Journal of comparative effectiveness research最新文献

{"title":"Elranatamab versus physician's choice of treatment in patients with triple-class exposed/refractory multiple myeloma: an updated matching-adjusted indirect comparison.","authors":"Isha Mol, Yannan Hu, Thomas W LeBlanc, Joseph C Cappelleri, Haitao Chu, Guido Nador, Didem Aydin, Isabel Perez Cruz, Patrick Hlavacek","doi":"10.57264/cer-2024-0236","DOIUrl":"https://doi.org/10.57264/cer-2024-0236","url":null,"abstract":"<p><p><b>Aim:</b> Despite the availability of novel treatment options for patients with triple-class exposed/refractory multiple myeloma , there is a lack of consensus on the optimal regimen. A previous unanchored matching-adjusted indirect comparison (MAIC) of the MagnetisMM-3 (NCT04649359) and LocoMMotion (NCT04035226) study reported significant improvements in progression-free survival (PFS) and overall survival (OS) with elranatamab versus physician's choice of treatment. <b>Materials & methods:</b> We conducted an updated MAIC based on more recent data (28.4 months for MagnetisMM-3). Following reweighting of MagnetisMM-3 individual patient data to match the LocoMMotion population, the effective sample size was 64 for PFS and 63 for OS in the base-case analysis. <b>Results:</b> Consistent with the original MAIC, significantly improved PFS (hazard ratio [HR] [95% confidence interval (CI)]: 0.32 [0.21, 0.50], p < 0.01) and OS (HR [95% CI]: 0.50 [0.33, 0.78], p < 0.01) were observed with elranatamab versus physician's choice of treatment. The robustness of the results was demonstrated in sensitivity analyses in which missing baseline characteristics data for elranatamab were imputed. <b>Conclusion:</b> Overall, the findings of this study align with and add confidence to the conclusions of the previous MAIC that elranatamab is associated with significantly improved outcomes versus standard treatment, supporting the value to elranatamab for the treatment of patients with triple-class exposed/refractory multiple myeloma.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240236"},"PeriodicalIF":1.9,"publicationDate":"2025-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143795541","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":"A process to validate prognostic factors for unanchored matching-adjusted indirect comparison of single-arm trials in oncology: a proof-of-concept study.","authors":"Yao Yi, Yawen Jiang","doi":"10.57264/cer-2024-0235","DOIUrl":"https://doi.org/10.57264/cer-2024-0235","url":null,"abstract":"<p><p><b>Aim:</b> The choice of covariates in unanchored matching-adjusted indirect comparisons (MAICs) of single-arm cancer trials with time-to-event outcomes remains a challenge. Currently, there is a lack of a systematic approach for validating the selection of covariates for bias reduction in unanchored MAIC. <b>Materials & methods:</b> This study proposes a validation framework to evaluate the appropriateness of selected prognostic factors before their use in unanchored MAIC. The process involves identifying potential prognostic factors from individual patient data and calculating risk scores using the prognostic factors with regression; artificially creating two groups that are unbalanced in risk such that a predetermined hazard ratio (HR) between the two groups is achieved; creating weights based on the prognostic factors; running a re-weighted Cox regression to assess the HR, the value of which should suggest balanced risks across groups to indicate the sufficiency of prognostic factors being included. We also conducted a proof-of-concept analysis using a simulated dataset to showcase this process. <b>Results:</b> The process successfully stratified the sample into two risk groups with a pre-determined HR of 1.8. When all covariates were included in the weighting, the HR was 0.9157 (95% CI: 0.5629-2.493), which was close to one. When one of the critical prognostic factors was omitted from the covariates, the HR became 1.671 (95% CI: 1.194-2.340), which was significantly different from one. <b>Conclusion:</b> Filling a gap in the existing evidence synthesis literature, the study introduces a structured data-driven approach for covariate prioritization in unanchored MAIC. The process may be a useful tool for quantitative covariate selection.