Journal of comparative effectiveness research最新文献

{"title":"Visualizing the target estimand in comparative effectiveness studies with multiple treatments.","authors":"Gabrielle Simoneau, Marian Mitroiu, Thomas Pa Debray, Wei Wei, Stan Rw Wijn, Joana Caldas Magalhães, Justin Bohn, Changyu Shen, Fabio Pellegrini, Carl de Moor","doi":"10.57264/cer-2023-0089","DOIUrl":"10.57264/cer-2023-0089","url":null,"abstract":"<p><p><b>Aim:</b> Comparative effectiveness research using real-world data often involves pairwise propensity score matching to adjust for confounding bias. We show that corresponding treatment effect estimates may have limited external validity, and propose two visualization tools to clarify the target estimand. <b>Materials & methods:</b> We conduct a simulation study to demonstrate, with bivariate ellipses and joy plots, that differences in covariate distributions across treatment groups may affect the external validity of treatment effect estimates. We showcase how these visualization tools can facilitate the interpretation of target estimands in a case study comparing the effectiveness of teriflunomide (TERI), dimethyl fumarate (DMF) and natalizumab (NAT) on manual dexterity in patients with multiple sclerosis. <b>Results:</b> In the simulation study, estimates of the treatment effect greatly differed depending on the target population. For example, when comparing treatment B with C, the estimated treatment effect (and respective standard error) varied from -0.27 (0.03) to -0.37 (0.04) in the type of patients initially receiving treatment B and C, respectively. Visualization of the matched samples revealed that covariate distributions vary for each comparison and cannot be used to target one common treatment effect for the three treatment comparisons. In the case study, the bivariate distribution of age and disease duration varied across the population of patients receiving TERI, DMF or NAT. Although results suggest that DMF and NAT improve manual dexterity at 1 year compared with TERI, the effectiveness of DMF versus NAT differs depending on which target estimand is used. <b>Conclusion:</b> Visualization tools may help to clarify the target population in comparative effectiveness studies and resolve ambiguity about the interpretation of estimated treatment effects.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230089"},"PeriodicalIF":2.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842272/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139521018","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":"Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments.","authors":"Katherine Appiah, Maria Rizzo, Grammati Sarri, Luis Hernandez","doi":"10.57264/cer-2023-0140","DOIUrl":"10.57264/cer-2023-0140","url":null,"abstract":"<p><p><b>Background:</b> The drive to expedite patient access for diseases with high unmet treatment needs has come with an increasing use of single-arm trials (SATs), especially in oncology. However, the lack of control arms in such trials creates challenges to assess and demonstrate comparative efficacy. External control (EC) arms can be used to bridge this gap, with various types of sources available to obtain relevant data. <b>Objective:</b> To examine the source of ECs in single-arm oncology health technology assessment (HTA) submissions to the National Institute for Health and Care Excellence (NICE) and the Pharmaceutical Benefits Advisory Committee (PBAC) and how this selection was justified by manufacturers and assessed by the respective HTA body. <b>Methods:</b> Single-arm oncology HTA submission reports published by NICE (England) and PBAC (Australia) from January 2011 to August 2021 were reviewed, with data qualitatively synthesized to identify themes. <b>Results:</b> Forty-eight oncology submissions using EC arms between 2011 and 2021 were identified, with most submissions encompassing blood and bone marrow cancers (52%). In HTA submissions to NICE and PBAC, the EC arm was typically constructed from a combination of data sources, with the company's justification in data source selection infrequently provided (PBAC [2 out of 19]; NICE [6 out of 29]), although this lack of justification was not heavily criticized by either HTA body. <b>Conclusion:</b> Although HTA bodies such as NICE and PBAC encourage that EC source justification should be provided in submissions, this review found that this is not typically implemented in practice. Guidance is needed to establish best practices as to how EC selection should be documented in HTA submissions.