Journal of comparative effectiveness research最新文献

{"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":"","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":"","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 7.","authors":"Sreeram V Ramagopalan, Annie Jullien Pannelay","doi":"10.57264/cer-2025-0043","DOIUrl":"10.57264/cer-2025-0043","url":null,"abstract":"<p><p>In this update, we discuss the implementation progress of the Medicare Drug Price Negotiation Program, with particular focus on the second round of negotiations for 15 additional Part D drugs; explore the role of health technology assessment in US commercial health plan decision-making; and examine significant changes to pharmaceutical pricing systems in Germany, where international reference pricing has been eliminated as part of the Medical Research Act.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250043"},"PeriodicalIF":1.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144039597","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":"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":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact analysis of expanded access to ketamine for treatment-resistant depression.","authors":"Thanh Lu, Sophia D'Angelo, Zohra Tayebali, Matthew Dempsey, Kristen Giombi, Olga Khavjou","doi":"10.57264/cer-2024-0233","DOIUrl":"10.57264/cer-2024-0233","url":null,"abstract":"<p><p><b>Aim:</b> This study aimed to estimate the economic impacts of expanded access to ketamine relative to electroconvulsive therapy (ECT) by offering intravenous ketamine to US patients with nonpsychotic treatment-resistant depression (TRD) and moderate-to-severe depression. <b>Materials & methods:</b> A population-level Markov simulation model with key parameters from a randomized trial was used to simulate the economic impacts of managing TRD with intravenous ketamine versus ECT over a 5-year horizon. Health states included response of depression in the acute treatment phase and continued treatment and relapse in the maintenance phase. The model estimated costs associated with healthcare utilization (direct costs) and time loss (indirect costs) from patient, caregiver, payer and societal perspectives. Model uncertainty was assessed with one-way sensitivity, probabilistic sensitivity and scenario analyses. <b>Results:</b> In year 1, our model included 350,000 eligible patients. In years 2 through 5, our model added 11,296 eligible patients annually. Expanded access to ketamine to manage TRD was projected to increase the number of patients receiving treatment by 75,000 patients in year 1 and 4292 patients annually in subsequent years. Over 5 years, expanded access to ketamine would result in a net positive societal savings of $828.2 million annually ($95.3 million to patients and $743.7 million to payers). However, expanded ketamine access would impose an additional $10.8 million burden on caregiver time annually. <b>Conclusion:</b> For US patients with TRD and moderate-to-severe depression, ketamine may be a noninferior treatment relative to ECT to improve depression symptoms. Expanded access to ketamine treatment would result in net savings to the patients, payers and society.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240233"},"PeriodicalIF":1.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144119849","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":"Network connectivity, between-study heterogeneity and timepoint challenges in generalized myasthenia gravis: a feasibility assessment of indirect treatment comparisons.","authors":"Nils Erik Gilhus, Saiju Jacob, Mahmoud Hashim, Suzy Van Sanden, Christopher Drudge, Anna Nero, Sumeet Singh, Kavita Gandhi, Brian Hutton","doi":"10.57264/cer-2025-0009","DOIUrl":"10.57264/cer-2025-0009","url":null,"abstract":"<p><p><b>Aim:</b> We performed a feasibility assessment to systematically evaluate randomized controlled trials (RCTs) for generalized myasthenia gravis (gMG) treatments. The goal was to identify the advantages and disadvantages of different indirect treatment comparison (ITC) methods. <b>Materials & methods:</b> A systematic literature review was conducted to identify relevant gMG RCTs for ITCs. The feasibility of ITCs was assessed by comparing design (including study duration and dosing schedules), population and outcome characteristics of retrieved trials, investigating network connectivity and considering appropriate ITC methods to address identified challenges. <b>Results:</b> The feasibility assessment considered 15 relevant RCTs for gMG treatments. Several barriers to conducting robust ITCs were identified, including within-trial imbalances in patient characteristics, small trial sizes and cross-trial differences in potential treatment effect modifiers (TEMs; e.g., antibody status, disease duration and prior treatment exposure). Further, heterogeneity in placebo administration characteristics and background therapies, and cross-trial variation in placebo response for key outcomes were noted. Additionally, treatment strategies (i.e., cyclical vs continuous), dosing schedules and outcome assessment timepoints were inconsistent across trials, necessitating careful consideration of methods and timepoints when interpreting outcomes. The findings suggest that ITCs anchored on placebo as a common comparator may be prone to bias, and more than one ITC approach may be necessary. <b>Conclusion:</b> ITC analyses in gMG have inherent challenges related to imbalanced treatment effect modifiers, network connectivity, varying dosing strategies and assessment timepoints. Multiple approaches to ITCs, with careful evaluation of underlying assumptions and limitations, are advised to limit bias and ensure robust comparative efficacy estimates are available to decision makers.