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240235"},"PeriodicalIF":1.9,"publicationDate":"2025-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143795538","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":"The need to consider market access for pharmaceutical investment decisions: a primer.","authors":"Sreeram V Ramagopalan, Catherine Bacon, Mel Walker, Michael L Ryan","doi":"10.57264/cer-2025-0036","DOIUrl":"https://doi.org/10.57264/cer-2025-0036","url":null,"abstract":"<p><p>Biotech/Pharma investors employ valuation methods to support capital deployment that consider the costs of drug development, projected sales and risks of failure. Often, the major focus for valuation is placed on the likely success rates of taking a compound from phase I to regulatory approval, with the notion being that just by obtaining regulatory approval sales will follow. However, as exemplified by recent cases with hemophilia gene therapies, achieving forecasted sales depends not only on regulatory success but crucially on market access outcomes. This primer examines how pharmaceutical investment decisions must consider market access factors, particularly in light of recent regulatory changes such as the US Inflation Reduction Act and European Union Joint Clinical Assessment. Effective market access strategies can enhance commercial success through better pricing, broader reimbursement, and/or faster uptake, and having a clear market access plan should encourage investment by providing a clearer path to commercial success. As health technology assessment processes become more sophisticated globally, treating market access as an essential strategic capability rather than a tactical exercise will be important for attracting investment and ultimately, successful drug development and commercialization.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250036"},"PeriodicalIF":1.9,"publicationDate":"2025-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143780153","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":"Economic burden of sickle cell disease in the United States: a retrospective analysis of a commercial insurance database.","authors":"Giovanna Tedesco Barcelos, Telma Peixoto, Jose Alvir, Jay Lin, Christine L Baker","doi":"10.57264/cer-2025-0006","DOIUrl":"https://doi.org/10.57264/cer-2025-0006","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate healthcare resource utilization (HCRU) and costs for US commercially insured adult and pediatric patients with sickle cell disease (SCD) and matched non-SCD cohorts. <b>Materials & methods:</b> Patients with ≥3 SCD diagnosis codes (D57.0-D57.219; D57.4-D57.819) from July 2016 to December 2020 were identified from the IBM^® MarketScan^® Commercial database. The earliest SCD diagnosis was defined as the index date. Non-SCD control patients were matched 1:1 on age, gender and region. Continuous 6-month baseline and ≥12-month follow-up coverage was required. Follow-up HCRU and costs (2020 USD) were calculated per patient per year. Pediatric (<18 years) and adult (≥18 years) patients were analyzed separately. <b>Results:</b> For 1299 pediatric patients with SCD and matched controls, mean (SD) age was 10.0 (4.8) years and 51% were female; mean (SD) follow-up was 34.3 (14.4) months. In the first 12 months, pediatric patients with SCD had higher HCRU (hospitalizations: 0.6 vs 0.01; hospital length of stay: 2.4 vs 0.05 days; outpatient visits: 13.4 vs 6.0; office visits: 6.9 vs 4.7; prescriptions: 12.8 vs 3.8) and mean total costs ($31,445 vs $2844), mainly due to hospitalizations ($15,195 vs $477) and outpatient visits ($12,746 vs $1758), versus controls (all p < 0.0001). For 2792 adults with SCD and matched controls, mean (SD) age was 38.0 (13.2) years and 62% were female; mean (SD) follow-up was 31.8 (13.7) months. Adults with SCD had higher per-patient per-year HCRU (hospitalizations: 0.8 vs 0.06; hospital length of stay: 4.3 vs 0.2 days; outpatient visits: 20.9 vs 9.3; office visits: 10.4 vs 6.9; prescriptions: 20.5 vs 11.7) and mean total costs ($42,550 vs $7522), also due to hospitalizations ($20,056 vs $1326) and outpatient visits ($17,508 vs $4301), versus controls (all p < 0.0001). <b>Conclusion:</b> The economic burden of SCD among pediatric and adult patients is substantial with increased HCRU and costs compared with matched controls. Better treatments for SCD could reduce the economic burden for patients, as well as payers.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250006"},"PeriodicalIF":1.9,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143764157","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":"Comparison of real-world healthcare resource utilization and costs among patients with hereditary angioedema on lanadelumab or berotralstat long-term prophylaxis.","authors":"Nicole Princic, Kristin A Evans, Chintal H Shah, Krystal Sing, Salomé Juethner, Bob G Schultz","doi":"10.57264/cer-2024-0205","DOIUrl":"10.57264/cer-2024-0205","url":null,"abstract":"<p><p><b>Aim:</b> Hereditary angioedema (HAE) is a rare and chronic genetic condition. Lanadelumab and berotralstat, two plasma kallikrein inhibitors, have both been approved for long-term