in extent and often low quality. For all devices, except Personal KinetiGraph, there was little to no evidence on the clinical impact of the technology.
    UNASSIGNED: Personal KinetiGraph could reasonably be used in practice to monitor patient symptoms and modify treatment where required. There is too little evidence on STAT-ON, Kinesia 360, KinesiaU or PDMonitor to be confident that they are clinically useful. The cost-effectiveness of remote monitoring appears to be largely unfavourable with incremental cost-effectiveness ratios in excess of £30,000 per quality-adjusted life-year across a range of alternative assumptions. The main driver of cost-effectiveness was the durability of improvements in patient symptoms.
    UNASSIGNED: This study is registered as PROSPERO CRD42022308597.
    UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis programme (NIHR award ref: NIHR135437) and is published in full in Health Technology Assessment; Vol. 28, No. 30. See the NIHR Funding and Awards website for further award information.
    Parkinson’s disease is a brain condition causing loss of co-ordination and movement problems. Levodopa is the most prescribed treatment for early disease. Patients should be seen by a specialist every 6–12 months to assess their treatment needs. Wearable devices (like smart watches) may aid management by directly monitoring patients for disease symptoms including tremor and slowness of movement (bradykinesia), or side effects of treatment like involuntary movement (dyskinesia). This assessment considered the clinical and economic value of five wearable devices: Personal KinetiGraph, STAT-ON, Kinesia 360, KinesiaU and PDMonitor. We searched medical databases to find all studies of the five devices. We assessed the quality of these studies and reviewed their results. We found 77 studies of the devices. There was some evidence to suggest that Personal KinetiGraph can accurately measure bradykinesia and dyskinesia, leading to treatment modification in some patients, and a possible improvement in symptoms. The evidence for STAT-ON suggested it may be of value for diagnosing symptoms, but there is currently no evidence on its clinical value. There was insufficient evidence for Kinesia 360, KinesiaU and PDMonitor to draw any conclusions. An economic analysis was conducted to investigate whether using any of these technologies is economically viable. The economic analysis found that the quality-of-life benefits generated by remote monitoring devices were small relative to the additional costs of implementing them in the NHS. As such, none of the remote monitoring devices were good value for money when compared with the current standard of care.
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  • 文章类型: Journal Article
    康复技术为运动障碍患者的干预提供了有希望的机会。然而,由于它们的高成本和对其成本效益的持续怀疑,它们在常规护理中的使用仍然有限。提供康复技术经济效率的确凿证据将有助于消除这些疑虑,以便更好地利用这些技术。在这种情况下,本系统综述旨在研究基于数字技术使用的康复干预措施的成本效益.总的来说,确定了2011年至2021年之间发表的660篇文章,其中11项研究符合所有纳入标准。在这11项研究中,七个被证明具有成本效益,而四个没有。四项研究使用了成本效用分析(CUA),七项使用了成本最小化分析(CMA)。大多数(十项研究)集中在中风后上肢和/或下肢的康复,而只有一项研究检查了膝关节置换术后下肢的康复情况。关于评估的设备,七项研究分析了机器人康复的成本效益,四项分析了虚拟现实康复。使用CHEERS(综合卫生经济评估报告标准)对纳入研究的质量进行评估表明,质量与经济分析方法有关:所有采用成本效用分析的研究都获得了高质量分数(超过80%),而成本最小化分析的质量分数是平均值,CMA获得的最高分为72%。所有文章的平均质量得分为75%,在52到100之间。在四项有考虑分数的研究中,两个人的结论是,在成本效益方面,干预和常规护理是等效的,一个人得出结论,干预占主导地位,而最后一个结论是通常的护理占主导地位。这表明,即使考虑到纳入研究的质量,基于数字技术的康复干预措施仍然具有成本效益,它们改善了运动障碍患者的健康结果和生活质量,同时还可以节省成本。
