Journal of comparative effectiveness research最新文献

{"title":"EU HTA Joint Clinical Assessment: are patients with rare disease going to lose out?","authors":"Alejandra Castanon, Rebecca Sloan, Luisamanda Selle Arocha, Sreeram V Ramagopalan","doi":"10.57264/cer-2024-0052","DOIUrl":"10.57264/cer-2024-0052","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240052"},"PeriodicalIF":1.9,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145525/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140852029","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A clinical systematic literature review of treatments among patients with advanced and/or metastatic human epidermal growth factor receptor 2 positive breast cancer.","authors":"Kerigo Ndirangu, Rachel Goldgrub, Vanita Tongbram, Rajee Antony, Bagrat Lalayan, Joyce O'Shaughnessy, Sarah E Schellhorn","doi":"10.57264/cer-2023-0153","DOIUrl":"10.57264/cer-2023-0153","url":null,"abstract":"<p><p><b>Aim:</b> This systematic literature review aims to summarize the efficacy/effectiveness of treatments, including eribulin (ERI)-based and anti-human epidermal growth factor receptor 2 (HER2) treatments in advanced/metastatic HER2+ breast cancer. <b>Methods:</b> Three databases from 2016 to September 2021 were searched for clinical trials and observational studies in patients receiving first-line (1L) standard of care (SOC), second-line (2L) SOC or third-line or subsequent lines (3L+). <b>Results:</b> 2692 citations were screened, and 38 studies were included. Eleven studies were randomized-controlled trials (RCTs; 5 in 1L, 6 in 3L+), 6 were single-arm trials (5 in 1L, 1 in 3L+) and 21 were observational studies (13 in 1L, 6 in 2L, 4 in 3L+ [note that studies with subgroups for 1L, 2L, 3L+ are double-counted]). Longer overall survival (OS) was associated with 1L and 2L treatment, and for 3L+ studies that included ERI, ERI or trastuzumab (Tmab) + ERI led to longer OS than treatments of physician's choice (median OS of 11, 10 and 8.9 months, respectively). Progression-free survival was 9 months in Tmab + pertuzumab (Pmab) + ERI, 4 months in Tmab + ERI and 3.3 months in ERI. <b>Conclusion:</b> Available treatments provide a wide range of efficacy. However, later lines lack standardization and conclusions on comparative effectiveness are limited by differing trial designs. Thus, the chance of prolonged survival with new agents warrants further research.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230153"},"PeriodicalIF":2.1,"publicationDate":"2024-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145522/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141161271","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing the performance of physician's prescribing preference as an instrumental variable in comparative effectiveness research with moderate and small sample sizes: a simulation study.","authors":"Lisong Zhang, Jim Lewsey, David A McAllister","doi":"10.57264/cer-2023-0044","DOIUrl":"https://doi.org/10.57264/cer-2023-0044","url":null,"abstract":"<p><p><b>Aim:</b> This simulation study is to assess the utility of physician's prescribing preference (PPP) as an instrumental variable for moderate and smaller sample sizes. <b>Materials & methods:</b> We designed a simulation study to imitate a comparative effectiveness research under different sample sizes. We compare the performance of instrumental variable (IV) and non-IV approaches using two-stage least squares (2SLS) and ordinary least squares (OLS) methods, respectively. Further, we test the performance of different forms of proxies for PPP as an IV. <b>Results:</b> The percent bias of 2SLS is around approximately 20%, while the percent bias of OLS is close to 60%. The sample size is not associated with the level of bias for the PPP IV approach. <b>Conclusion:</b> Irrespective of sample size, the PPP IV approach leads to less biased estimates of treatment effectiveness than OLS adjusting for known confounding only. Particularly for smaller sample sizes, we recommend constructing PPP from long prescribing histories to improve statistical power.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":"13 5","pages":"e230044"},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11036905/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140849794","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Gastrointestinal adverse effects associated with the use of intravenous oliceridine compared with intravenous hydromorphone or fentanyl in acute pain management utilizing adjusted indirect treatment comparison methods.","authors":"Joseph Biskupiak, Gary Oderda, Diana Brixner, Todd L Wandstrat","doi":"10.57264/cer-2023-0041","DOIUrl":"10.57264/cer-2023-0041","url":null,"abstract":"<p><p><b>Background:</b> In the absence of head-to-head comparative data from randomized controlled trials, indirect treatment comparisons (ITCs) may be used to compare the relative effects of treatments versus a common comparator (either placebo or active treatment). For acute pain management, the effects of oliceridine have been compared in clinical trials to morphine but not to fentanyl or hydromorphone. <b>Aim:</b> To assess the comparative safety (specifically differences in the incidence of nausea, vomiting and opioid-induced respiratory depression [OIRD]) between oliceridine and relevant comparators (fentanyl and hydromorphone) through ITC analysis. <b>Methods:</b> A systematic literature review identified randomized clinical trials with oliceridine versus morphine and morphine versus fentanyl or hydromorphone. The ITC utilized the common active comparator, morphine, for the analysis. <b>Results:</b> A total of six randomized controlled trials (oliceridine - 2; hydromorphone - 3; fentanyl - 1) were identified for data to be used in the ITC