Journal of comparative effectiveness research最新文献

{"title":"Cost impact of Bruton's tyrosine kinase inhibitor selection in Medicare patients with chronic lymphocytic leukemia.","authors":"Adam S Kittai, Dipen A Patel, Jason Shafrin, Nadine Zawadzki, Vikram S Shetty, Yazan K Barqawi, Joanna M Rhodes","doi":"10.57264/cer-2025-0035","DOIUrl":"https://doi.org/10.57264/cer-2025-0035","url":null,"abstract":"<p><p><b>Aim:</b> To estimate cost savings associated with covalent Bruton's tyrosine kinase inhibitor (cBTKi) choice in patients with treatment-naive (TN) and relapsed/refractory (RR) chronic lymphocytic leukemia (CLL) from a Medicare perspective. <b>Materials</b> <b>&</b> <b>methods:</b> An economic model with Markov structure simulated outcomes in patients with CLL initiating ibrutinib, acalabrutinib or zanubrutinib monotherapy. Modeled population included TN and RR patients who had no prior cBTKi. Treatments were dosed per US FDA label and efficacy assumed identical across cBTKis. Cumulative grade ≥3 adverse event (AE) rates were drawn from extended follow-up of cBTKi phase III clinical trials at similar duration. Costs included drug price per 2024 wholesale acquisition cost and AE management medical costs from literature, adjusted for Medicare reimbursement. Outcomes were total change in payer cost over 1, 3 and 5 years. <b>Results:</b> A cohort of 13,726 patients with CLL was modeled (44% TN, 56% RR). Acalabrutinib's aggregate grade ≥3 AE rate was 25.8% points less in TN patients (35.8% vs 61.6%) and 8.0% points less in RR patients (75.0% vs 83.0%) compared with ibrutinib, and 20.6% points less in TN patients (35.8% vs 56.4%) and 11.1% points less in RR patients (75.0% vs 86.1%) compared with zanubrutinib. Acalabrutinib saved $15,478 more per patient versus ibrutinib in year 1 due to lower treatment cost (-$12,076) and lower AE cost (-$3402). Acalabrutinib also saved $1901 more per patient versus zanubrutinib as acalabrutinib higher treatment cost (+$1663) was offset by lower AE cost (-$3563). Across all patients, acalabrutinib saved $212 million more versus ibrutinib and $26 million more versus zanubrutinib from a Medicare perspective. Acalabrutinib cost savings persisted over 3 and 5 years. <b>Conclusion:</b> Acalabrutinib yielded cost savings versus ibrutinib and zanubrutinib for patients with CLL in Medicare due to lower treatment cost versus ibrutinib and fewer grade ≥3 AEs versus both ibrutinib and zanubrutinib.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250035"},"PeriodicalIF":1.9,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144368899","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":"Access in all areas? A round up of developments in market access and health technology assessment: part 8.","authors":"Sreeram V Ramagopalan, Annie Jullien Pannelay","doi":"10.57264/cer-2025-0091","DOIUrl":"https://doi.org/10.57264/cer-2025-0091","url":null,"abstract":"<p><p>In this update, we discuss the pricing paradox of combination therapies in health technology assessment; examine the Inflation Reduction Act's impact on pharmaceutical innovation and analyze the revised Dutch economic evaluation guidelines.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250091"},"PeriodicalIF":1.9,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144310007","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":"Quality in qualitative evidence: new best practice principles from NICE's real-world evidence framework.","authors":"Vandana Ayyar Gupta, Sarah Scott, Mandy Tonkinson, Pall Jonsson, Lesley Goodburn, Stephen Duffield","doi":"10.57264/cer-2025-0064","DOIUrl":"https://doi.org/10.57264/cer-2025-0064","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250064"},"PeriodicalIF":1.9,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144284899","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":"Resource utilization and economic outcomes following repetitive transcranial magnetic stimulation for treatment-resistant depression: a retrospective observational analysis.","authors":"Joseph J Taylor, Andrew J Manett, Michael Feyder, Brandon S Bentzley","doi":"10.57264/cer-2025-0019","DOIUrl":"https://doi.org/10.57264/cer-2025-0019","url":null,"abstract":"<p><p><b>Aim:</b> We investigated the impact of repetitive transcranial magnetic stimulation (rTMS) for treatment-resistant depression on healthcare resource utilization as well as commercial and Medicare Fee-for-Service payer costs. <b>Materials & methods:</b> We conducted a retrospective observational analysis of claims data using Medicare Fee-for-Service datasets and commercial (Merative MarketScan Research Databases) datasets from 1 January 2021 to 30 September 2023. We identified two cohorts, a cohort that received rTMS and a cohort not treated with rTMS over an 18-month period. We used propensity score matching to balance the baseline characteristics of the cohorts, and we calculated the total cost of care based on payer allowed amounts from