Journal of Medical Economics最新文献

{"title":"Health state utilities associated with weight loss: preferences of people with type 2 diabetes and obesity in Japan.","authors":"Louis S Matza, Katie D Stewart, Josefine Redig, Timothy A Howell, Walter Morris, Rachel S Newson, Alexander Yasui, Jack Ishak, Kristina S Boye","doi":"10.1080/13696998.2024.2316400","DOIUrl":"10.1080/13696998.2024.2316400","url":null,"abstract":"<p><strong>Aims: </strong>Health state utilities associated with weight change are needed for cost-utility analyses (CUAs) examining the value of treatments for type 2 diabetes and obesity. Previous studies have estimated the utility benefits associated with various amounts of weight reduction in the US and Europe, but preferences for weight change in Asian cultures may differ from these published values. The purpose of this study was to estimate utilities associated with reductions in body weight based on preferences of individuals with type 2 diabetes and obesity in Japan.</p><p><strong>Methods: </strong>Health state vignettes represented type 2 diabetes with respondents' own current weight and weight reductions of 2.5%, 5%, 7.5%, 10%, 12.5%, 15%, and 20%. Utilities were elicited in time trade-off interviews with a sample of respondents in Japan with type 2 diabetes and body mass index (BMI) ≥25 kg/m² (the cutoff for obesity in Japan).</p><p><strong>Results: </strong>Analyses were conducted with data from 138 respondents (84.8% male; mean age = 58.0 years; mean BMI = 29.4 kg/m²) from all eight regions of Japan. Utility gains gradually increased with rising percentage of weight reductions ranging from 2.5% to 15%. Weight reductions of 2.5% to 15% resulted in utility increases of 0.013 to 0.048. The health state representing a 20% weight reduction yielded a wide range of preferences (mean utility increase of 0.044). Equations are recommended for estimating utility change based on any percentage of weight reduction (up to 20%) in Japanese people with type 2 diabetes and obesity.</p><p><strong>Limitations: </strong>This study was conducted in a sample with limited representation of patients with BMI >35 kg/m² (<i>n</i> = 13) and relatively few women (<i>n</i> = 21).</p><p><strong>Conclusion: </strong>Results may be used to provide inputs for CUAs examining the value of treatments that are associated with weight loss in patients with type 2 diabetes and obesity in Japan.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"370-380"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139722987","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":"Characterizing the real-world economic burden of metastatic castration-sensitive prostate cancer in the United States.","authors":"Deborah R Kaye, Ibrahim Khilfeh, Erik Muser, Laura Morrison, Frederic Kinkead, Patrick Lefebvre, Dominic Pilon, Daniel George","doi":"10.1080/13696998.2024.2323901","DOIUrl":"10.1080/13696998.2024.2323901","url":null,"abstract":"<p><strong>Aims: </strong>To describe healthcare resource utilization (HRU) and costs of patients with metastatic castration-sensitive prostate cancer (mCSPC).</p><p><strong>Methods: </strong>Linked data from Flatiron Metastatic PC Core Registry and Komodo's Healthcare Map were evaluated (01/2016-12/2021). Patients with chart-confirmed diagnoses for metastatic PC without confirmed castration resistance in Flatiron who initiated androgen deprivation therapy (ADT) monotherapy or advanced therapy for mCSPC in 2017 or later (index date) with a corresponding pharmacy or medical claim in Komodo Health were included. Advanced therapies considered were androgen-receptor signaling inhibitors, chemotherapies, estrogens, immunotherapies, poly ADP-ribose polymerase inhibitors, and radiopharmaceuticals. Patients with <12 months of continuous insurance eligibility before index were excluded. Per-patient-per-month (PPPM) all-cause and PC-related HRU and costs (medical and pharmacy; from a payer's perspective in 2022 $USD) were described in the 12-month baseline period and follow-up period (from the index date to castration resistance, end of continuous insurance eligibility, end of data availability, or death).</p><p><strong>Results: </strong>Of 871 patients included (mean age: 70.6 years), 52% initiated ADT monotherapy as their index treatment without documented advanced therapy use. During baseline, 31% of patients had a PC-related inpatient admission and 94% had a PC-related outpatient visit; mean all-cause costs were $2551 PPPM and PC-related costs were $839 PPPM with $787 PPPM attributable to medical costs. Patients had a mean follow-up of 15 months, during which 38% had a PC-related inpatient admission and 98% had a PC-related outpatient visit; mean all-cause costs were $5950 PPPM with PC-related total costs of $4363 PPPM, including medical costs of $2012 PPPM.</p><p><strong>Limitations: </strong>All analyses were descriptive; statistical testing was not performed. Treatment effectiveness and clinical outcomes were not assessed.</p><p><strong>Conclusion: </strong>This real-world study demonstrated a significant economic burden in mCSPC patients, and a propensity to use ADT monotherapy in clinical practice despite the availability and guideline recommendations of advanced life-prolonging therapies.