analyses. The oliceridine data were reported in two studies (plastic surgery and orthopedic surgery) and were also reported in a pooled analysis. The ITC focused on nausea and vomiting due to limited data for OIRD. When oliceridine was compared with hydromorphone in the ITC analysis, oliceridine significantly reduced the incidence of nausea and/or vomiting requiring antiemetics compared with hydromorphone (both orthopedic surgery and pooled data), while results in plastic surgery were not statistically significant. When oliceridine was compared with hydromorphone utilizing data from Hong, the ITC only showed a trend toward reduced risk of nausea and vomiting with oliceridine that was not statistically significant across all three comparisons (orthopedic surgery, plastic surgery and combined). An ITC comparing oliceridine with a study of fentanyl utilizing the oliceridine orthopedic surgery data and combined orthopedic and plastic surgery data showed a trend toward reduced risk that was not statistically significant. <b>Conclusion:</b> In ITC analyses, oliceridine significantly reduced the incidence of nausea and/or vomiting or the need for antiemetics in orthopedic surgery compared with hydromorphone and a non-significant trend toward reduced risk versus fentanyl.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230041"},"PeriodicalIF":1.9,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11036942/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140143635","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Letter to the editor: network meta-analysis for indirect comparison of lanadelumab and berotralstat for the treatment of hereditary angioedema.","authors":"Max Schlueter, Sandra Nestler-Parr","doi":"10.57264/cer-2023-0165","DOIUrl":"10.57264/cer-2023-0165","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230165"},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11036881/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140305793","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 15.","authors":"Alejandra Castanon, Benjamin D Bray, Sreeram V Ramagopalan","doi":"10.57264/cer-2024-0033","DOIUrl":"10.57264/cer-2024-0033","url":null,"abstract":"<p><p>In this latest update we discuss real-world evidence (RWE) guidance from the leading oncology professional societies, the American Society of Clinical Oncology and the European Society for Medical Oncology, and the PRINCIPLED practical guide on the design and analysis of causal RWE studies.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240033"},"PeriodicalIF":2.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11037032/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140305794","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early-stage hepatocellular carcinoma screening in patients with chronic hepatitis B in China: a cost-effectiveness analysis.","authors":"Yuemin Nan, Osvaldo Ulises Garay, Xianzhong Lu, Yue Zhang, Li Xie, Zhongyi Niu, Wen Chen","doi":"10.57264/cer-2023-0146","DOIUrl":"10.57264/cer-2023-0146","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the cost-effectiveness of seven screening strategies for chronic hepatitis B (CHB) patients in China. <b>Methods:</b> A discrete event simulation model combining a decision tree and Markov structure was developed to simulate a CHB cohort aged ≥40 years on a lifetime horizon and evaluate the costs and health outcomes (quality-adjusted life years [QALYs] gained) of ultrasonography (US), alpha-fetoprotein (AFP), protein induced by vitamin K absence-II (PIVKA-II), AFP+US, AFP+PIVKA-II, GAAD (a diagnostic algorithm based on gender and age combined with results of AFP and PIVKA-II) and GAAD+US. Epidemiologic, clinical performance, utility and cost data were obtained from the literature, expert interviews and real-world data. Uncertainties on key parameters were explored through deterministic and probabilistic sensitivity analyses (DSA and PSA). <b>Results:</b> Compared with other strategies, GAAD+US detected the most HCC patients at early stage, and GAAD was the screening strategy with the lowest average cost per HCC case diagnosed. Using 3× China's 2022 GDP per capita ($38,233.34) as the threshold, the three strategies of US, GAAD and GAAD+US formed a cost-effectiveness frontier. Screening with US, GAAD, or GAAD+US was associated with costs of $6110.46, $7622.05 and $8636.32, and QALYs of 13.18, 13.48 and 13.52, respectively. The ICER of GAAD over US was $4993.39/QALY and the ICER of GAAD+US over GAAD was $26,691.45/QALY, which was less than 3× GDP per capita. Both DSA and PSA proved the stability of the results. <b>Conclusion:</b> GAAD+US was the most cost-effective strategy for early HCC diagnosis among CHB patients which could be considered as the liver cancer screening scheme for the high-risk population in China.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230146"},"PeriodicalIF":2.1,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11044951/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139983049","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Use of interrupted time-series analyses in evaluating health economic outcomes following implementation of multilayer water-tight wound closure in a primary total joint arthroplasty population.","authors":"Ziyu Tan, Joerg Tomaszewski, Brian Po-Han Chen, Najmuddin J Gunja, Katherine Etter","doi":"10.57264/cer-2023-0110","DOIUrl":"10.57264/cer-2023-0110","url":null,"abstract":"<p><p><b>Aim:</b> Total joint arthroplasty (TJA) with multi-layer, watertight closure (MLWC) using knotless barbed suture and 2-octyl cyanoacrylate plus polymer mesh tape was compared with conventional closure (CC) using Vicryl™ sutures and staples. <b>Patients & methods:</b> Electronic medical records of patients undergoing TJA (1574: total