Merative MarketScan Research Databases and Standard Analytical Files. <b>Results:</b> Relative to the non-TMS cohort, the rTMS cohort incurred 37% more hospital outpatient visits (14.00 vs 10.21; p ≤ 0.0001) with 7% higher outpatient cost ($8946 vs $8363; p = 0.3400). Simultaneously, the rTMS cohort incurred 24% fewer inpatient admissions (0.25 vs 0.33; p = 0.0003) with 19% lower inpatient admission costs ($5666 vs $6978; p = 0.0392), 48% fewer emergency room visits (0.27 vs 0.53; p ≤ 0.0001) with 34% lower emergency room costs ($322 vs $487; p ≤ 0.0001), and $893 less in episode of care costs. <b>Conclusion:</b> This study suggests that patients who receive rTMS for treatment-resistant depression required fewer high acuity hospital visits and incurred less expensive episode-of-care costs compared with patients who do not receive rTMS. From this perspective, rTMS is an investment that returns health and economic dividends through fewer high acuity hospital visits.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250019"},"PeriodicalIF":1.9,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144258180","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-per-remitter for esketamine nasal spray versus quetiapine for treatment-resistant depression.","authors":"Kristin Clemens, Amanda Teeple, Benoit Rive, Noam Kirson, Urvi Desai, Jason Doran, Diab Eid, Alice Qu, Hannah Bowrey, Kruti Joshi","doi":"10.57264/cer-2024-0092","DOIUrl":"https://doi.org/10.57264/cer-2024-0092","url":null,"abstract":"<p><p><b>Aim:</b> Estimate the cost-per-remitter with esketamine nasal spray plus an oral antidepressant (ESK NS + OAD) versus quetiapine extended release plus an oral antidepressant (QTP XR + OAD) among adults with treatment-resistant depression (TRD). <b>Materials & methods:</b> An Excel-based model was developed to estimate the cost-per-remitter for ESK NS + OAD and QTP XR + OAD from the perspective of a US commercial insurance plan and Medicaid. Remission and response rates were estimated in 4-week intervals over 32 weeks using data from the ESCAPE-TRD phase IIIb clinical trial comparing ESK NS + OAD versus QTP XR + OAD in adults with TRD. Direct healthcare costs were sourced from health economic literature and the RED BOOK^® drug pricing database. Indirect costs were derived from a separate analysis of ESCAPE-TRD using the Work Productivity and Activity Impairment: Depression questionnaire. Adults not remitting/responding either stayed on current treatment or discontinued current treatment and initiated either augmented therapy with antipsychotics or repetitive transcranial magnetic stimulation. In a scenario analysis, all individuals who did not achieve response and discontinued treatment initiated repetitive transcranial magnetic stimulation. <b>Results:</b> The remission rate at 32 weeks was 50% for adults receiving ESK NS + OAD and 33% for adults receiving QTP XR + OAD. The cost-per-remitter for ESK NS + OAD compared with QTP XR + OAD was $3102.17 lower in the commercial setting and $456.12 lower in the Medicaid setting. Under the scenario analysis, the cost-per-remitter for ESK NS + OAD compared with QTP XR + OAD was $15,133.66 lower in the commercial setting and $12,487.62 lower in the Medicaid setting. <b>Conclusion:</b> The findings suggest that ESK NS + OAD is a cost-effective treatment for adults with TRD compared with QTP XR + OAD in the commercial and Medicaid settings.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240092"},"PeriodicalIF":1.9,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248129","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":"Expert perspectives on the use of safinamide for Parkinson's disease in Portugal: insights from a Portuguese Delphi Consensus.","authors":"Ana Margarida Rodrigues, Cristina Costa, Miguel Gago, Miguel Grunho, Leonor Correia Guedes, Ana Morgadinho, Maria José Rosas, Rita Simões, Ana Graça Velon","doi":"10.57264/cer-2024-0228","DOIUrl":"https://doi.org/10.57264/cer-2024-0228","url":null,"abstract":"<p><p><b>Aim:</b> Safinamide is an approved medication for managing motor fluctuations in Parkinson's disease (PD). However, limited data exist regarding its application in clinical practice in Portugal and the perspectives of Portuguese neurologists on its use. To address this, a group of Portuguese specialists with recognized expertise in PD management convened to compile the insights on various aspects of safinamide use in PD patients among the field and to develop recommendations aimed at informing and guiding physicians in Portugal on its optimal clinical application. <b>Materials & methods:</b> A focus group composed of nine Portuguese PD experts developed a questionnaire building on the 2022 European Delphi study, and employed a Delphi methodology approach to gather the views of Portuguese neurologists with a minimum of 5 years of clinical experience with safinamide (n = 35). A final online questionnaire comprising 35 statements was administered in