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230140"},"PeriodicalIF":2.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842296/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139087094","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":"Radiofrequency ablation using the ThermoCool SmartTouch Catheter guided by ablation index versus antiarrhythmic drugs in atrial fibrillation treatment in China: a cost-consequence analysis.","authors":"Biqi Zhang, Jie Han, Dongchen Zhou, Jiajia Jiang, Jiawei Zhong, Yunlong Lu, Chenyu Zuo, Xingwei Lu, Jianwei Xuan, Aohan Zu, Yuxian He, Xingang Sun, Lu Chen, Liangrong Zheng","doi":"10.57264/cer-2023-0035","DOIUrl":"10.57264/cer-2023-0035","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the costs and consequences of two front-line atrial fibrillation (AF) treatments from Chinese healthcare system perspective: radiofrequency catheter ablation (RFCA) using ThermoCool SmartTouch Catheter guided by Ablation Index (STAI), in comparison to antiarrhythmic drugs (AADs). <b>Patients & methods:</b> We simulated clinical and economic consequences for AF patients initially receiving STAI or AADs using a short-term decision tree model leading to a 10-year long-term Markov model. The model projected both clinical consequences and costs associated with, among others, AF, heart failure (HF), strokes, and deaths due to AF or AF related complications. Data informing the models included combination of a local real-world study and published clinical studies. <b>Results:</b> STAI was advantageous versus AADs on all 4 main clinical outcomes evaluated; AF: 25.83% lower (12.84% vs 38.67%), HF: 2.22% lower (1.33% vs 3.55%), stroke or post stroke: 1.82% lower (10.00% vs 11.82%) and deaths due to AF or AF related complications: 0.64% lower (4.11% vs 4.75%). The average total cost per patient in STAI group was ¥16,682 lower (¥123,124 vs ¥139,806). The one-way sensitivity analysis indicated that the difference in total cost was most sensitive to annual AF recurrence probability in AADs-treated patients. Probabilistic sensitivity analysis indicated a 98.5% probability that RFCA treatment would result in cost savings by the end of the 10th year. <b>Conclusion:</b> Radiofrequency catheter ablation using SmartTouch catheter guided by Ablation Index was superior to AADs as the first-line AF treatment in Chinese setting with better clinical outcomes and at lower costs over a 10-year time horizon.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230035"},"PeriodicalIF":2.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842291/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139417261","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":"Navigating the unknown: how to best 'reflect' standard of care in indications without a dedicated treatment pathway in health technology assessment submissions.","authors":"Grammati Sarri, Maria Rizzo, Sheela Upadhyaya, Victoria Federico Paly, Luis Hernandez","doi":"10.57264/cer-2023-0145","DOIUrl":"10.57264/cer-2023-0145","url":null,"abstract":"<p><p>There is an urgent need for expedited approval and access for new health technologies targeting rare and very rare diseases, some of which are associated with high unmet treatment needs. Once a new technology achieves regulatory approval, the technology needs to be assessed by health technology assessment (HTA) bodies to inform coverage and reimbursement decisions. This assessment quantitatively examines the clinical effectiveness, safety and/or economic impact of the new technology relative to standard of care (SoC) in a specific market. However, in rare and very rare diseases, the patient populations are small and there is often no established treatment pathway available to define 'SoC'. In these situations, several challenges arise to assess the added benefit of a new technology - both clinically and economically - due to lack of established SoC to guide an appropriate comparator selection. These challenges include: How should 'SoC' be defined and characterized in HTA submissions for new technologies aiming to establish new treatment standards? What is usual care without an established clinical pathway? How should the evidence for the comparator 'SoC' (i.e., usual care) arm be collected in situations with low patient representation and, sometimes, limited disease-specific clinical knowledge in certain geographies? This commentary outlines the evidence generation challenges in designing clinical comparative effectiveness for a new technology when there is a lack of established SoC. The commentary also proposes considerations to facilitate the reliable integration of real-world evidence into HTA and decision-making based on the collective experience of the authors.