    Rehabilitation technologies offer promising opportunities for interventions for patients with motor disabilities. However, their use in routine care remains limited due to their high cost and persistent doubts about their cost-effectiveness. Providing solid evidence of the economic efficiency of rehabilitation technologies would help dispel these doubts in order to better take advantage of these technologies. In this context, this systematic review aimed to examine the cost-effectiveness of rehabilitation interventions based on the use of digital technologies. In total, 660 articles published between 2011 and 2021 were identified, of which eleven studies met all the inclusion criteria. Of these eleven studies, seven proved to be cost-effective, while four were not. Four studies used cost-utility analyses (CUAs) and seven used cost-minimization analyses (CMAs). The majority (ten studies) focused on the rehabilitation of the upper and/or lower limbs after a stroke, while only one study examined the rehabilitation of the lower limbs after knee arthroplasty. Regarding the evaluated devices, seven studies analyzed the cost-effectiveness of robotic rehabilitation and four analyzed rehabilitation with virtual reality.The assessment of the quality of the included studies using the CHEERS (Consolidated Health Economic Evaluation Reporting Standards) suggested that the quality was related to the economic analysis method: all studies that adopted a cost-utility analysis obtained a high quality score (above 80%), while the quality scores of the cost-minimization analyses were average, with the highest score obtained by a CMA being 72%. The average quality score of all the articles was 75%, ranging between 52 and 100. Of the four studies with a considering score, two concluded that there was equivalence between the intervention and conventional care in terms of cost-effectiveness, one concluded that the intervention dominated, while the last one concluded that usual care dominated. This suggests that even considering the quality of the included studies, rehabilitation interventions based on digital technologies remain cost-effective, they improved health outcomes and quality of life for patients with motor disorders while also allowing cost savings.
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  • 文章类型: Journal Article
    卫生经济评估用于确定产前或新生儿筛查计划产生净收益所需的资源,在多重利益和危害的驱使下,是合理的。目前尚不清楚评估这些方案的经济评价采取了哪些利弊,以及它们是否忽略了对相关利益攸关方重要的利弊。
    (1)确定卫生经济评估在该领域采用的益处和危害,并评估它们是如何被衡量和评估的;(2)确定未来经济评估中应考虑的属性或与利益相关者的相关性;(3)就这些研究应考虑的利益和危害提出建议。
    将系统回顾和定性工作相结合的混合方法。
    我们使用所有主要的电子数据库搜索了2000年1月至2021年1月的已发布和灰色文献。一个或多个经济合作与发展组织国家的产前或新生儿筛查计划的经济评估被认为是合格的。使用综合卫生经济评估报告标准清单评估报告质量。我们使用综合描述性分析确定了利弊,并构建了主题框架。
    我们对新生儿筛查经验的现有文献进行了元人种学研究,对与产前或新生儿筛查或生活在筛查条件下相关的现有个人访谈的二次分析,以及与利益相关者一起收集的有关其筛查经验的主要数据的主题分析。
    文献检索确定了52,244篇文章和报告,并纳入336项独特研究。专题框架产生了七个主题:(1)诊断筛查条件,(2)生命年和健康状况调整,(3)治疗,(4)长期成本,(5)过度诊断,(6)妊娠损失和(7)对家庭成员的溢出效应。筛查条件的诊断(115,47.5%),生命年和健康状况调整(90,37.2%)和治疗(88,36.4%)占评估产前筛查的大部分益处和危害。相同的主题占了评估新生儿筛查的研究中的大部分益处和危害。长期成本,过度诊断和溢出效应往往被忽视。筛查的广泛家庭影响被认为对利益相关者很重要。我们观察到专题框架和定性证据之间有很好的重叠。
    由于纳入了大量研究,因此在系统文献综述中提取双重数据是不可行的。很难在利益相关者的面试中招募医疗保健专业人员。
    在该领域的卫生经济评估中使用的益处和危害的选择没有一致性,建议需要额外的方法指导。我们提出的主题框架可用于指导未来卫生经济评估的发展,以评估产前和新生儿筛查计划。
    本研究注册为PROSPEROCRD42020165236。
    该奖项由美国国家卫生与护理研究所(NIHR)卫生技术评估计划(NIHR奖参考:NIHR127489)资助,并在《卫生技术评估》中全文发表;卷。28号25.有关更多奖项信息,请参阅NIHR资助和奖励网站。