a single-round Delphi format, utilizing a 5-point Likert scale. Consensus was defined as achieving ≥66% agreement or disagreement among the panelists. <b>Results:</b> A strong consensus emerged among Portuguese neurologists regarding the therapeutic efficacy of safinamide in addressing motor symptoms, motor fluctuations and quality of life. Additionally, agreement was reached on its positive effects on nonmotor symptoms such as sleep, fatigue, mood, quality of life and pain management. However, no consensus was achieved regarding safinamide's efficacy in managing orthostatic hypotension, cognitive issues, urinary and sexual dysfunction, as well as its safety profile in PD patients with hallucinations. Overall, the opinions of Portuguese neurologists aligned closely with those of their European counterparts. <b>Conclusion:</b> This Delphi study highlights the consensus among Portuguese neurologists on the efficacy of safinamide for managing motor symptoms in PD. The considerations presented herein offer essential guidance for effectively managing PD with safinamide, ultimately enhancing patient care in Portugal.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240228"},"PeriodicalIF":1.9,"publicationDate":"2025-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144234310","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":"Tr14 gel for the treatment of acute ankle sprains: a plain language summary of the TRAUMED trial.","authors":"Ludger Gerdesmeyer, Helmut Pabst, Konstantin Cesnulevicius, Myron Schultz, Alta Smit, Gino Kerkhoffs","doi":"10.57264/cer-2025-0018","DOIUrl":"https://doi.org/10.57264/cer-2025-0018","url":null,"abstract":"<p><strong>What is this summary about?: </strong>This is a summary of an article discussing the results of the TRAUMED trial, which was originally published in the <i>Journal of Clinical Medicine</i>. This trial studied how well Tr14 gel helped with ankle sprains compared with either a dummy treatment (placebo) or a common painkiller called diclofenac.</p><p><strong>What were the results of this trial, and what do they mean?: </strong>The results of this trial showed that Tr14 was effective at reducing pain caused by an acute or sudden ankle sprain compared with placebo and worked just as well as diclofenac. The results also suggested that Tr14 led to faster pain relief and improved foot and ankle function compared with placebo and was at least as effective as diclofenac. <b>Clinical Trial Registration:</b> NCT06192420.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"CER"},"PeriodicalIF":1.9,"publicationDate":"2025-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144225639","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":"Single-use endoscopy for the evaluation of swallowing: a performance survey.","authors":"Christina Cool, Ian Haislip, Christian Escobar, Jacqueline Mojica","doi":"10.57264/cer-2024-0113","DOIUrl":"https://doi.org/10.57264/cer-2024-0113","url":null,"abstract":"<p><p><b>Aim:</b> Single-use endoscopes have become of interest across a multitude of procedure types given the risk of cross-contamination and availability of scopes; the performance capability of a single-use scope has not formally been evaluated for the flexible endoscopic evaluation of swallowing (FEES) indication. The primary objective was to measure FEES procedural success with single-use rhinolaryngoscopes without the need for a secondary scope, while the secondary objective was to evaluate the overall performance and usability of the single-use platform. <b>Materials & methods:</b> Twenty-three speech-language pathologists across 16 sites performed FEES procedures with the Ambu aScope 4 RhinoLaryngo Slim. After the procedures, participants completed a nine-question survey questionnaire. A five-point rating system was used to quantify scope performance. Mean ratings were calculated and a one-sample <i>t</i>-test was performed. <b>Results:</b> The primary end point of procedural success was achieved in 100% of procedures. Of the nine performance metrics captured for aScope 4 RhinoLaryngo Slim, six received a score of excellent by 100% of participants with the lowest score being a 4.2. <b>Conclusion:</b> Single-use rhinolaryngoscopes can successfully perform FEES procedures without additional scoping needed, and received high performance ratings among users. <b>Level of evidence:</b> 4.