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230145"},"PeriodicalIF":2.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842305/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139471539","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":"Estimating utility values for non-alcoholic steatohepatitis health states: a discrete choice experiment.","authors":"Daniel Aggio, Katy Gallop, Villum Wittrup-Jensen, Soulmaz Fazeli Farsani, Andrew J Lloyd","doi":"10.57264/cer-2023-0033","DOIUrl":"10.57264/cer-2023-0033","url":null,"abstract":"<p><p><b>Background:</b> This study estimated utility values for non-alcoholic steatohepatitis (NASH). Previous studies have assumed that health-related quality of life does not vary between the early stages of NASH. <b>Materials & Methods:</b> Discrete choice experiment (DCE) surveys estimated the value of avoiding fibrosis progression. Patients also completed the EQ-5D-5L. Marginal rates of substitution estimated utility change associated with fibrosis progression. <b>Results:</b> DCE surveys were completed by the UK general public (n = 520) and patients with NASH (n = 154). The utility decline between fibrosis stages F1 and F4 decompensated was between -0.521 to -0.646 (depending on method). <b>Conclusion:</b> Three methods were used to estimate utilities for NASH, each one showed sensitivity to advancing fibrosis, including in the early stages, which is often considered asymptomatic.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230033"},"PeriodicalIF":2.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842270/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139471463","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":"Real-world evidence on the effectiveness and safety of gliclazide MR 60 mg in Bangladeshi patients with Type II diabetes during fasting: a sub-analysis from the global DIA-RAMADAN study.","authors":"Mohammed Farid Uddin, Murshed Ahamed Khan, Shahjada Selim, Nusrat Sultana, Mohammad Abu Sayem, Mohammed Mahboob Iftekhar, Maruf Bin Habib, Nazma Akter, Shahjamal Khan","doi":"10.57264/cer-2023-0132","DOIUrl":"10.57264/cer-2023-0132","url":null,"abstract":"<p><p><b>Aim:</b> Many Muslims with Type II diabetes (T2DM) fast during Ramadan, which can put them at increased risk of hypoglycemia. This sub-analysis of the global DIA-RAMADAN study assessed the effectiveness and safety of gliclazide modified release (MR) 60 mg in the Bangladeshi cohort. <b>Materials & methods:</b> DIA-RAMADAN was an international, prospective, observational study conducted in adult T2DM patients intending to fast and receiving gliclazide MR 60 mg once daily for ≥90 days before Ramadan. Dosing was switched from morning to evening at the start of Ramadan. The primary outcome was the proportion of patients with ≥1 symptomatic hypoglycemic event. Secondary outcomes included changes between inclusion (V0) and end of study visit (V1) in glycated hemoglobin (HbA1c), body weight and fasting plasma glucose (FPG). <b>Results:</b> Among the 98 Bangladeshi patients, 80 (81.6%) were at moderate/low-risk (category 3) for fasting and 18 (18.4%) were high-risk (category 2), as per International Diabetes Federation and Diabetes and Ramadan International Alliance (IDF-DAR) guidelines. Gliclazide MR was being prescribed as monotherapy to 59 (60.2%) patients and in combination with metformin to 39 (39.8%). There was no incidence of severe hypoglycemic events. Mean (±SD) HbA1c change from V0 was -0.1 ± 0.8% (p = 0.159). Mean (±SD) changes in FPG and body weight were -0.8 ± 39.7 mg/dl (p = 0.876) and -0.0 ± 1.5 kg (p = 0.810), respectively. <b>Conclusion:</b> In a real-world setting, this sub-analysis in Bangladeshi patients shows that patients with T2DM treated with gliclazide MR 60 mg can fast safely during Ramadan with a very low risk of hypoglycemia, while maintaining glycemic control and body weight.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230132"},"PeriodicalIF":2.