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Aim: Dostarlimab plus carboplatin-paclitaxel (CP) significantly increased progression-free survival in patients with primary advanced or recurrent endometrial cancer (pA/rEC) vs CP alone in the RUBY trial (NCT03981796). This analysis estimated the per-member-per-month (PMPM) costs of introducing dostarlimab + CP as a treatment alternative from a third-party US payer perspective. Materials and methods: A budget impact model was developed to estimate the costs of introducing dostarlimab + CP into commercial and Medicare health plans over a 3-year time horizon (2023–2025). Costs were sourced from relevant literature and US-specific databases and were calculated using epidemiology data, clinical inputs, treatment costs, and market share estimates. Clinical inputs were sourced from primary clinical trials for each respective treatment (i.e. dostarlimab + CP, CP, pembrolizumab, pembrolizumab plus lenvatinib, bevacizumab + CP, and pembrolizumab + CP). Current and future market shares assumed dostarlimab + CP reduced the market share of CP only. Analyses were performed in mismatch repair–deficient/microsatellite instability–high (dMMR/MSI-H) and overall populations using a US 2023 cost year. Results: For a commercial plan, the model estimated (dMMR/MSI-H and overall populations) that 7 and 26 patients would be treated with dostarlimab + CP, respectively; average annual budget impacts per patient treated were $118,257 and $116,094; average budget impacts per patient treated per month (PPPM) were $9,855 and $9,675; average budget impacts PMPM were $0.02 and $0.06. For a Medicare plan, the model estimated that 28 and 93 patients, respectively, would be treated with dostarlimab + CP. Average annual budget impacts per patient treated and PPPM were the same as those for the commercial plan in both populations; average budget impacts PMPM were $0.07 and $0.22, respectively. Conclusions: Introducing dostarlimab + CP as a first-line treatment for patients with pA/rEC results in minimal budget impact PMPM from a US third-party payer's perspective. Together with the efficacy and safety results from RUBY, these results support the use of dostarlimab + CP as a treatment option. PLAIN LANGUAGE SUMMARY: Dostarlimab with carboplatin–paclitaxel is a recently approved treatment for newly diagnosed advanced or recurrent endometrial cancer. This analysis was done to estimate the added costs that US commercial and Medicare health plans would have over 3 years if this treatment was covered. This analysis found that the budget increase for covering dostarlimab with carboplatin–paclitaxel was small ($0.02–$0.06 per commercial plan member per month; $0.07–$0.22 per Medicare plan member per month). [ABSTRACT FROM AUTHOR]

OBJECTIVES: To determine the lifetime cost-effectiveness of nivolumab vs docetaxel in advanced squamous and nonsquamous non--small cell lung cancer (NSCLC) following platinum-based chemotherapy from a US payer perspective. STUDY DESIGN: Trial- and cohort-based cost-effectiveness analyses. METHODS: The analyses used partitioned survival models with 3 mutually exclusive health states: progression free, progressed disease, and death. The mean starting age was 61 years. Clinical parameters were derived from the 2 registrational, randomized, phase 3 trials with a minimum follow-up of 5 years. Costs were derived from published literature. The primary outcomes were quality-adjusted life-years (QALYs), life-years gained (LYG), and incremental cost-effectiveness ratios (ICERs). Costs and outcomes were discounted at 3% per annum. Uncertainty was examined using univariate and probabilistic sensitivity analyses. RESULTS: In patients with squamous NSCLC, the use of nivolumab improved life-years (LYs) and QALYs by 1.23 and 0.99, respectively, compared with docetaxel. Costs were increased by $99,677, resulting in ICERs of $100,776 per QALY and $81,294 per LYG. In patients with nonsquamous NSCLC, nivolumab increased LYs and QALYs by 0.99 and 0.80, respectively. Costs were increased by $94,174, resulting in ICERs of $117,739 per QALY and $94,849 per LYG. ICERs were most sensitive to the discount rates applied to costs and outcomes. At a willingness-to-pay threshold of $150,000, nivolumab had probabilities of 91% and 99% of being cost-effective in patients with squamous and nonsquamous NSCLC, respectively. CONCLUSIONS: Nivolumab is likely to be cost-effective for the treatment of patients with advanced NSCLC following platinum-based chemotherapy in the United States. [ABSTRACT FROM AUTHOR]