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240113"},"PeriodicalIF":1.9,"publicationDate":"2025-06-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144215995","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":"Time-limited reimbursement and Temporary Access Process for early access to oncology treatments in Canada: a perspective based on the epcoritamab experience.","authors":"Chakrapani Balijepalli, Lakshmi Gullapalli, Swati Prasad, Nancy Paul Roc, Ana Rusu, Natalia Price, William Dempster, Stephane Barakat","doi":"10.57264/cer-2025-0024","DOIUrl":"10.57264/cer-2025-0024","url":null,"abstract":"<p><p>For years, Canadians have faced long wait times for access to new medicines. These delays are largely attributed to complex health technology assessments, extended price negotiations and protracted provincial listing decisions. To address these challenges, in November 2023, Canada's Drug Agency (CDA) introduced its first early access program - the time-limited reimbursement recommendation (TLR) - aimed at accelerating the reimbursement of promising drugs undergoing Health Canada's Notice of Compliance with Conditions (NOC/c) process. In conjunction, the pan-Canadian Pharmaceutical Alliance developed the Temporary Access Process (pTAP) to support price negotiations for drugs that go through CDA's TLR pathway. AbbVie corporation was the first company to participate in the TLR and pTAP processes with EPKINLY (epcoritamab) - a novel treatment for advanced lymphoma. On 18 June 2024, EPKINLY became the first therapy in Canada to receive a positive CDA TLR recommendation and on 19 July 2024, AbbVie and the pan-Canadian Pharmaceutical Alliance successfully concluded pTAP negotiations. As of 1 November 2024, EPKINLY was listed in nine provinces, achieving a 10.7 month faster time-to-patient than the average time for the standard process, which is significant and meaningful to patients. This achievement demonstrates the potential of the TLR and pTAP processes to improve medicine access timelines for patients. However, an analysis of drugs that received NOC/c status from Health Canada between 2020 and 2024 reveals that very few drugs would have met the current strict eligibility criteria required to benefit from the TLR, limiting the potential benefits of these programs. While TLR and pTAP are promising initiatives, refinements are needed to maximize their impact and ensure faster access to life-saving therapies for Canadian patients.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250024"},"PeriodicalIF":1.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12142399/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144012005","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":"Factors impacting chronic disease medication adherence in the UAE: a prospective cohort study, 2021-2022.","authors":"Joel Ladner, Alshurafa Sawsan, Anas Nofal, Mohamed Rana, Malak Ammar, Joseph Saba, Etienne Audureau","doi":"10.57264/cer-2025-0020","DOIUrl":"10.57264/cer-2025-0020","url":null,"abstract":"<p><p><b>Aim:</b> To assess the evolution of chronic disease medication adherence factors and identify factors predictive of long-term adherence in the UAE. <b>Materials & methods:</b> Patients ≥18 years old; newly diagnosed with one of the following diseases: ankylosing spondylitis, heart failure, multiple sclerosis, psoriasis, or asthma and prescribed long-term medication were followed ≥12 months (M12), then categorized as followed (continued treatment by prescribing physician) or lost to follow-up. Adherence was assessed using the Patient Needs Assessment Tool (PNAT), which is based on the WHO's five dimensional framework. <b>Results:</b> A total of 111 patients were included, 17 (15.3%) were lost of follow-up at M12. Time spent in consultation by medical doctor (MD) (adjusted odds ratio = 6.89, 95% CI = 2.07-12.76) and anxiety and stress level (adjusted odds ratio = 0.18, 95% CI = 0.11-0.67) were significant predictive factors associated with remaining on treatment at M12. Self organizing map methodology identified predictive factors associated with remaining on treatment at M12 as: patient satisfaction with time spent with prescribing MD, patient involvement in treatment decision, disease management ability, satisfaction with support from family/friends, low dependence on others for daily life activities, difficulties joining community activities, and acknowledgement of an influential role of cultural habits/spiritual beliefs. The highest means score differences from M0 to M12 were for difficulties joining community activities (difference [diff] M12-M0 = 1.32, p < 10^-4), role of cultural habits (diff = 1.05, p < 10^-4), role of spiritual beliefs (diff = 1.02, p < 10^-4), patient involved in treatment decision (diff = 0.67, p = 0.007), and memory difficulties (diff = 0.62, p < 10^-4). <b>Conclusion:</b> Socio-economic factors changed most significantly over 12 months. The identified factors may be used to develop strategies to improve patient satisfaction with the time they spend with the prescribing MD as well as reduce stress, each of which may improve medication adherence. Understanding patient behavior and accurately quantifying adherence are essential for improving outcomes for patients prescribed chronic disease medication in Gulf Arabic countries.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250020"},"PeriodicalIF":1.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12142380/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144020852","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}