Journal of comparative effectiveness research最新文献

{"title":"Real-world evidence on the use of hospital resources for subcutaneous and intravenous trastuzumab administration in breast cancer patients at a referral public hospital in Mexico.","authors":"Tatiana Fiordelisio, Francisco Valdés-Souto, Diana Del-Rio Valdes, Diego Zamarrón Hernández, Karla Ramírez Pulido, Cristopher Alejandro Escamilla Soto, Yair Uriel Correa Trejo, Claudia Itzel Gutiérrez Sánchez, Isabel Espino Gutiérrez, Claudia Haydeé Arce Salinas","doi":"10.57264/cer-2025-0199","DOIUrl":"10.57264/cer-2025-0199","url":null,"abstract":"<p><p><b>Aim:</b> The availability of subcutaneous (SC) trastuzumab has introduced a practical alternative to traditional intravenous (IV) delivery for HER2-positive breast cancer. Beyond clinical equivalence, the shift toward SC administration offers the possibility of reorganizing workflow, reducing the pressure on infusion units and improving the treatment experience for both patients and healthcare professionals. This study provides real-world evidence from a large public oncology hospital in Mexico City, examining how SC and IV trastuzumab compare in everyday practice with respect to treatment times, resources use and the perceptions of patients and staff. <b>Materials & methods:</b> A prospective time and motion study was conducted to compare the efficiency and operational impact of SC and IV routes of trastuzumab administered as monotherapy in women with HER2-positive breast cancer at a public hospital in Mexico City. Sixty administrations (30 SC and 30 IV) were analyzed, recorded in real time using a digital system specifically developed for this study. The system documented waiting times, preparation, administration and time spent in the treatment room. Resource utilization was estimated based on the average number of supplies required for each route. In addition, patients and healthcare personnel completed structured questionnaires on comfort, satisfaction and overall experience. <b>Results:</b> Treatment using the SC route was noticeably shorter. Compared with the IV route, median chair time and overall procedure duration dropped by close to 94%, and treatment room occupancy decreased by approximately 83%. This route also required fewer materials and avoided the drug loss due to weight-based IV dosing. Patients described the SC injection as more comfortable and less fatiguing, while healthcare professionals noted less physical effort, lower stress levels and a more manageable workflow. <b>Conclusion:</b> In a routine public oncology setting with limited resources, the use of SC trastuzumab was associated with clear advantages in terms of efficiency and the treatment experience while also mitigating the workload of infusion areas. These results provide practical, context-specific evidence that could help healthcare systems to improve the organization of oncology services and make better use of scarce hospital resources.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250199"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147723042","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":"Health economic analysis of pulsed field ablation compared to conventional thermal ablation for patients with paroxysmal atrial fibrillation in China.","authors":"Jian Ye, Youqi Fan, Ye Wang, Yifei Jian, Yue Gao, Shuting Chen, Jianwei Xuan, Hesheng Hu","doi":"10.57264/cer-2025-0153","DOIUrl":"10.57264/cer-2025-0153","url":null,"abstract":"<p><p><b>Aim:</b> To evaluate the cost-utility of pulsed field ablation (PFA) compared with radiofrequency ablation (RFA) and cryoablation, respectively, in Chinese patients with paroxysmal atrial fibrillation. <b>Materials &</b> <b>methods:</b> Patients with paroxysmal atrial fibrillation at different levels of atrial arrhythmia burden (<0.1%, 0.1-9.9%, ≥10%) may experience various atrial arrhythmia burden states after undergoing ablation. A decision tree model was developed to simulate this process from a healthcare perspective, where patients could undergo a repeat ablation or experience a nonfatal stroke. Transition probabilities, clinical outcome and quality of life data were obtained from published sources and confirmed by expert physicians. Cost data were estimated from a survey of clinicians at tertiary hospitals, based on actual clinical practices. The uncertainty of results was explored through one-way sensitivity analysis and probabilistic sensitivity analysis by Monte Carlo simulation. The secondary outcome, return on investment, was calculated from hospital administrator perspective, as net revenue divided by total