prophylaxis in patients with HAE; however, real-world data comparing costs and healthcare resource utilization (HCRU) are lacking. <b>Materials & methods:</b> This retrospective study used administrative healthcare insurance claims data (Merative™ MarketScan^® Commercial, Medicare and Early View Research Databases; 1 July 2017-31 July 2023) to identify patients with HAE who initiated lanadelumab or berotralstat and were persistent for ≥18 months or 6 months, respectively. Sex, baseline healthcare costs and baseline number of on-demand treatment/short-term prophylaxis medication claims were used to calculate covariate balancing propensity scores for inverse probability of treatment weighting. Following weighting, outcomes during the 6-month follow-up period in patients receiving berotralstat were compared with those during months 0-6, 7-12 and 13-18 in lanadelumab-treated patients. <b>Results:</b> Fifty-seven lanadelumab- and 32 berotralstat-treated patients were included. After weighting, more berotralstat-treated patients had an all-cause inpatient admission (berotralstat, 9.4%; lanadelumab, months 0-6, 4.0%, 7-12, 1.8%, months 13-18, 2.0%) and emergency room visit (berotralstat, 21.9%; lanadelumab, months 0-6, 14.0%, 7-12, 8.0%, months 13-18, 17.9%). Total HAE treatment costs were similar during months 0-6 (lanadelumab, $377,326 vs berotralstat, $373,010), but decreased in months 7-12 ($319,967) and 13-18 ($283,241) of lanadelumab. On-demand treatment/short-term prophylaxis costs were lower for lanadelumab across the three follow-up periods than for berotralstat during months 0-6 (berotralstat, $60,451; lanadelumab, months 0-6, $46,336, months 7-12, $37,578, months 13-18, $23,968). The proportion of lanadelumab-treated patients who reduced dosing frequency was 24.8% during months 7-12 and 21.6% during months 13-18. <b>Conclusion:</b> Patients with HAE initiating lanadelumab versus berotralstat may require less on-demand and supportive HAE treatments and incur lower treatment-related and total healthcare costs. The ability to reduce lanadelumab dosing frequency after an attack-free period may be key in treatment selection, given the combination of cost savings and lower healthcare resource utilization.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240205"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963383/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143458260","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":"Eculizumab or ravulizumab treatment effect in people with neuromyelitis optica spectrum disorder: a plain language summary of three studies.","authors":"Alfredo Damasceno, Mariano Marrodan","doi":"10.57264/cer-2024-0177","DOIUrl":"10.57264/cer-2024-0177","url":null,"abstract":"<p><strong>What is this summary about?: </strong>Neuromyelitis optica spectrum disorder (NMOSD for short) is a rare autoimmune health condition, meaning that the body's natural defense system (the immune system) attacks the body's own tissues. This summary describes NMOSD and the results of three studies of the effects of treatment with two medicines called eculizumab and ravulizumab. Eculizumab and ravulizumab are approved to treat people with a type of NMOSD called AQP4-Ab+ NMOSD. The three studies included in this summary are the PREVENT and CHAMPION-NMOSD clinical studies and a study done in everyday clinical practice in Japan.</p><p><strong>What are the key conclusions from these studies?: </strong>The PREVENT study was a phase 3 study (a large study testing safety and effectiveness of a treatment before it is approved) with three parts. The PREVENT main study compared eculizumab to a placebo (a treatment with no active ingredients, used to test how well a new treatment works). A long-term follow-up study assessed the safety and effectiveness of eculizumab over time, without a placebo group. Another long-term follow-up focused on people taking eculizumab alone, without other immunosuppressive treatments. The CHAMPION-NMOSD study was another phase 3 study testing ravulizumab, a treatment based on eculizumab but given less often, using the placebo group from the PREVENT study for comparison. A daily clinical practice study with eculizumab in Japan looked at how eculizumab works in everyday medical practice (outside of controlled clinical trials) after the medication was already approved. The studies found eculizumab and ravulizumab to be safe and effective for preventing relapses in AQP4-Ab+ NMOSD. More than 95% of people treated with these medicines stayed relapse-free during the months or years of the studies follow-up periods. Most people reported side effects that were mild or moderate. The most common side effects were headache, runny nose or sore throat (nasopharyngitis), and infections in the upper respiratory system. The daily practice study confirmed that eculizumab works well in everyday medical practice.