Journal of comparative effectiveness research最新文献

{"title":"Phosphodiesterase type 5 inhibitors as treatment for erectile dysfunction: a webinar-based poll unveiling perceptions of healthcare professionals.","authors":"Maher S Moazin, Abdulaziz Baazeem, Ayman Al-Bakri, Adel Al Dayel, Ashraf Amir, Saud Al Sifri, Mona Reda, Fahad Bashraheel, Abdullah Alfakhri, Yousef Hamdy, Fayez Elshaer","doi":"10.57264/cer-2023-0155","DOIUrl":"10.57264/cer-2023-0155","url":null,"abstract":"<p><p><b>Aim:</b> Erectile dysfunction (ED) is marked by a recurring incapacity to achieve or uphold a satisfactory erection during sexual activities. The study aims to increase awareness about male reproductive health, dispel misconceptions about ED and encourage physician-patient discussions. <b>Materials & methods:</b> A live online poll was conducted during a 3-day webinar titled \"Turning the Tide of Men's Health\" organized by Viatris™ in collaboration with the Saudi Society of Family and Community Medicine and attended by healthcare professionals (HCPs) from diverse specialties. The attendees voluntarily responded to nine poll questions on adherence to ED medication, use of phosphodiesterase type 5 inhibitors (PDE5is) as cure versus management of ED and patients' challenges. The responses to the poll questions were recorded and assessed to understand the perceptions of HCPs. <b>Results:</b> The poll garnered 10,423 responses from 5831 attendees on the second day of the webinar. The key findings included HCPs' perceptions that PDE5is contribute to ED management not complete cure. The respondents acknowledged that adherence to ED medications might decline on days without planned sexual activity, and long-term adherence on a daily PDE5i is exhibited by a relatively modest percentage of ED patients. The consensus among respondents was that PDE5is do not enhance or generate sexual desire, and the daily schedule of ED treatment may be burdensome for some patients. <b>Conclusion:</b> The findings from this poll offer insights into the perspectives of HCPs regarding the usage of PDE5is to treat ED. Responders of the poll generally agreed that PDE5is can help manage ED without affecting desire, though adherence may be lower on days without planned sexual activity. Furthermore, most respondents acknowledged that adhering to a daily pill regimen posed a greater burden than waiting for the medication to take effect.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230155"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225304/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141076178","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 among nursing home residents with Parkinson's disease psychosis: an analysis of Medicare beneficiaries treated with pimavanserin or other-atypical antipsychotics.","authors":"Krithika Rajagopalan, Nazia Rashid, Daksha Gopal, Dilesh Doshi","doi":"10.57264/cer-2024-0038","DOIUrl":"10.57264/cer-2024-0038","url":null,"abstract":"<p><p><b>Aim:</b> Real-world healthcare resource use (HCRU) burden among patients with Parkinson's disease psychosis (PDP) treated with pimavanserin (PIM) versus other atypical antipsychotics (other-AAPs) including quetiapine (QUE) in long term care (LTC) and nursing home (NH) settings are lacking. This analysis examines HCRU differences among residents in LTC/NH settings who initiate PIM versus QUE or other-AAPs. <b>Methods:</b> A retrospective analysis of LTC/NH residents with PDP from the 100% Medicare claims between 1 April 2015 and 31 December 2021 was conducted. Treatment-naive residents who initiated ≥6 months continuous monotherapy with PIM or QUE or other-AAPs between 04/01/16 and 06/30/2021 were propensity score matched (PSM) 1:1 using 31 variables (age, sex, race, region and 27 Elixhauser comorbidity characteristics). Post-index (i.e., 6 months) HCRU outcomes included: proportion of residents with ≥1 all-cause inpatient (IP) hospitalizations and emergency room (ER) visits. HCRU differences were assessed via log binomial regression and reported as relative risk ratios (RR) and 95% confidence intervals after controlling for dementia, insomnia and index year. <b>Results:</b> From a total of PIM (n = 1827), QUE (n = 7770) or other-AAPs (n = 9557), 1:1 matched sample (n = 1827) in each cohort were selected. All-cause IP hospitalizations (PIM [29.8%]) versus QUE [36.7%]) and ER visits (PIM [47.3%] versus QUE [55.8%]), respectively, were significantly lower for PIM. PIM versus QUE cohort also had significantly lower RR for all-cause IP hospitalizations and ER visits, respectively, (IP hospitalizations RR: 0.82 [0.75. 