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250009"},"PeriodicalIF":1.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143995588","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A comparison of healthcare resource utilization and costs between patients with chronic lymphocytic leukemia treated with first-line ibrutinib or acalabrutinib using two large US real-world databases.","authors":"Kerry A Rogers, Benyam Muluneh, Zaina P Qureshi, Jinghua He, Alex Bokun, Zhijie Ding, Marie-Hélène Lafeuille, Priyanka Gogna, Bruno Emond, Michael Fradley","doi":"10.57264/cer-2024-0210","DOIUrl":"10.57264/cer-2024-0210","url":null,"abstract":"<p><p><b>Aim:</b> Real-world evidence comparing healthcare resource utilization (HRU) and costs between ibrutinib and acalabrutinib, two Bruton's tyrosine kinase inhibitors for the treatment of chronic lymphocytic leukemia and small lymphocytic lymphoma (CLL/SLL) is limited. <b>Materials & methods:</b> Commercial claims from IQVIA PharMetrics Plus and electronic medical records from Acentrus were used to separately evaluate HRU and costs in CLL/SLL patients initiating first-line (1L) single-agent ibrutinib or acalabrutinib on or after 21 November 2019 (index date). Imputed costs were used for Acentrus using previously published assumptions. Regression analyses adjusted for baseline characteristics were used to compare HRU and costs between ibrutinib and acalabrutinib during 1L therapy. <b>Results:</b> In IQVIA, 537 and 355 patients initiated 1L ibrutinib and acalabrutinib, respectively; in Acentrus, 710 and 373 patients initiated 1L ibrutinib and acalabrutinib, respectively. The mean duration of 1L (in years) was longer for ibrutinib (IQVIA: 1.2; Acentrus: 1.3) than acalabrutinib (IQVIA: 0.8; Acentrus: 0.9). The number of CLL/SLL-related outpatient visits were significantly lower for ibrutinib versus acalabrutinib (IQVIA: 0.86 vs 1.09 per-patient-per-month, rate ratio: 0.85, p = 0.018; Acentrus: 0.57 vs 0.74 per-patient-per-month, rate ratio: 0.80, p = 0.036). Using claims data for IQVIA and imputed costs for Acentrus, total all-cause costs (IQVIA: mean monthly cost difference [MMCD]: -$764, p = 0.279; Acentrus: MMCD: -$1355, p = 0.004) and CLL/SLL related costs (IQVIA: MMCD: -$649, p = 0.133; Acentrus: MMCD: -$1215, p = 0.004) were lower for ibrutinib versus acalabrutinib. <b>Conclusion:</b> In this large real-world study using a mix of claims data and imputed cost estimates, CLL/SLL patients treated with ibrutinib had longer duration of 1L, fewer days with CLL/SLL-related outpatient services and numerically lower all-cause and CLL/SLL-related costs versus acalabrutinib, showing that ibrutinib can be an optimal cost-effective option in 1L.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240210"},"PeriodicalIF":1.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144027630","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-effectiveness and budget impact analysis of switching from apixaban to rivaroxaban treatment among patients with nonvalvular atrial fibrillation in a German healthcare setting.","authors":"Rupesh Subash, Thomas Strakosch, Michelle Zhang, Melissa Hagan, Elke Dworatzek, Agnes Kisser, Vasileios Vasilopoulos, Chloe Salter, Carissa Dickerson, Ewa Stawowczyk","doi":"10.57264/cer-2025-0008","DOIUrl":"10.57264/cer-2025-0008","url":null,"abstract":"<p><p><b>Aim:</b> Direct oral anticoagulant (DOAC) switching often occurs in patients with nonvalvular atrial fibrillation (NVAF) for medical and nonmedical reasons. Limited data describe the economic consequences of DOAC switching in patients with NVAF. This study evaluates the cost-effectiveness and budget impact of initiating apixaban and switching to rivaroxaban versus initiating and continuing apixaban for patients with NVAF, from a German payer perspective. <b>Materials & methods:</b> Built on an existing model, a cohort-level lifetime Markov model was developed, including dynamic pricing assumptions to account for anticipated generic entry of DOACs. The modeled population (n = 1000) included German patients with NVAF, eligible for oral anticoagulation, who initiated on apixaban. The primary model outcome was the incremental cost-effectiveness ratio, assessed using cost per quality-adjusted life year (QALY) gained and a willingness-to-pay threshold of €48,750/QALY. A secondary model