Journal of comparative effectiveness research最新文献

{"title":"The impact of a patient advisory board on a clinical comparative effectiveness trial: a comparison of patient and researcher perspectives.","authors":"Laura M Kernan, Monica Baczko Pearl, Adina Harri, Carol A Lambourne, Robert Schlegel, C McCollister Evarts, Mary Beth Crummer, Conrad Persels, Nancy Mullen, Vincent D Pellegrini","doi":"10.57264/cer-2024-0050","DOIUrl":"10.57264/cer-2024-0050","url":null,"abstract":"<p><p><b>Aim:</b> To examine contributions of a patient advisory board (PAB) to the design and conduct of The Pulmonary Embolism Prevention after Hip and Knee Replacement (PEPPER) Trial (NCT02810704) and compare perceptions of PAB members and researchers on the Trial. <b>Materials & methods</b> This evaluation of the PAB was conducted by Clinical Coordinating Center (CCC) members who first discussed PAB contributions, leading to the design of a semi-structured WebEx interview individually querying PAB members on their experience. Two study team members analyzed transcriptions of the interviews for common themes, which were discussed and affirmed at an in-person meeting with PAB members. <b>Results:</b> The contribution most frequently cited as meaningful by PAB members was the creation of a recruitment video. In contrast, the research team considered the most impactful PAB recommendation to be omission of pneumatic compression boots as a study variable. PAB members spoke highly of their involvement in the trial and emphasized shared decision-making in the patient-physician relationship. <b>Conclusion:</b> Researchers and PAB members had different opinions about which PAB contributions were most impactful to the study. This likely derives from differences in perspective; PAB members focused on patient experience and the patient-surgeon relationship while researchers focused primarily on trial outcomes. PAB contributions led to two major protocol changes that had a substantial positive effect on trial design, recruitment and enrollment. This evaluation adds to the engagement literature, which contains little on what patients think of their involvement in the design and conduct of clinical research studies and will aid in encouraging treatment preference discussions between patient and surgeon, thereby supporting the goal of improved patient outcomes.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240050"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864086/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143066003","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":"Clinical characteristics and treatment outcomes in multiple sclerosis patients treated with anti-CD20s who switched to fumarates: a retrospective analysis of a US healthcare claims database.","authors":"Aliza B Ben-Zacharia, Jenny J Feng, Brandon P Moss, Nicholas Belviso, Yu Zhang, Filipe Branco, Jason P Mendoza, James B Lewin, Sarah M England","doi":"10.57264/cer-2024-0071","DOIUrl":"10.57264/cer-2024-0071","url":null,"abstract":"<p><p><b>Aim:</b> Anti-CD20 monoclonal antibodies and fumarates are common multiple sclerosis (MS) disease-modifying therapies (DMTs). Data on switching from anti-CD20s to other DMTs are limited. This retrospective, observational study of the US Komodo Health Sentinel claims database aimed to evaluate a de-escalation strategy in a real-world cohort, comparing clinical characteristics, relapses, healthcare encounters (HCEs) and healthcare costs (HCCs) between patients aged ≥18 years with stable MS who switched from anti-CD20s to fumarates ('Switchers') versus patients who stayed on anti-CD20s ('Stayers'). <b>Materials & methods:</b> Patients with MS (diagnosed 1 January 2015-31 August 2022) were propensity score matched 5:1 (Stayers:Switchers) and followed from index to end of study; end of insurance eligibility; >45-day gap in index DMT; or DMT switch. Primary outcomes were clinical characteristics and claims-based annualized relapse rate (ARR). Rates of HCEs and HCCs were estimated. <b>Results:</b> Baseline characteristics were well balanced between cohorts (Stayers, n = 540; Switchers, n = 108). Mean (SD) duration of post-index follow-up was 341.4 (250.0) days for both cohorts. Mean (SD) ARR was 0.08 (0.41; Stayers) versus 0.14 (0.5; Switchers; p = 0.3). Twenty-one Stayers (3.9%) and 1 Switcher (0.9%) were hospitalized for infections, with mean stays of 9.9 and 1 day, respectively. Mean annualized all-cause HCEs were similar between cohorts; annualized inpatient infection-related HCEs were higher for Stayers versus Switchers (mean difference: -0.05; p = 0.005). Annualized all-cause HCCs were similar between cohorts; Switchers had lower