    NHS每年都会为孕妇提供筛查测试,以评估她们或未出生的婴儿患有或发展为健康状况的机会。它还为新生婴儿提供筛查测试,以寻找一系列健康状况。筛查计划的实施以及对妇女和婴儿的护理需要NHS的许多资源和资金,因此,重要的是筛选程序代表物有所值。这意味着NHS在计划上花费的金额由该计划提供的收益来证明。我们想看看研究人员在计算物有所值时是否考虑了与孕妇和新生儿筛查相关的所有重要益处和危害。要做到这一点,我们搜索了发达国家的所有研究,以确定他们认为的益处和危害.我们还考虑了父母和医疗保健专业人员对为家庭和更广泛的社会创造的好处和危害筛查的意见。我们发现,筛查的益处和危害的识别是复杂的,因为筛查结果会影响一系列人群(母婴,父母,大家庭和更广泛的社会)。研究人员计算筛查项目的物有所值,到目前为止,集中在狭窄范围的益处和危害上,而忽略了许多对筛查结果影响的人很重要的因素。从我们与父母和医疗保健专业人员的讨论中,我们发现,对家庭的更广泛影响是一个重要的考虑因素。我们研究的只有一项研究考虑了对家庭的更广泛影响。我们的工作还发现父母的识别能力,吸收和应用新的信息,以了解他们的孩子的筛查结果或条件是重要的。参与筛查的医疗保健专业人员在支持患有某种疾病的儿童家庭时应考虑这一点。我们为研究人员创建了一份清单,以确定未来研究中重要的益处和危害。我们还确定了研究人员评估这些益处和危害的不同方式,所以他们以一种有意义的方式融入到他们的研究中。
    UNASSIGNED: Health economic assessments are used to determine whether the resources needed to generate net benefit from an antenatal or newborn screening programme, driven by multiple benefits and harms, are justifiable. It is not known what benefits and harms have been adopted by economic evaluations assessing these programmes and whether they omit benefits and harms considered important to relevant stakeholders.
    UNASSIGNED: (1) To identify the benefits and harms adopted by health economic assessments in this area, and to assess how they have been measured and valued; (2) to identify attributes or relevance to stakeholders that ought to be considered in future economic assessments; and (3) to make recommendations about the benefits and harms that should be considered by these studies.
    UNASSIGNED: Mixed methods combining systematic review and qualitative work.
    UNASSIGNED: We searched the published and grey literature from January 2000 to January 2021 using all major electronic databases. Economic evaluations of an antenatal or newborn screening programme in one or more Organisation for Economic Co-operation and Development countries were considered eligible. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist. We identified benefits and harms using an integrative descriptive analysis and constructed a thematic framework.
    UNASSIGNED: We conducted a meta-ethnography of the existing literature on newborn screening experiences, a secondary analysis of existing individual interviews related to antenatal or newborn screening or living with screened-for conditions, and a thematic analysis of primary data collected with stakeholders about their experiences with screening.
    UNASSIGNED: The literature searches identified 52,244 articles and reports, and 336 unique studies were included. Thematic framework resulted in seven themes: (1) diagnosis of screened for condition, (2) life-years and health status adjustments, (3) treatment, (4) long-term costs, (5) overdiagnosis, (6) pregnancy loss and (7) spillover effects on family members. Diagnosis of screened-for condition (115, 47.5%), life-years and health status adjustments (90, 37.2%) and treatment (88, 36.4%) accounted for most of the benefits and harms evaluating antenatal screening. The same themes accounted for most of the benefits and harms included in studies assessing newborn screening. Long-term costs, overdiagnosis and spillover effects tended to be ignored. The wide-reaching family implications of screening were considered important to stakeholders. We observed good overlap between the thematic framework and the qualitative evidence.
    UNASSIGNED: Dual data extraction within the systematic literature review was not feasible due to the large number of studies included. It was difficult to recruit healthcare professionals in the stakeholder\'s interviews.
    UNASSIGNED: There is no consistency in the selection of benefits and harms used in health economic assessments in this area, suggesting that additional methods guidance is needed. Our proposed thematic framework can be used to guide the development of future health economic assessments evaluating antenatal and newborn screening programmes.