outcome was a 5-year budget impact analysis. <b>Results:</b> Switching patients from apixaban to rivaroxaban led to 285 additional events per 1000 patient years, resulting in 0.079 fewer QALYs and higher total costs per patient (€21,357 vs €16,390 for apixaban continuers). In the base case analysis (with generic pricing assumptions), switching from apixaban to rivaroxaban was dominated (i.e., less effective and more costly) by continuing apixaban. In the budget impact analysis (with generic pricing assumptions), switching from apixaban to rivaroxaban led to additional cumulative costs of €490 per patient over 5 years. <b>Conclusion:</b> Despite the introduction of generic discounting, switching patients with NVAF from apixaban to rivaroxaban led to higher total costs and fewer QALYs under base case assumptions, meaning apixaban switchers were dominated by apixaban continuers from a German payer perspective. Switching patients from apixaban to rivaroxaban also led to greater budget impact over 5 years.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250008"},"PeriodicalIF":1.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144110691","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":"Corrigendum.","authors":"","doi":"10.57264/cer-2025-0050","DOIUrl":"10.57264/cer-2025-0050","url":null,"abstract":"","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250050"},"PeriodicalIF":1.9,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143968871","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":"Comparative safety of B/F/TAF versus other antiretroviral therapy regimens for treatment-naïve people with HIV-1: a systematic literature review and network meta-analysis.","authors":"Tristan Curteis, Lucy A Eddowes, Megan Chen, Bhumi Gandhi-Patel, Andrei Karlsson, Hannah Luedke, Manon Rubinstein, Mathias Hempfling, Ernesto G Scerpella, Paresh Chaudhari, James Jarrett","doi":"10.57264/cer-2024-0231","DOIUrl":"https://doi.org/10.57264/cer-2024-0231","url":null,"abstract":"<p><p><b>Aim:</b> Bictegravir, emtricitabine and tenofovir alafenamide (B/F/TAF) is a single-tablet regimen approved for the treatment of HIV-1 in treatment-naive (TN) and virologically suppressed people with HIV-1 (PWH). While the efficacy of antiretroviral therapy (ART) regimens in TN PWH is well established, regimen selection is often influenced by safety and tolerability concerns. This systematic literature review and network meta-analysis compares the safety outcomes of B/F/TAF with other ART regimens in TN PWH, addressing a key aspect of therapeutic decision-making. <b>Materials & methods:</b> A systematic literature review was performed to identify phase III and IV RCTs assessing B/F/TAF and comparator regimens in TN adults (≥18 years) with HIV-1. MEDLINE, Embase, Cochrane Database of Systematic Reviews and CENTRAL databases were last searched on 14 June 2023. Study design, population and outcome definitions were evaluated to ensure consistency across studies. Bayesian network meta-analyses were conducted where feasible, following key methodological guidelines. <b>Results:</b> Nineteen studies were included in indirect comparisons following assessments of heterogeneity and network connectivity. B/F/TAF performed better than the majority of interventions in safety and tolerability outcomes, notably discontinuation due to adverse events (AEs), treatment-related AEs and nausea. Additionally, with comparable risk of experiencing grade 3/4 treatment-emergent AEs, diarrhea and all-cause discontinuation rates to other interventions, B/F/TAF was non-inferior to any other regimen for all outcomes. B/F/TAF typically outperformed interventions containing non-nucleoside reverse transcriptase inhibitor third agents in terms of treatment-related AEs, nausea, and discontinuation due to AEs, although treatment class effects were not estimated separately. <b>Conclusion:</b> This analysis highlights the favorable safety profile of B/F/TAF compared with other ART regimens in TN PWH, demonstrating that B/F/TAF remains a safe and well-tolerated ART option for most TN PWH.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e240231"},"PeriodicalIF":1.9,"publicationDate":"2025-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144181430","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 19.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2025-0063","DOIUrl":"https://doi.org/10.57264/cer-2025-0063","url":null,"abstract":"<p><p>In this update, we examine a checklist for real-world evidence use in Medicare drug price negotiations under the Inflation Reduction Act, a review of health technology assessment reassessments across major health technology assessment agencies, and two complementary studies that used natural experiment approaches to explore the relationship between herpes zoster vaccination and reduced dementia risk.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250063"},"PeriodicalIF":1.9,"publicationDate":"2025-05-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144022664","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}