annualized infection-related HCCs overall (mean difference: -$2412; p = 0.002) and in the inpatient setting (mean difference: -$2325; p = 0.002). <b>Conclusion:</b> After 1 year, no significant differences in ARR emerged between cohorts. Switchers experienced lower inpatient infection-related HCEs, shorter inpatient infection-related hospital stays and lower overall infection-related HCCs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240071"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864085/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399374","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":"Efficacy of treatment options for complicated urinary tract infections including acute pyelonephritis: a systematic literature review and network meta-analysis.","authors":"Florian Wagenlehner, Verónica Rico Caballero, Vikalp Maheshwari, Ayantika Biswas, Priyanka Saini, Juan Quevedo, Juergen Polifka, Leonardo Ruiz, Sandrine Cure","doi":"10.57264/cer-2024-0214","DOIUrl":"10.57264/cer-2024-0214","url":null,"abstract":"<p><p><b>Aim:</b> Compared with uncomplicated urinary tract infections (UTIs), complicated UTIs (cUTIs) including acute pyelonephritis (AP) present with significant morbidity, a higher risk of treatment failure and typically require longer courses of treatment, or alternative antibiotics. The emergence of drug-resistant organisms represents a considerable challenge in the treatment of patients with cUTIs/AP and has limited antibiotic options. Carbapenems are considered the current last line of therapy, however, carbapenem resistance represents a growing problem. Although several established and novel treatment options are available, direct comparative evidence is lacking. <b>Methods:</b> Randomized controlled trials (RCTs) were identified by systematic literature review of Embase^®, MEDLINE^® and Cochrane databases (database inception to 15th June 2022). Relevant conference proceedings (2020-2022) were also reviewed. Following feasibility assessment to verify network connectivity at an overall level, outcome specific networks were prepared. Bayesian network meta-analysis (NMA) was performed (using R version 4.2.1) to determine the relative efficacy of various treatments for cUTI/AP, including cefepime + enmetazobactam. Convergence was assessed by visual inspection of trace plots. The accuracy of the posterior estimates was assessed using the Monte Carlo error for each parameter. Published study results were included in the synthesis of the relative risk (RR) of efficacy end points, using a logit link with binomial likelihood distribution. <b>Results:</b> Feasibility assessment was conducted for 40 RCTs identified, to assess the viability of constructing a network of interlinked RCTs. Of those, 28 studies were included in the master NMA network. A fixed effects model (FEM) was selected due to low statistical heterogeneity, according to I² values. For composite outcome at test of cure (TOC), ceftolozane + tazobactam, cefepime + enmetazobactam, cefiderocol, levofloxacin and plazomicin demonstrated significantly higher RRs versus carbapenems. For microbiological eradication at TOC, cefepime + enmetazobactam, plazomicin, cefiderocol, fosfomycin, meropenem + vaborbactam and ceftazidime + avibactam demonstrated significantly higher RRs versus carbapenems. RRs for cefepime + enmetazobactam were also significantly higher versus several established and novel treatment options for composite outcome, microbiological eradication and clinical cure. <b>Conclusion:</b> Against the backdrop of increasing bacterial resistance, these findings suggest that cefepime + enmetazobactam may represent an effective carbapenem-sparing treatment option in patients with cUTI including AP.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240214"},"PeriodicalIF":1.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864080/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143006384","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":"Mapping methods gaps between EU joint clinical assessments and local health technology assessment decision-making: an environmental scan of guidance in select EU markets and harmonization challenges.","authors":"Grammati Sarri, Lydia Vinals, Lilia Leisle, Ingrid Claverie Chau, David Smalbrugge, Kai Lucassen, Yannis Jemiai","doi":"10.57264/cer-2024-0240","DOIUrl":"https://doi.org/10.57264/cer-2024-0240","url":null,"abstract":"<p><p><b>Aim:</b> Under the newly instituted health technology assessment (HTA) regulation (HTAR), health technology developers must build evidence packages that meet the needs for both the upcoming EU joint clinical assessment (JCA) and national