cost of certain ablation type. <b>Results:</b> PFA demonstrated favorable cost-effectiveness compared with both RFA and cryoablation under the three-times China's per capita GDP threshold. When compared with RFA, PFA yielded an incremental 0.016 quality-adjusted life years (QALYs) (0.859 vs 0.842) with an incremental cost-effectiveness ratio of ¥37,000 per QALY gained. This cost-effectiveness was primarily driven by savings of ¥1558 in weighted repeat ablation surgery costs and ¥506 in long-term medication costs for anti-arhythmic and anticoagulants. When compared with cryoablation, PFA resulted in an incremental 0.006 QALYs (0.859 vs 0.852) with an incremental cost-effectiveness ratio of ¥231,167 per QALY gained, mainly attributed to savings of ¥1300 in repeat ablation surgery costs and ¥133 in long-term medication costs. As a secondary outcome, PFA yielded a return on investment of 0.313. <b>Conclusion:</b> PFA was likely to be more cost effective than both radiofrequency ablation and cryoablation in China. The study suggests that PFA represents a high-value intervention that aligns superior clinical outcomes with favorable hospital financial sustainability, supporting its prioritized adoption in the management of atrial fibrillation in China.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250153"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147772911","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":"Antiretroviral therapy persistence among treatment-experienced people with HIV and mental health disorders and/or substance use disorders in the USA (2017-2024): a retrospective cohort study.","authors":"Uche Mordi, Mary J Christoph, Benjamin Chastek, Travis Lim, Neia Prata Menezes, Sunil Majethia, Lisa B Le, Joshua Cohen","doi":"10.57264/cer-2025-0124","DOIUrl":"10.57264/cer-2025-0124","url":null,"abstract":"<p><p><b>Aim:</b> There are limited studies of antiretroviral therapy persistence among people with HIV (PWH) who have a mental health disorder and/or substance use disorder (MHD/SUD). This real-world study analyzed persistence among treatment-experienced PWH who had an MHD/SUD or suboptimal adherence (proportion of days covered [PDC] < 85%). <b>Materials & methods:</b> In this retrospective cohort study of claims from the Optum Research Database, nonpersistence (i.e., discontinuation, switch, add-on or death) was assessed in treatment-experienced PWH who switched to or restarted an antiretroviral therapy regimen of interest (i.e., bictegravir [B]/emtricitabine [F]/tenofovir alafenamide [TAF], dolutegravir [DTG]/lamivudine [3TC], DTG/abacavir [ABC]/3TC, DTG + F/TAF, DTG + F/tenofovir disoproxil fumarate [TDF] or cabotegravir + rilpivirine) from 1 July 2017 to 30 November 2023. Baseline characteristics across regimens were balanced by inverse probability treatment weighting. Kaplan-Meier analysis was conducted on weighted data to examine regimen persistence at 12 months. Adjusted Cox proportional hazards models assessed nonpersistence throughout follow-up. <b>Results:</b> Overall, 14,826 PWH were eligible and treatment experienced; 5310 had an MHD/SUD and 4090 had PDC < 85% during follow-up. Among treatment-experienced PWH who received B/F/TAF, persistence at 12 months was 80.3% for those with an MHD/SUD and 78.6% for those with PDC < 85%; this was greater compared with PWH who received DTG/3TC (76.7% and 70.8%), DTG/ABC/3TC (68.9% and 66.3%), DTG + F/TAF (62.4% and 58.9%) and DTG + F/TDF (36.7% and 41.9%; all p < 0.05). Compared with B/F/TAF, nonpersistence risk was greater with DTG/3TC, DTG/ABC/3TC, DTG + F/TAF and DTG + F/TDF in the MHD/SUD (all p < 0.05) and PDC < 85% (all p < 0.05 except DTG/3TC) groups. <b>Conclusion:</b> These results indicate that B/F/TAF may offer greater likelihood of persistence and emphasize the importance of regimen selection to optimize outcomes in treatment-experienced PWH with an MHD/SUD or suboptimal adherence.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250124"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147772870","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":"Switching adult patients with spasticity from onabotulinumtoxinA to abobotulinumtoxinA: a real-world data analysis across three US-based treatment centers.","authors":"Nate Way, Edward Dabrowski, Mitchell Paulin, Martin Taylor, John Madden, Amandeep Mann, Jonathan Bouchard","doi":"10.57264/cer-2025-0181","DOIUrl":"10.57264/cer-2025-0181","url":null,"abstract":"<p><p><b>Aim:</b> To assess patient characteristics, treatment patterns, botulinum toxin type-A (BoNT-A) costs and describe safety in adults with spasticity who