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842293/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139642271","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":"Corrigendum.","authors":"","doi":"10.57264/cer-2024-0003c1","DOIUrl":"10.57264/cer-2024-0003c1","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240003c1"},"PeriodicalIF":2.1,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10945413/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139471460","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":"Corrigendum.","authors":"","doi":"10.57264/cer-2024-0003","DOIUrl":"https://doi.org/10.57264/cer-2024-0003","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240003c1"},"PeriodicalIF":2.1,"publicationDate":"2024-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139471446","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-effectiveness analysis of Avance^® allograft for the treatment of peripheral nerve injuries in the USA.","authors":"Amir Ansaripour, Ann Thompson, Joseph F Styron, Mehdi Javanbakht","doi":"10.57264/cer-2023-0113","DOIUrl":"10.57264/cer-2023-0113","url":null,"abstract":"<p><p><b>Aim:</b> Peripheral nerve injury (PNI) is a debilitating condition with significant associated morbidity, and which places a substantial socioeconomic burden on healthcare systems worldwide. Recently, allograft has emerged as a viable surgical alternative to autograft for the treatment of PNI. This study evaluated the cost effectiveness of allograft (Avance^® Nerve Graft) compared with autograft for the peripheral nerve repair, from a US payer perspective. <b>Methods:</b> A Markov cohort model was developed to consider the treatment pathways followed by a patient population undergoing a single transected nerve repair with either allograft, or autograft. The marginal difference in meaningful recovery (MR) (effectiveness), and costs, between the two groups were estimated over a lifetime horizon. Deterministic and probabilistic sensitivity analyses (PSA) were performed to consider the uncertainty surrounding the base-case input parameter values and their effect on the overall incremental cost-effectiveness ratio (ICER). <b>Results:</b> The base-case analysis indicates that there is a small difference in the average probability of MR between the two groups (75.15% vs 70.46%; +4.69% with allograft). Allograft also results in cost savings ($12,677 vs $14,023; -$-1346 with allograft) compared with autograft. Deterministic sensitivity analysis shows that the costs of the initial surgical procedures are the main drivers of incremental cost, but that the intervention is likely to be cost saving compared with autograft regardless of the parameter variations made. <b>Conclusion:</b> The use of allograft with the Avance Nerve Graft has the potential to be a cost-effective alternative to autograft for the surgical treatment of PNI in the USA.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230113"},"PeriodicalIF":2.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842286/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138460201","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 manifestations in pediatric and adult patients with Rett syndrome: an analysis of US claims and physician survey data.","authors":"Damian M May, Jeffrey Neul, Jesús Eric Piña-Garza, Kalé Kponee-Shovein, Ambika Satija, Malena Mahendran, Nathaniel Downes, Kristy Sheng, Neema Lema, Andra Boca, Patrick Lefebvre, Victor Abler, James M Youakim, Wendy Y Cheng","doi":"10.57264/cer-2023-0054","DOIUrl":"10.57264/cer-2023-0054","url":null,"abstract":"<p><p><b>Aim:</b> Patients with Rett syndrome (RTT) experience gastrointestinal (GI) manifestations. This study aimed to describe the prevalence of GI manifestations and the associated medical costs in patients with RTT in the USA. <b>Patients & Methods:</b> The study combined an insurance claims database analysis with a survey of 100 physicians experienced in RTT management. <b>Results:</b> GI manifestations affected 43.0% of 5940 patients, with increased prevalence in pediatric patients (45.6%) relative to adult patients (40.2%). Annualized mean medical cost of managing GI manifestations was $4473. Only 5.9-8.2% of neurologists and pediatricians ranked GI symptom management among the five most important treatment goals. <b>Conclusion:</b> Patients with RTT experience a high burden of GI manifestations, which translate to considerable medical costs. Importantly, the prevalence of GI manifestations was likely underestimated in this study, as only those symptoms which resulted in a healthcare encounter were captured.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230054"},"PeriodicalIF":2.1,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10842289/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136397679","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}