</p><p><strong>What do the findings of the study mean?: </strong>These studies suggest that eculizumab and ravulizumab are safe and effective treatments for people with AQP4-Ab+ NMOSD. Eculizumab may help reduce or stop the need for other treatments that weaken the immune system (immunosuppressive therapies). These treatments help people maintain their ability to carry out daily activities and their quality of life.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240177"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963372/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399377","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":"Evaluation of reporting in time-driven activity-based costing studies on cardiovascular diseases: a scoping review.","authors":"Nayê Balzan Schneider, Erica Caetano Roos, Miriam Allein Zago Marcolino, Fabio Caldana, Filipe Rodrigues Vargas do Nascimento, Sérgio Renato da Rosa Decker, Ana Paula Beck da Silva Etges, Carisi Anne Polanczyk","doi":"10.57264/cer-2024-0013","DOIUrl":"10.57264/cer-2024-0013","url":null,"abstract":"<p><p><b>Aim:</b> This scoping review evaluates the application of the time-driven activity-based costing (TDABC) methodology in cardiovascular disease (CVD) studies. <b>Materials & methods:</b> The evaluation was conducted using the 32-item TDABC Healthcare Consortium Consensus Statement Checklist. A systematic search was performed in Medline, Embase and Scopus in September 2023, including only full-text, peer-reviewed studies reporting the application of TDABC in CVD research. <b>Results:</b> Twenty studies were included in the review. The positive response rate for individual studies ranged from 31 to 81%. The most frequently addressed checklist item was the clear definition of study objectives, while presenting costs per patient included in the analysis was the least reported item. Although 70% of the studies achieved a positive response rate above 50%, adherence to the TDABC checklist remains inconsistent. <b>Conclusion:</b> There is significant room for improvement in the reporting of TDABC methodology in CVD studies. Providing a more comprehensive and standardized description of the methodology would enhance the utility, reproducibility and accuracy of the information generated, supporting the development of evidence-based health policies and improving accountability in healthcare cost assessments.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240013"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963363/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143501842","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":"Burden of illness for patients with primary biliary cholangitis: an observational study of clinical characteristics and healthcare resource utilization.","authors":"Robert G Gish, Joanna P MacEwan, Alina Levine, Dannielle Lebovitch, Leona Bessonova, Darren Wheeler, Radhika Nair, Alan Bonder","doi":"10.57264/cer-2024-0174","DOIUrl":"10.57264/cer-2024-0174","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the clinical characteristics and healthcare resource utilization for acute care and its costs for patients with primary biliary cholangitis (PBC) with or without cirrhosis. <b>Materials & methods:</b> This retrospective observational cohort study was conducted using two datasets (Komodo's Healthcare Map™ [Komodo Health] and Optum Clinformatics^® Data Mart [CDM] database) between 2015 and 2023. Patients (≥18 years) with PBC were identified based on ≥1 inpatient or ≥2 outpatient claims. Healthcare resource utilization for acute care (hospitalizations and emergency department [ED] visits [not leading to hospitalization]) were assessed in both datasets, and associated medical costs were evaluated in Optum CDM. <b>Results:</b> In Komodo Health, of the 29,758 patients with PBC (mean age: 59.2 years), 21.6% had cirrhosis and 50.4% of patients with cirrhosis had Medicaid or Medicare coverage. Of the total 8143 patients in Optum CDM (mean age: 67.0 years), 20.7% had cirrhosis, and most were enrolled in Medicare (69.7%). There was a larger proportion of men in the cirrhosis group compared with the no-cirrhosis group in Komodo Health (31.7 vs 16.3%) and Optum CDM (29.7 vs 16.5%). Annually, among patients with cirrhosis who had a hospitalization, 69.3% had additional hospitalizations, and among patients who had an ED visit, 52.9% had additional ED visits in Komodo Health; similar results were observed in Optum CDM. Among patients with at least one acute-care event, the mean annual acute-care costs with and without cirrhosis were $113,568 and $47,436, respectively. <b>Conclusion:</b> Data from two large healthcare claims databases showed that the majority of patients who had at least one acute-care event experienced additional acute-care events, particularly among those with cirrhosis. Timely treatment to avoid hospitalization and disease progression may help mitigate the clinical and economic burden for patients with PBC.