switched from onabotulinumtoxinA to abobotulinumtoxinA. <b>Materials & methods:</b> Chart data from three US-based treatment centers was collected in patients aged ≥18 years with upper limb (ULS), lower limb (LLS) or ULS and LLS (ULS + LLS) spasticity. Eligible patients had ≥2 onabotulinumtoxinA treatment cycles before switching to abobotulinumtoxinA; they were followed for three additional abobotulinumtoxinA treatment cycles. A <i>post hoc</i> analysis of estimated drug costs was conducted. <b>Results:</b> Eighty-eight patients (mean age 44.9 years; 62.5% male) switched from onabotulinumtoxinA to abobotulinumtoxinA; in 84 (95.5%), the switch was due to 'medical need/effectiveness not achieved.' Most common spasticity etiologies were cerebral palsy (43.2%) and stroke (25.0%). Fifty-one patients (58.0%) had bilateral spasticity with a mean ± SD of 5.1 ± 2.2 muscles injected at each visit over the 5-injection-cycle treatment period. No adverse events were reported following switching. Mean estimated cost per visit was $2731 with onabotulinumtoxinA and $1452 with abobotulinumtoxinA. <b>Conclusion:</b> In this real-world study, patients with ULS, LLS or ULS + LLS who were switched from onabotulinumtoxinA to abobotulinumtoxinA continued for ≥3 treatment cycles without any reported adverse events. Switching resulted in lower estimated BoNT-A costs.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250181"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147816053","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 meta-analysis: relative clinical efficacy and safety of elafibranor versus seladelpar as second-line treatment for patients with primary biliary cholangitis.","authors":"David Jones, Emily Combe, Harun Knight, Vicki Laskier-Owens, Shijie Ren, Tom Wright, Elaine A Böing, Alex Pashley","doi":"10.57264/cer-2025-0206","DOIUrl":"10.57264/cer-2025-0206","url":null,"abstract":"<p><p><b>Aim:</b> To indirectly compare the efficacy and safety of elafibranor and seladelpar, as second-line treatments for primary biliary cholangitis. <b>Materials & methods:</b> Bayesian network meta-analyses compared data from randomized-controlled studies of elafibranor and seladelpar identified by a systematic literature review up to June 2024: (a) elafibranor (n = 108) versus placebo (n = 53; ELATIVE [NCT04526665]) and (b) seladelpar (n = 128) versus placebo (n = 65; RESPONSE [NCT03301506]). Patients from ELATIVE not meeting the RESPONSE upper limit of normal (ULN) criteria for alkaline phosphatase (ALP) and total bilirubin were excluded (n = 16); summary statistics for ELATIVE were recalculated using the new dataset. Random-effects models assessed the outcomes of cholestasis response (ALP <1.67 × ULN, ALP reduction ≥15% from baseline and total bilirubin ≤ULN), ALP normalization, change from baseline in ALP and pruritus, pruritus as a treatment-emergent adverse event and all-cause discontinuation. <b>Results:</b> Elafibranor-treated patients had greater odds of achieving cholestasis response than placebo- (median odds ratio [95% credible interval]: 84.79 [12.49, 2513.00]) or seladelpar-treated patients (13.02 [1.45, 420.20]), with posterior probabilities ≥99% that odds were higher with elafibranor than seladelpar or placebo. Among patients with ALP ≥350 U/l, the median odds ratio [95% credible interval] of cholestasis response for elafibranor-treated patients versus seladelpar-treated patients was 18.71 [0.65, 10,610.00], with a 95.2% posterior probability that odds were higher with elafibranor than seladelpar. For all other outcomes, there was no strong evidence of a difference between treatments. <b>Conclusion:</b> Bayesian network meta-analyses found strong probabilistic evidence supporting the treatment benefit of elafibranor compared with seladelpar for the achievement of cholestasis response at 52 weeks, while the treatment effect on other outcomes was uncertain. Head-to-head studies are needed to validate results of these indirect comparisons.