decision-making. In-depth knowledge of local methodological requirements as well as preparedness for effective strategic development is crucial. This study aimed to review methodological guidance documents to map similarities/misalignments between the EU HTAR and select HTA agencies. <b>Materials & methods:</b> An environmental scan was performed in March 2024 and updated in December 2024 of the websites for European Network for HTA, the European Commission and HTA agencies in France, Germany, The Netherlands and Spain. The search aimed to systematically identify and summarize methodological guidance documents from the respective organizations on scoping considerations, evidence identification and synthesis. <b>Results:</b> Overall, published EU HTAR methods guidelines are detailed, prescriptive and make reference to a preference (or lack thereof) for specific analytical methods. There was consensus among EU JCA and local HTA guidelines that clinical comparative assessments should be based on a systematically identified, unbiased selected evidence base derived from various sources. However, agencies differed on guidance related to evidence derived from indirect treatment comparisons. <b>Conclusion:</b> An environmental scan of methods documents revealed that it will likely be challenging for health technology developers to build strong evidence packages that can support both EU JCA and local reimbursement decision-making. A greater understanding of the similarities and differences between EU and local HTA requirements will be needed, including a greater capacity to demonstrate value through advanced analytics.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240240"},"PeriodicalIF":1.9,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143501861","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":"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":"https://doi.org/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":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Matching-adjusted indirect comparisons of efficacy outcomes between etrasimod and ozanimod for moderately to severely active ulcerative colitis.","authors":"Vipul Jairath, Tim Raine, Thomas P Leahy, Ravi Potluri, Karolina Wosik, David Gruben, Joseph C Cappelleri, Peter Hur, Lauren Bartolome","doi":"10.57264/cer-2024-0193","DOIUrl":"https://doi.org/10.57264/cer-2024-0193","url":null,"abstract":"<p><p><b>Aim:</b> Etrasimod and ozanimod are selective sphingosine 1-phosphate receptor modulators targeting the S1P_1,4,5, and S1P_1,5 receptors, respectively, for the treatment of patients with moderately to severely active ulcerative colitis (UC). No head-to-head trial data exist between the two treatments. We compared these treatments indirectly using key efficacy outcomes from pivotal trials with induction and maintenance phase data adjusting for differences in clinical trial design and populations. <b>Materials & methods:</b> Individual patient data for etrasimod were matched to published aggregate data of ozanimod by key baseline characteristics. An anchored matching-adjusted indirect comparison (MAIC) was conducted for the induction period. An unanchored MAIC was utilized during the maintenance period due to differences in placebo arms between trials as a result of differing trial designs. Matching characteristics measured at baseline were age, sex, corticosteroid use, duration of UC, biologic exposure, modified Mayo score, and presence of left-sided colitis. Outcomes were clinical response and clinical remission for the induction period, and clinical response and clinical remission among induction phase responders for the maintenance period. Two sensitivity analyses were conducted. The first matched on prior TNFi exposure rather than biologic exposure, the second sensitivity analysis included an induction only etrasimod trial (ELEVATE UC 12). <b>Results:</b> There were no significant differences between etrasimod and ozanimod at the end of the induction period for clinical response and clinical remission, respectively (relative risk [RR] 0.98 [95% confidence interval (CI): 0.76-1.33], RR: 1.25 [95% CI: 0.71-2.92]). At the end of maintenance, etrasimod demonstrated improved outcomes compared with ozanimod for both clinical response (RR: 1.18 [95% CI: 1.05-1.30]) and clinical remission among induction phase responders (RR: 1.33 [95% CI: 1.12-1.55]). In the sensitivity analysis that matched on prior TNFi exposure rather than biologic exposure, there were no notable differences compared with the primary analyses. In the sensitivity analysis pooling ELEVATE UC 12 and ELEVATE UC 52 data, results were similar for clinical response (RR: 0.90 [95% CI: 0.75-1.10]) but etrasimod showed reduced efficacy for clinical remission (RR: 0.72 [95% CI: 0.50-1.12]) compared with the primary analysis, though overall remained not significantly different from ozanimod. <b>Conclusion:</b> MAIC results suggest that patients receiving etrasimod have similar induction results but are more likely to have clinical response and clinical remission at the end of the maintenance phase compared with patients receiving ozanimod. Despite the approach to ensure similarity between the trials by weighting, residual imbalance is possible, and results should be interpreted in the context of the assumptions.