Journal of comparative effectiveness research最新文献

{"title":"First in class, best in class or a wild card: who will dominate the anti-obesity medication market?","authors":"Emma Collins, Alice Beattie, Sreeram V Ramagopalan, Jonathan Pearson-Stuttard","doi":"10.57264/cer-2024-0044","DOIUrl":"10.57264/cer-2024-0044","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240044"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225158/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141161300","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":"Evaluating the robustness of an AI pathfinder application on eligibility criteria in multiple myeloma trials using real-world data and historical trials.","authors":"Rana Jreich, Hao Zhang, Zhaoling Meng, Fei Wang","doi":"10.57264/cer-2023-0164","DOIUrl":"10.57264/cer-2023-0164","url":null,"abstract":"<p><p><b>Background:</b> Eligibility criteria are pivotal in achieving clinical trial success, enabling targeted patient enrollment while ensuring the trial safety. However, overly restrictive criteria hinder enrollment and study result generalizability. Broadening eligibility criteria enhances the trial inclusivity, diversity and enrollment pace. Liu <i>et al.</i> proposed an AI pathfinder method leveraging real-world data to broaden criteria without compromising efficacy and safety outcomes, demonstrating promise in non-small cell lung cancer trials. <b>Aim:</b> To assess the robustness of the methodology, considering diverse qualities of real-world data and to promote its application. <b>Materials/Methods:</b> We revised the AI pathfinder method, applied it to relapsed and refractory multiple myeloma trials and compared it using two real-world data sources. We modified the assessment and considered a bootstrap confidence interval of the AI pathfinder to enhance the decision robustness. <b>Results & conclusion:</b> Our findings confirmed the AI pathfinder's potential in identifying certain eligibility criteria, in other words, prior complications and laboratory tests for relaxation or removal. However, a robust quantitative assessment, accounting for trial variability and real-world data quality, is crucial for confident decision-making and prioritizing safety alongside efficacy.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230164"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225521/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141310768","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":"Progress of patients hospitalized with acute heart failure treated with empagliflozin.","authors":"Manuel Raya-Cruz, Javier Gascón Jurado, Gonzalo Olalla de la Torre Peregrín, Nicolás Montúfar, Agustín Rodríguez Sánchez, Francisco Gómez Delgado","doi":"10.57264/cer-2024-0027","DOIUrl":"10.57264/cer-2024-0027","url":null,"abstract":"<p><p><b>Aim:</b> To describe the epidemiological, clinical and laboratory characteristics and clinical progress of patients hospitalized with heart failure (HF) who started treatment with empagliflozin before discharge. <b>Methods:</b> We performed a retrospective observational study of patients aged ≥18 years admitted to the Internal Medicine Department of University Hospital Jaen, Jaen, Spain with acute HF between 1 May 2022 and 31 May 2023. Patients had to have a life expectancy of ≥1 year and have started treatment with empagliflozin during admission. <b>Results:</b> We included 112 patients (mean age, 85.2 ± 6.5 years; 67.9% women; 35.7 and 31.3% in NYHA functional classes III and IV; 73.2% with HF and preserved ejection fraction). Before admission, 80.4% were taking loop diuretics, 70.6% renin-angiotensin-aldosterone system inhibitors, 49.1% betablockers and 25% mineralocorticoid receptor antagonists. At admission, 94.6% were taking furosemide (15.2% at high doses, 36.6% at intermediate doses). The dose of furosemide was reduced at initiation of empagliflozin. At the end of follow-up, 13.4% of patients had died, 93.8% of the survivors continued treatment with empagliflozin and 26.8% had attended the emergency department with signs and symptoms of HF. <b>Conclusion:</b> Introduction of empagliflozin before discharge from hospital in patients admitted with HF made it possible to reduce the dose of diuretics during admission. The frequency of complications was as expected, and treatment was largely maintained.