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250206"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147717009","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":"Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD.","authors":"Cuixia Tian, Shannon Grabich, Aravindhan Veerapandiyan, Rebecca J Scharf, Sourav Santra, Shane Hornibrook, Kerri Drummond, Ihor Sehinovych, Megan Waldrop","doi":"10.57264/cer-2025-0108","DOIUrl":"10.57264/cer-2025-0108","url":null,"abstract":"<p><p><b>Aim:</b> Eteplirsen, golodirsen and casimersen are phosphorodiamidate morpholino oligomers (PMOs) that have received, based on biomarker data, accelerated approval from the US FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients with pathogenic variants amenable to 51, 53 and 45 exon skipping, respectively. The objectives of this study were to describe patient demographic and baseline functional characteristics, safety and treatment continuation in patients with DMD who were treated with a commercially available PMO in the US from the ongoing phase IV, multicenter, prospective, observational EVOLVE study. <b>Patients & methods:</b> Patients who received or initiated treatment with a PMO at the time of study enrollment as prescribed by treating physicians as part of routine care were included. Approximately 300 patients will be enrolled across the three PMOs. <b>Results:</b> As of this 2023 interim data report, 161 patients were enrolled: 126 were treated with eteplirsen (enrollment complete), mean (standard deviation [SD]) age 14.0 (5.51) years; 23 received golodirsen, mean (SD) age 13.3 (4.25) years; and 12 were treated with casimersen, mean (SD) age 16.1 (7.21) years. Mean (SD) total duration of treatment was 6.2 (1.92) years for eteplirsen, 2.4 (0.83) years for golodirsen and 1.7 (0.62) years for casimersen. All PMOs demonstrated favorable safety profiles, with no treatment-emergent serious adverse events related to treatment. Most patients taking eteplirsen (95.2%, n = 120) continued treatment. Among the 85 patients who were ambulatory at treatment initiation, 37 patients lost ambulation, 34 (91.9%) of whom remained on eteplirsen. <b>Conclusion:</b> Consistent with the safety findings from previous clinical trials, eteplirsen, golodirsen and casimersen showed favorable safety profiles in patients with DMD in routine clinical practice. EVOLVE will continue to describe long-term clinical outcomes. Clinical Trial Registration Number: NCT06606340.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250108"},"PeriodicalIF":2.5,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147645235","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":"Efficacy of selpercatinib as a first-line treatment for <i>RET</i>-fusion positive non-small-cell lung cancer: a novel two-stage Bayesian network meta-analysis.","authors":"Jessica E Forsyth, Manoj Khanal, Urpo Kiiskinen, Akkula Jyothi, Rajat Goel, Tarun Puri, Shijie Ren, Michael D Sonksen, Xiaofei Wang, Min-Hua Jen","doi":"10.57264/cer-2026-0006","DOIUrl":"https://doi.org/10.57264/cer-2026-0006","url":null,"abstract":"<p><p><b>Aim:</b> Single-arm trial data is frequently used during the reimbursement of new oncology interventions. Evaluating treatment effects relative to multiple relevant comparators via network meta-analysis (NMA) using data from single-arm trials; however, remains a challenge. This work introduces a two-stage approach to incorporate single-arm trial data into an NMA and applies this to the LIBRETTO-001 (NCT03157128) trial where selpercatinib (a selective rearranged during transfection [<i>RET</i>] inhibitor) was trialed as a treatment for <i>RET</i>-fusion positive, nonsquamous non-small-cell lung cancer in the first line setting. <b>Materials & methods:</b> Using data from KEYNOTE-189 (NCT02578680) and a real-world database, a pseudo comparator arm was constructed by propensity score matching and adjusted via an acceleration factor to account for the prognostic effect of <i>RET</i> status. NMAs were conducted using a Bayesian random-effects model. <b>Results:</b> The hazard ratios of selpercatinib relative to pemetrexed + platinum-based chemotherapy (the most common comparator in the network used) were found to be 0.304 (95% credible interval [CrI] 0.165, 0.553) and 0.368 (95% CrI 0.178, 0.757) for progression-free survival and overall survival, respectively. The validation of the NMA results could be assessed for progression-free survival of selpercatinib versus pembrolizumab + pemetrexed + platinum-based chemotherapy. A good agreement with published results from the Phase III LIBRETTO-431 trial (NCT04194944) was found (0.586 [95% CrI 0.302,1.123] from the NMA vs 0.46 [95% CI 0.31, 0.70] from LIBRETTO-431 the intention to treat [pembrolizumab] population). <b>Conclusion:</b> The two-stage approach to incorporate single-arm trial data within NMAs is readily applicable within health technology assessment. Enabling the earlier assessment of single-arm trials, via pseudo comparator arms, will provide payers with greater confidence in anticipated treatment effects. In light of the joint clinical assessment, incorporation of single-arm trials within NMA facilitates the reporting of predicted treatment effects relative to multiple relevant comparators, which is important when considering the use of interventions for the global market.