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240193"},"PeriodicalIF":1.9,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143483330","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":"https://doi.org/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":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The role of health economic evidence in clinical practice guidelines for colorectal cancer: a comparative analysis across countries.","authors":"Xiaoyu Yan, Yue Wang, Aixia Ma, Hongchao Li","doi":"10.57264/cer-2024-0226","DOIUrl":"https://doi.org/10.57264/cer-2024-0226","url":null,"abstract":"<p><p><b>Aim:</b> Colorectal cancer (CRC) is among the most prevalent malignancies globally and causes massive resource consumption and economic burden. Health economic evidence (HEE) has been used in clinical practice guidelines (CPGs) for cancer to facilitate the rational allocation of health resources. However, in certain guideline development organizations, HEE is not yet utilized as a formal decision-making criterion. This study aimed to compare the discrepancies in the utilization of health economics as evidence in CRC CPGs across different countries and review specific features of economic evidence concerning the guidelines' applicability. <b>Materials & methods:</b> A systematic review was conducted using databases including Medline, Embase, CNKI, WanFang, and other guidelines databases to identify CPGs for CRC published in English or Chinese from January 2017 to September 2023. Data on the incorporation and application of HEE were extracted, and the method and quality of cost-effectiveness analysis (CEA) studies were evaluated. Descriptive analyses were used to summarize the results. <b>Results:</b> Out of 53 CPGs from 14 countries, most originated from the USA (n = 17 of 53 [32%]) and Canada (n = 9 of 53 [17%]). Sixty-eight percent (36/53) considered cost justification, and 57% (30/53) incorporated health economics studies as evidence. The included HEE cited in CPGs ranged from 1990 to 2021 and were not aligned with the countries in which the guidelines were issued. Among these CEA studies, 52% (26/50) were related to screening strategies, and 32% (16/50) pertained to treatment measures. The Markov model was the most frequently used (n = 27 of 50 [54%]). Based on the CHEQUE tool, the methodological quality of these CEA studies was inadequate in areas such as multiple data sources, approaches to select data sources, assessing the quality of data, and relevant equity or distribution. <b>Conclusion:</b> In summary, 57% of guidelines incorporated health economics studies as evidence, with a variation between different countries. The included HEE still had deficiencies in methodology and reporting quality. In the future, it is suggested that health economics research should use a standardized methodology and reporting approach to assist in clinical decision making.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240226"},"PeriodicalIF":1.9,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143449213","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":"R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 18.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2025-0014","DOIUrl":"https://doi.org/10.57264/cer-2025-0014","url":null,"abstract":"<p><p>In this update, we discuss recent publications examining the use of real-world data as a measure of treatment effectiveness in submissions to the National Institute of Health and Care Excellence, the results of a pilot study from the Coalition to Advance Real-World Evidence initiative and a validation study of synthetic data.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250014"},"PeriodicalIF":1.9,"publicationDate":"2025-02-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441107","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":"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":"https://doi.org/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":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}