0.9]; ER visits RR: 0.85 [0.8. 0.9]). PIM versus other-AAPs also had lower likelihood of HCRU outcomes. <b>Conclusion:</b> In this analysis, LTC/NH residents on PIM monotherapy (versus QUE) had a lower likelihood of all-cause hospitalizations (18%) and ER (15%) visits. In this setting, PIM also had lower likelihood of all-cause HCRU versus other-AAPs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240038"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225156/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141288182","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":"Comparing prospectively assigned trial and real-world lung cancer patients.","authors":"Brigham Walker, Herman E Ray, Ping Shao, Claudio D'Ambrosio, Craig White, Mark S Walker","doi":"10.57264/cer-2023-0176","DOIUrl":"10.57264/cer-2023-0176","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the comparability of a probable clinical trial (CT) cohort derived from electronic medical records (EMR) data with a real-world cohort treated with the same therapy and identified using the same inclusion and exclusion criteria to emulate an external control. <b>Methods:</b> We utilized de-identified patient-level structured data sourced from EMRs. We then compared patterns of overall survival (OS) between probable CT patients with those drawn from non-contemporaneous real-world data (RWD) using a two-sided log-rank test, hazard ratios (HRs) using a Cox proportional-hazards model and Kaplan-Meier (KM) survival curves. Each regression estimate was calculated with a corresponding 95% confidence interval. We additionally conducted multiple matching methods to assess their relative performance. <b>Results:</b> Median (standard deviation) OS was 10.2 (0.7) months for the RWD arm and 11.3 (1.3) for the probable CT arm with a Log rank p-value equal to 0.4771. OS in both cohorts is longer than the reported CT median OS of 9.2 (0.6). The HRs generated under all five assessed matching methods (including without adjustment) were not statistically significant at the 95% confidence level. <b>Conclusion:</b> Our results suggest, with caveats noted, that survival patterns between real-world and CT cohorts in this NSCLC setting are not statistically significantly different.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230176"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11225159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141087722","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":"Six-minute walk test as clinical end point in cardiomyopathy clinical trials, including ATTR-CM: a systematic literature review.","authors":"Jose Nativi-Nicolau, Ali Yilmaz, Noel Dasgupta, Richard Macey, James Cochrane, Judith Peatman, Catherine Summers, Jennifer Luth, Ronald Zolty","doi":"10.57264/cer-2023-0158","DOIUrl":"10.57264/cer-2023-0158","url":null,"abstract":"<p><p><b>Aim:</b> The six-minute walk test (6MWT) is a common measure of functional capacity in patients with heart failure (HF). Primary clinical study end points in cardiomyopathy (CM) trials, including transthyretin-mediated amyloidosis with CM (ATTR-CM), are often limited to hospitalization and mortality. <b>Objective:</b> To investigate the relationship between the 6MWT and hospitalization or mortality in CM, including ATTR-CM. <b>Method:</b> A PRISMA-guided systematic literature review was conducted using search terms for CM, 6MWT, hospitalization and mortality. <b>Results:</b> Forty-one studies were identified that reported 6MWT data and hospitalization or mortality data for patients with CM. The data suggest that a greater 6MWT distance is associated with a reduced risk of hospitalization or mortality in CM. <b>Conclusion:</b> The 6MWT is an accepted alternative end point in CM trials, including ATTR-CM.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e230158"},"PeriodicalIF":1.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11234454/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141310769","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":"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}