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260006"},"PeriodicalIF":2.5,"publicationDate":"2026-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147772873","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":"Health care resource use in the management of patients with relapsed/refractory diffuse large B-cell lymphoma - Canadian perspective.","authors":"Kimberly Guinan, Mathieu Pelletier, Monika Ham, Dipti Tankala, Nancy Paul Roc, Adi Klil-Drori, Anthony W Wang, Isabelle Fleury, David MacDonald, Jean Lachaine, Stephane Barakat","doi":"10.57264/cer-2025-0187","DOIUrl":"https://doi.org/10.57264/cer-2025-0187","url":null,"abstract":"<p><p><b>Aim:</b> Epcoritamab, the first subcutaneous (SC) bispecific approved for relapsed/refractory diffuse large B-cell lymphoma (R/R-DLBCL), offers potential advantages in terms of healthcare resource utilization (HCRU) associated with its SC administration. This study aimed to estimate HCRU and associated costs of R/R-DLBCL treatments, to inform health technology assessment agencies, institutional decision makers and healthcare professionals (HCP) from both a Canadian and Quebec perspective. Secondary objectives included using a societal perspective and estimating chair time and HCP time involved in administering treatments. <b>Materials & methods:</b> A 1-year costing analysis was developed comparing epcoritamab to other R/R-DLBCL treatments, including glofitamab, CAR-T cell therapies, chemotherapy, Pola-BR and Tafa-Len. HCRU and associated costs included pretreatment, administration, monitoring, and adverse event management. Acquisition costs of active treatments were excluded. Multiple time horizons were assessed. Model inputs were retrieved from product labels and validated by clinical experts to reflect practice. <b>Results:</b> From the Canadian and Quebec healthcare system perspective, total 1-year HCRU costs ranged from $11,009 to $54,946 and $10,041 to $54,362, respectively. Epcoritamab ranked as the second least costly comparator after chemotherapy, with notable HCRU savings driven by low administration costs of SC injections and adverse event management costs. Epcoritamab ranked similarly from a societal perspective and scenario analysis evaluating a 2-year time-horizon. Epcoritamab had the lowest annual chair time and HCP time, freeing up resources and HCP availability. <b>Conclusion:</b> Considering the highly constrained Canadian healthcare system, SC epcoritamab offers substantial HCRU-related cost saving, chair time savings and HCP time savings when compared with other available treatments, making it an effective, efficient and patient-centric treatment option for R/R-DLBCL in Canada.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e250187"},"PeriodicalIF":2.5,"publicationDate":"2026-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147723011","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 25.","authors":"Paul Arora, Sreeram V Ramagopalan","doi":"10.57264/cer-2026-0073","DOIUrl":"https://doi.org/10.57264/cer-2026-0073","url":null,"abstract":"<p><p>In this update, we discuss the use of real-world data in the Haute Autorité de Santé assessment of economic evaluations, review how real-world evidence is supporting regulatory approvals in multiple myeloma, and consider lessons from the failed EVOKE trials of semaglutide in Alzheimer's disease for the broader application of target trial emulation.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260073"},"PeriodicalIF":2.5,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147690077","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 13.","authors":"Sreeram V Ramagopalan, Annie Jullien Pannelay","doi":"10.57264/cer-2026-0041","DOIUrl":"10.57264/cer-2026-0041","url":null,"abstract":"<p><p>In this update we cover the US-UK Economic Prosperity Deal announced in December 2025, which includes NICE's first major cost-effectiveness threshold increase in over two decades. We also analyze the latest developments in US pharmaceutical pricing policy, including the second cycle of Inflation Reduction Act drug price negotiations and the implementation of the GENEROUS, GUARD and GLOBE Models for Most-Favored-Nation pricing.</p>","PeriodicalId":15539,"journal":{"name":"Journal of comparative effectiveness research","volume":" ","pages":"e260041"},"PeriodicalIF":2.5,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13044808/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147271225","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}