Abstract Background Although community pharmacists in the United Kingdom are expected to assess elderly patients' needs for additional support in managing their medicines, there is limited data on potentially useful assessment tools. We sought to evaluate a 13-item assessment instrument among community dwelling elderly patients, 65 years and above. The instrument is composed of a cognitive risk sub-scale of 6 items and a physical risk sub-scale of 7 items. Findings The instrument was administered to elderly patients in a survey performed in a community to the west of Glasgow, Scotland. The survey recruited 37 participants, 31 from 4 community pharmacies and 6 patients whose medication management tasks were managed by the West Glasgow Community Health and Care Partnership (managed patients). Community pharmacists independently rated 29 of the 37 participants' comprehension of, and dexterity in handling their medicines. We assessed scale reliability, convergent validity and criterion validity. In sub-analyses, we assessed differences in scores between the managed patients and those recruited from the community pharmacies, and between multi-compartment compliance aid users and non-users. The instrument showed satisfactory internal consistency (Cronbach's alpha of 0.792 for 13-item scale). There was significant strong negative correlation between the cognitive risk sub-scores and community pharmacists' assessment of comprehension (ρ = -0.546, p = 0.0038); and physical risk sub-scores and community pharmacists' assessment of dexterity (ρ = -0.491, p = 0.0093). The Area Under the Receiver Operator Characteristic Curve (AUC ± SE; 95%CI) showed that the instrument had good discriminatory capacity (0.86 ± 0.07; 0.68, 0.96). The best cut-off (sensitivity, specificity) was ≥4 (65%, 100%). In the sub-analyses, managed patients had significantly higher cognitive risk sub-scores (6.5 versus 4.0, p = 0.0461) compared to non-managed patients. There was a significant difference in total risk score (4 versus 2, p = 0.0135) and cognitive risk sub-score (4 versus 1.5, p = 0.0029) between users and non-users of multi-compartment compliance aids. Conclusions This instrument shows potential for use in identifying elderly patients who may have problems managing their own medicines in the community setting. However, more robust validity and reliability assessments are needed prior to introduction of the tool into routine practice.

OBJECTIVES: Recent advances have created options for first-line (1L) treatment of advanced/metastatic non--small cell lung cancer (aNSCLC). The study objectives were to describe the utilization of 3 classes of 1L treatment--chemotherapy (CT), immunotherapy (IO), and chemoimmunotherapy (IO+CT)--and the total, third-party payer, direct health care costs. STUDY DESIGN: Retrospective, administrative claims database analysis of patients with aNSCLC who initiated 1L treatment between January 1, 2017, and May 31, 2019, with IO, CT, or IO+CT. METHODS: Microcosting enumerated health care resource utilization, including antineoplastic drug costs, using standardized costs. Generalized linear models estimated per-patient per-month (PPPM) costs during 1L treatment, and adjusted cost differences in 1L among treatment cohorts were calculated using recycled predictions. RESULTS: A total of 1317 IO-, 5315 CT-, and 1522 IO+CTtreated patients were identified. Utilization of CT declined from 72.3% to 47.6% between 2017 and 2019, replaced by use of IO+CT, which increased from 1.8% to 29.8%. Total PPPM costs in 1L were highest with IO+CT at $32,436, compared with $19,000 and $17,763 in the CT and IO cohorts, respectively. Adjusted analyses showed that PPPM costs were $13,933 (95% CI, $11,760-$16,105) higher in the IO+CT vs IO cohort (P < .001) and IO costs were $1024 (95% CI, $67-$1980) lower than CT (P = .04). CONCLUSIONS: IO+CT accounts for almost one-third of 1L aNSCLC treatment modalities, coinciding with a reduction in treatment with CT. Costs for patients treated with IO were lower than those for patients treated with both IO+CT and CT alone, driven primarily by antineoplastic drug and associated medical costs. [ABSTRACT FROM AUTHOR]

Highlights from the article: Our analysis did conclude a gain of 1.3 quality-adjusted life-years (QALYs) for VN compared with SoC as stated by Buessing et al., and they conclude that this implies an 8% improvement in the patient's quality of life. Nevertheless, we did complete a scenario analysis around this assumption, available in the publicly available ICER report, which resulted in higher health gains and lower costs for VN patients relative to the base-case scenario. Future studies of long-term health outcomes, quality of life, and indirect costs for patients with RPE65-mediated inherited retinal disease would improve our understanding of the economic and health impacts of new treatments.