knee arthroplasty; 580: total hip arthroplasty; 13: unknown) from a single surgeon at a US hospital (CC 2011 to 2013; MLWC 2015 to 2020) were reviewed. Outcomes were length of stay (LOS), discharge to skilled nursing facility (SNF), 90-day surgical site infection (SSI) and 90-day readmission. Logistic regression controlled for baseline characteristics. Adjusted interrupted time series (ITS) analyses accounted for decreasing trends in LOS and SNF discharge over time. <b>Results:</b> Among 2167 TJA cases (mean [standard deviation] age 66.0 [9.7] years, 53.3% female), 906 received CC and 1261 received MLWC. Bivariate analysis showed no statistically significant differences in 90-day SSI rates; however, MLWC patients had 60% lower 90-day readmission rates (1.5 vs 3.8%, p < 0.05), 44% lower LOS (1.4 vs 2.5 days, p < 0.05) and 40% lower discharge rates to a skilled care facility (8.5 vs 14.1%, p < 0.05). Multivariable analyses showed CC patients were 2.45-times more likely to be readmitted within 90 days, 1.88-times more likely to be discharged to SNF and had 1.67-times longer LOS compared with MLWC. ITS analyses showed a sharp decline in LOS (0.9 days) and discharge to SNF (5.6% incidence) after implementation of MLWC, followed by no further changes for the remainder of the study period. <b>Conclusion:</b> MLWC was associated with ≥40% reduction in 90-day readmission, LOS and SNF discharge compared with TJA CC. LOS and discharge rate to SNF declined sharply after the implementation of MLWC.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230110"},"PeriodicalIF":1.9,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11044954/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139983050","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Understanding economic analysis and cost-effectiveness of CT scan-guided, 3-dimensional, robotic-arm assisted lower extremity arthroplasty: a systematic review.","authors":"Kara Sarrel, Daniel Hameed, Jeremy Dubin, Michael A Mont, David J Jacofsky, Andréa B Coppolecchia","doi":"10.57264/cer-2023-0040","DOIUrl":"10.57264/cer-2023-0040","url":null,"abstract":"<p><p><b>Aim:</b> The overall goal of this review was to examine the cost-utility of robotic-arm assisted surgery versus manual surgery. <b>Methods:</b> We performed a systematic review of all health economic studies that compared CT-based robotic-arm assisted unicompartmental knee arthroplasty, total knee arthroplasty and total hip arthroplasty with manual techniques. The papers selected focused on various cost-utility measures. In addition, where appropriate, secondary aims encompassed various clinical outcomes (e.g., readmissions, discharges to subacute care, etc.). Only articles directly comparing CT-based robotic-arm assisted joint arthroplasty with manual joint arthroplasty were included, for a resulting total of 21 reports. <b>Results:</b> Almost all twenty-one studies demonstrated a positive effect of CT scan-guided robotic-assisted joint arthroplasty on health economic outcomes. For studies reporting on 90-day episodes of costs, 10 out of 12 found lower costs in the robotic-arm assisted groups. <b>Conclusion:</b> Robotic-arm assisted joint arthroplasty patients had shorter lengths of stay and cost savings based on their 90-day episodes of care, among other metrics. Payors would likely benefit from encouraging the use of this CT-based robotic technology.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230040"},"PeriodicalIF":1.9,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11044952/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140131656","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acceptability and consumers' willingness to pay for comprehensive medication management services in Brazil.","authors":"Lucas Lima Tôrres, Pâmela Santos Azevedo, Túlio Tadeu Rocha Sarmento, Djenane Ramalho-de-Oliveira, Edna Afonso Reis, Isabella Piassi Dias Godói, Augusto Afonso Guerra Júnior, Cristina Mariano Ruas","doi":"10.57264/cer-2023-0127","DOIUrl":"10.57264/cer-2023-0127","url":null,"abstract":"<p><p><b>Aim:</b> Comprehensive medication management (CMM) is a clinical service that aims to optimize the therapeutic results of patients at the individual level. Studies carried out in Brazil and in several parts of the world have found a positive impact of the service, mainly in the resolution of drug therapy problems and in improving clinical outcomes and patients' quality of life. This service is not widespread and its acceptability and willingness to pay were not defined by the population yet. <b>Objective:</b> This work aims to conduct a study with users of private health services to determine the acceptability and willingness to pay for CMM services. <b>Methods:</b> This is a cross-sectional survey conducted through face-to-face interviews, among residents over 18 years of age of the metropolitan region of Belo Horizonte, capital of Minas Gerais State, Brazil. <b>Results:</b> For this study, 563 individuals were interviewed. Most respondents were female (55.1%), had completed high school (46.8%) and were employed (62.5%). The acceptability for the service was 93,25%, and among all respondents, 37 would not accept the service even if it was free. The amount of consumers' willingness to pay for the CMM service was estimated at $17.75 (40.00 BRL). <b>Conclusion:</b> The research results show that most people are willing to pay for the CMM service. This study can contribute to the decision-making regarding the implementation and pricing of the service in Brazil.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230127"},"PeriodicalIF":2.1,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11044949/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139702616","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}