    UNASSIGNED: This study is registered as PROSPERO CRD42020165236.
    UNASSIGNED: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: NIHR127489) and is published in full in Health Technology Assessment; Vol. 28, No. 25. See the NIHR Funding and Awards website for further award information.
    Every year the NHS offers pregnant women screening tests to assess the chances of them or their unborn baby having or developing a health condition. It also offers screening tests for newborn babies to look for a range of health conditions. The implementation of screening programmes and the care for women and babies require many resources and funding for the NHS, so it is important that screening programmes represent good value for money. This means that the amount of money the NHS spends on a programme is justified by the amount of benefit that the programme gives. We wanted to see whether researchers consider all the important benefits and harms associated with screening of pregnant women and newborn babies when calculating value for money. To do this, we searched all studies available in developed countries to identify what benefits and harms they considered. We also considered the views of parents and healthcare professionals on the benefits and harms screening that creates for families and wider society. We found that the identification of benefits and harms of screening is complex because screening results affect a range of people (mother–baby, parents, extended family and wider society). Researchers calculating the value for money of screening programmes have, to date, concentrated on a narrow range of benefits and harms and ignored many factors that are important to people affected by screening results. From our discussions with parents and healthcare professionals, we found that wider impacts on families are an important consideration. Only one study we looked at considered wider impacts on families. Our work also found that parent’s ability to recognise, absorb and apply new information to understand their child’s screening results or condition is important. Healthcare professionals involve in screening should consider this when supporting families of children with a condition. We have created a list for researchers to identify the benefits and harms that are important to include in future studies. We have also identified different ways researchers can value these benefits and harms, so they are incorporated into their studies in a meaningful way.
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  • 文章类型: Journal Article
    目的:先前的工作确定了六个关键的价值要素(治疗属性和期望的结果),为患有重度抑郁症(MDD)的人管理他们的病情:治疗模式,帮助治疗的时间,MDD救济,工作质量,与他人互动,和负担能力。我们研究的目的是确定以前的MDD治疗成本效益分析(CEA)是否解决了这些价值因素。次要目标是确定是否有任何研究涉及患者,家庭成员,模型开发过程中的护理人员。
    方法:我们进行了系统的文献综述,以确定已发布的基于模型的CEA。我们将已发表研究的要素与先前工作中得出的MDD患者价值要素进行了比较,以确定未来研究的差距和领域。
    结果:在86个已发布的CEA中,我们发现七个包括病人自付费用,32包括生产率的衡量标准,这两个都是MDD患者的优先事项。我们发现,只有两项研究从患者那里获得了模型的度量,而两项研究则使患者参与了建模过程。
    结论:已发布的用于MDD治疗的CEA模型通常不包括作为该患者群体优先考虑的价值元素,他们在建模过程中也不包括患者。需要能够适应与患者经验一致的元素的灵活模型,并且多利益相关者参与方法将有助于实现这一目标。
    OBJECTIVE: Prior work identified 6 key value elements (attributes of treatment and desired outcomes) for individuals living with major depressive disorder (MDD) in managing their condition: mode of treatment, time to treatment helpfulness, MDD relief, quality of work, interaction with others, and affordability. The objective of our study was to identify whether previous cost-effectiveness analyses (CEAs) for MDD treatment addressed any of these value elements. A secondary objective was to identify whether any study engaged patients, family members, and caregivers in the model development process.
    METHODS: We conducted a systematic literature review to identify published model-based CEAs. We compared the elements of the published studies with the MDD patient value elements elicited in prior work to identify gaps and areas for future research.
    RESULTS: Of 86 published CEAs, we found that 7 included patient out-of-pocket costs, and 32 included measures of productivity, which were both priorities for individuals with MDD. We found that only 2 studies elicited measures from patients for their model, and 2 studies engaged patients in the modeling process.
    CONCLUSIONS: Published CEA models for MDD treatment do not regularly include value elements that are a priority for this patient population nor do they include patients in their modeling process. Flexible models that can accommodate elements consistent with patient experience are needed, and a multistakeholder engagement approach would help accomplish this.
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