Objective: Dostarlimab in combination with carboplatin-paclitaxel (CP) improves progression-free survival in patients with primary advanced or recurrent endometrial cancer (pA/rEC), including in patients whose cancer is mismatch repair-deficient (dMMR) or microsatellite instability-high (MSI-H). This study examined the cost-effectiveness of dostarlimab plus CP as a first-line treatment in the dMMR/MSI-H and overall populations., Methods: A partitioned survival model with three mutually exclusive health states (progression-free disease, progressed disease, death) was developed using a US base case and a third-party payer perspective. Clinical data were from the RUBY trial and published sources. Costs were from US databases. The primary outcomes were life-years (LYs), quality-adjusted life-years (QALYs), incremental costs, and incremental cost-effectiveness ratios (ICERs). One-way and probabilistic sensitivity analyses were also performed., Results: In the dMMR/MSI-H population, the model predicted gains of 6.9 LYs and 5.4 QALYs with dostarlimab plus CP compared with CP; costs were $307,696 higher with dostarlimab plus CP, resulting in an ICER of $57,151 per QALY gained. In the overall population, gains of 2.0 LYs and 1.5 QALYs were predicted with dostarlimab plus CP compared with CP; costs were $215,876 higher, resulting in an ICER of $143,783 per QALY gained. ICERs were most sensitive to the overall survival hazard ratio. At a willingness-to-pay threshold of $150,000, dostarlimab plus CP had cost-effectiveness probabilities of 100 % and 53.7 % in the dMMR/MSI-H and overall populations, respectively., Conclusions: Dostarlimab plus CP is cost-effective as a treatment for the dMMR/MSI-H and overall populations of US patients with pA/rEC., Competing Interests: Declaration of competing interest RLC discloses grants or contracts from AstraZeneca, Clovis, Genelux, Genmab, Merck, Immunogen, Roche/Genentech, and Karyopharm; royalties or licenses from Up-To-Date; consultancy for Agenus, Alkermes, AstraZeneca, Clovis, Deciphera, Genelux, Genmab, GSK, Immunogen, OncoQuest, Onxerna, Regeneron, Karyopharm, Roche/Genentech, Novocure, Merck, and Abbvie; payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from AstraZeneca, Clovis, Roche/Genentech, and Merck; participation on data safety monitoring or advisory boards from NRG Oncology and Eisai/BMS; and leadership or fiduciary roles in Onxeo/Valerio, Vaniam Group, GOG Foundation, International Gynecologic Cancer Society, OCNA and Ovarcome. SJL discloses employment with GSK, GSK funding for meetings/travel and ownership of GSK stock. QS discloses employment with GSK and ownership of GSK stock. LW and MB disclose consultancy for GSK and employment with FIECON, which received consultancy fees from GSK. CM discloses grants or contracts from Deciphera, Astellas Pharma, Seagen, Genmab, EMD Serono, Merck, Regeneron, Moderna, Astra Zeneca, AvengeBio, Zentalis, GSK, Immunogen, Elucida, Roche/Genentech, Pfizer, Laekna, Artios, Volatra, and AADI. This study was funded by GSK; editorial and writing support was provided by Nucleus Global, which received funding from GSK., (Copyright © 2024. Published by Elsevier Inc.)

Background: Methylene blue and visible light treatment and quarantine are two methods used to reduce adverse events, mostly infections, associated with the transfusion of fresh-frozen plasma. The objective of this study was to estimate and compare the budget impact and cost-utility of these two methods from a payer's perspective., Materials and Methods: A budget impact and cost-utility model simulating the risks of hepatitis B virus, hepatitis C virus, cytomegalovirus, a West Nile virus-like infection, allergic reactions and febrile non-haemolytic transfusion reactions achieved using plasma treated with methylene blue and visible light (MBP) and quarantine plasma (QP) was constructed for Spain. QP costs were estimated using data from one blood centre in Spain and published literature. The costs of producing fresh-frozen plasma from whole blood, apheresis plasma, and multicomponent apheresis, and separately for passive and active methods of donor recall for QP were included. Costs and outcomes over a 5-year and lifetime time horizon were estimated., Results: Compared to passive QP, MBP led to a net increase of € 850,352, and compared to active QP, MBP led to a net saving of € 5,890,425 over a 5-year period. Compared to passive QP, MBP increased the cost of fresh-frozen plasma per patient by € 7.21 and had an incremental cost-utility ratio of € 705,126 per quality-adjusted life-year. Compared to active QP, MBP reduced cost by € 50.46 per patient and was more effective., Discussion: Plasma collection method and quarantine approach had the strongest influence on the budget impact and cost-utility of MBP. If QP relies on plasma from whole blood collection and passive quarantine, it is less costly than MBP. However, MPB was estimated to be more effective than QP in all analyses.