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240174"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963345/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143567244","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":"Trends in adoption of knotless tissue control devices in robotic surgery.","authors":"Barbara H Johnson, Sinchana T, Stephen S Johnston, Najmuddin Gunja","doi":"10.57264/cer-2024-0229","DOIUrl":"10.57264/cer-2024-0229","url":null,"abstract":"<p><p><b>Aim:</b> Trends in the use and comparative outcomes for barbed and conventional sutures have not been well-reported for robotic surgery. <b>Materials & methods:</b> This retrospective study used hospital discharge data in the US to assess suture use during robotic colorectal surgery (CR), total hysterectomy (TH) and ventral hernia repair (VHR) performed between 1 October 2015 and 30 June 2022. We first examined quarterly trends in use of barbed sutures, (specifically STRATAFIX™ knotless tissue control devices [KTCD]) and then compared clinical and economic outcomes between KTCD and conventional sutures. Outcomes included wound-related complications (infection/wound dehiscence) through 90 days post discharge, operating room (OR) time, hospital costs and 30/60/90 days readmissions. Propensity score weighting was used to address potential confounding in the comparisons of outcomes, performed separately for each procedure. Generalized linear models, accounting for hospital-level clustering, with link functions and error distributions tailored to the empirical distribution of outcomes were used to test for statistically significant differences in outcomes between the KTCD and conventional suture cohorts. <b>Results:</b> We identified 15,875 patients with CR (668 KTCD), 175,963 patients with TH (15,075 KTCD) and 32,469 patients with VHR (6776 KTCD). Over the study period, the proportion of robotic surgeries using KTCD tripled for CR (2.0-6.4%) and TH (2.9-10.4%) and more than doubled for VHR (12.2-25.3%). Compared with conventional sutures, KTCD was associated with significantly shorter OR time for TH (-19.1 min, 95% CI: [-30.2, -8.0]) and VHR (-17.3 min, 95% CI: [-31.4, -3.2]), and was numerically shorter, but did not reach statistical significance for CR (-23.2 min 95% CI: [-48.1 to 1.7]). All other outcomes were similar between the two suture cohorts for all procedures, apart from CR 90-day readmissions, which were lower for the KTCD cohort (-2.8%, 95% CI: [-5.2 to -0.4%]). <b>Conclusion:</b> Adoption of KTCD has grown substantially over the past 6 years. While most clinical and economic outcomes were similar between the two groups, KTCD was associated with lower OR time versus conventional sutures for TH and VHR and lower 90-day readmissions for CR.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240229"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963344/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143647928","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: state-of-the-art and future perspectives.","authors":"Heather Fitzke, Tamanah Fayzan, Jonathan Watkins, Evgeny Galimov, Benjamin F Pierce","doi":"10.57264/cer-2024-0130","DOIUrl":"10.57264/cer-2024-0130","url":null,"abstract":"<p><p>Recent developments in digital infrastructure, advanced analytical approaches, and regulatory settings have facilitated the broadened use of real-world evidence (RWE) in population health management and evaluation of novel health technologies. RWE has uniquely contributed to improving human health by addressing unmet clinical needs, from assessing the external validity of clinical trial data to discovery of new disease phenotypes. In this perspective, we present exemplars across various health areas that have been impacted by real-world data and RWE, and we provide insights into further opportunities afforded by RWE. By deploying robust methodologies and transparently reporting caveats and limitations, real-world data accessed via secure data environments can support proactive healthcare management and accelerate access to novel interventions in England.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240130"},"PeriodicalIF":1.9,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11963347/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573175","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}