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240027"},"PeriodicalIF":1.9,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145528/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141087690","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":"Healthcare resource utilization and costs among patients with alpha-1 antitrypsin deficiency with liver and/or lung disease: a longitudinal retrospective study in the USA.","authors":"May Hagiwara, Victoria Divino, Swapna Munnangi, Mark Delegge, Suna Park, Ed G Marins, Kaili Ren, Charlie Strange","doi":"10.57264/cer-2023-0186","DOIUrl":"10.57264/cer-2023-0186","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate all-cause and liver-associated healthcare resource utilization (HCRU) and costs among patients with alpha-1 antitrypsin deficiency (AATD) with liver disease (LD) and/or lung disease (LgD). <b>Materials & methods:</b> This was a retrospective analysis of linked administrative claims data from the IQVIA PharMetrics^® Plus and the IQVIA Ambulatory Electronic Medical Records (AEMR) databases from 1 July 2021 to 31 January 2022. Patients with AATD in the IQVIA PharMetrics Plus database were included with ≥1 inpatient or ≥2 outpatient medical claims ≥90 days apart with a diagnosis of AATD, or with records indicating a protease inhibitor (Pi)*ZZ/Pi*MZ genotype in the IQVIA AEMR database with linkage to IQVIA PharMetrics Plus. For a patient's identified continuous enrollment period, patient time was assigned to health states based on the initial encounter with an LD/LgD diagnosis. A unique index date was defined for each health state, and HCRU and costs were calculated per person-year (PPY). <b>Results:</b> Overall, 5136 adult and pediatric patients from the IQVIA PharMetrics Plus and IQVIA AEMR databases were analyzed. All-cause and liver-associated HCRU and costs were substantially higher following onset of LD/LgD. All-cause cost PPY ranged from US $11,877 in the absence of either LD/LgD to US $74,015 in the presence of both LD and LgD. Among liver transplant recipients in the AATD with LD health state, liver-associated total costs PPY were US $87,329 1-year pre-transplantation and US $461,752 1-year post-transplantation. In the AATD with LgD and AATD with LD and LgD health states, patients who received augmentation therapy were associated with higher all-cause total costs PPY and lower liver-associated total costs PPY than their counterparts who did not receive augmentation therapy. <b>Conclusion:</b> Patients with AATD had increased HCRU and healthcare costs in the presence of LD and/or LgD. HCRU and healthcare costs were highest in the AATD with LD and LgD health state.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230186"},"PeriodicalIF":1.9,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145523/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140849826","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":"Cost-effectiveness of sotagliflozin for the treatment of patients with diabetes and recent worsening heart failure.","authors":"Jaehong Kim, Shanshan Wang, Slaven Sikirica, Jason Shafrin","doi":"10.57264/cer-2023-0190","DOIUrl":"10.57264/cer-2023-0190","url":null,"abstract":"<p><p><b>Aim:</b> To assesses the cost-effectiveness of sotagliflozin for the treatment of patients hospitalized with heart failure and comorbid diabetes. <b>Materials & methods:</b> A <i>de novo</i> cost-effectiveness model with a Markov structure was created for patients hospitalized for heart failure with comorbid diabetes. Outcomes of interest included hospital readmissions, emergency department visits and all-cause mortality measured over a 30-year time horizon. Baseline event frequencies were derived from published real-world data studies; sotagliflozin's efficacy was estimated from SOLOIST-WHF. Health benefits were calculated quality-adjusted life years (QALYs). Costs included pharmaceutical costs, rehospitalization, emergency room visits and adverse events. Economic value was measured using the incremental cost-effectiveness ratio (ICER). <b>Results:</b> Sotagliflozin use decreased annualized rehospitalization rates by 34.5% (0.228 vs 0.348, difference: -0.120), annualized emergency department visits by 40.0% (0.091 vs 0.153, difference: -0.061) and annualized mortality by 18.0% (0.298 vs 0.363, difference: -0.065) relative to standard of care, resulting in a net gain in QAYs of 0.425 for sotagliflozin versus standard of care. Incremental costs using sotagliflozin increased by $19,374 over a 30-year time horizon of the patient, driven largely by increased pharmaceutical cost. Estimated ICER for sotagliflozin relative to standard of care was $45,596 per QALY. <b>Conclusion:</b> Sotagliflozin is a cost-effective addition to standard of care for patients hospitalized with heart failure and comorbid diabetes.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230190"},"PeriodicalIF":1.9,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11145521/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141071025","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":"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":2.1,"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}