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"381-391"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139990264","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":"Correction.","authors":"","doi":"10.1080/13696998.2024.2402139","DOIUrl":"https://doi.org/10.1080/13696998.2024.2402139","url":null,"abstract":"","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"27 sup3","pages":"34-35"},"PeriodicalIF":2.9,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142289326","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":"Societal costs and quality of life associated with arginase 1 deficiency in a European setting - a multinational, cross-sectional survey.","authors":"Sara Olofsson, Sofia Löfvendahl, Julia Widén, Mattias Rudebeck, Peter Lindgren, Karolina M Stepien, Jean-Baptiste Arnoux, Maria Luz Couce Pico, Elisa Leão Teles, Lena Jacobson","doi":"10.1080/13696998.2024.2400856","DOIUrl":"10.1080/13696998.2024.2400856","url":null,"abstract":"<p><strong>Background and aims: </strong>Arginase 1 deficiency (ARG1-D) is a ultrarare disease with manifestations that cause mobility and cognitive impairment that progress over time and may lead to early mortality. Diseases such as ARG1-D have a major impact also outside of the health care sector and the aim of this study was to estimate the current burden of disease associated with ARG1-D from a societal perspective.</p><p><strong>Methods: </strong>The study was performed as a web-based survey of patients with ARG1-D and their caregivers in four European countries (France, Portugal, Spain, United Kingdom). The survey was distributed at participating clinics and included questions on e.g. symptoms (including the Gross Motor Function Classification System, GMFCS, and cognitive impairment), health care use, medication, ability to work, caregiving, and impact on health-related quality-of-life (HRQoL) using the EQ-5D-5L.</p><p><strong>Results: </strong>The estimated total mean societal cost per patient and year was £63,775 (SD: £49,944). The cost varied significantly with both mobility impairment (from £49,809 for GMFCS level 1 to £103,639 for GMFCS levels 3-5) and cognitive impairment (from £43,860 for mild level to £99,162 for severe level). The mean utility score on the EQ-5D-5L for patients was 0.498 (SD: 0.352). The utility score also varied significantly with both mobility impairment (from 0.783 for GMFCS level 1 to 0.153 for GMFCS level 3-5) and cognitive impairment (from 0.738 for mild level to 0.364 for severe level).</p><p><strong>Conclusions: </strong>Similar to other studies of rare diseases, the study is based on a limited number of observations. However, the sample appear to be reasonably representative when comparing to previous studies of ARG1-D. This study shows that ARG1-D is associated with a high societal cost and significant impact on HRQoL. Earlier diagnosis and better treatment options that can postpone or withhold progression may therefore have a potential for improved HRQoL and savings for the patient, caregiver, and society.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"1146-1156"},"PeriodicalIF":2.9,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142125961","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":"Promoting equality in utilization of basic public health services in China: the role of the family doctor contract service.","authors":"Sha Lai, Yawei Huang, Xiaolong Zhang, Zechen Wang, Junfei Feng, Zhongliang Zhou, Chi Shen, Li Lu","doi":"10.1080/13696998.2024.2421115","DOIUrl":"10.1080/13696998.2024.2421115","url":null,"abstract":"<p><strong>Background: </strong>The Family Doctor Contract Service (FDCS) system is a service model for primary care reform launched in 2016 to offer families and individuals active and continuous health care by a team of family doctors within primary care institutions in China.</p><p><strong>Objectives: </strong>This study aimed to estimate socioeconomic-related inequalities in the utilization of basic public health services, and to identify the contribution of FDCS to promoting equality.</p><p><strong>Methods: </strong>Data for the study were collected from a 2023 cross-sectional household health survey in western China, involving 39,456 participants. The concentration index (C) was employed for analyzing the extent of socioeconomic-related inequalities in the utilization of basic public health services and the coarsened exact matching technique was employed for sensitivity analysis in order to reduce selection bias.</p><p><strong>Results: </strong>Our results indicated pro-poor inequalities in the utilization of health records (C = -0.046), free health check-ups (C = -0.009), and follow-ups for hypertension (C = -0.051). Additionally, a more equitable distribution across the economic spectrum was observed within the FDCS group (people who voluntarily contracted for services) compared to the non-FDCS group. The FDCS demonstrated more favorable positive impacts among individuals with higher (quintiles 60-80%) and the highest (top 20%) socioeconomic status. The FDCS contributed 83.94%, 59.24%, and 36.92% to pro-poor inequalities in the utilization of three basic public health services. These contributions reflected the positive impact of the FDCS on utilization.</p><p><strong>Conclusions: </strong>Government policy and service delivery models require a paradigm shift to promote a stronger primary healthcare approach to practice, as evidenced by the effectiveness of the FDCS in promoting equality.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"1444-1455"},"PeriodicalIF":2.9,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142502109","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 of Ebola epidemics on the daily operation of existing systems in Eastern Democratic Republic of the Congo: a brief review.","authors":"Daniel Mukadi-Bamuleka, Antoine Nkuba-Ndaye, Placide Mbala-Kingebeni, Steve Ahuka-Mundeke, Jean-Jacques Muyembe-Tamfum","doi":"10.1080/13696998.2024.2305009","DOIUrl":"10.1080/13696998.2024.2305009","url":null,"abstract":"<p><strong>Aims: </strong>to provide insights into the recent Ebola virus disease (EVD) outbreaks on different aspects of daily life in the Democratic Republic of the Congo and propose possible solutions.</p><p><strong>Methods: </strong>We collected information regarding the effects of EVD outbreaks on existing systems in the eastern part of the Democratic Republic of the Congo (DRC). We searched the PubMed database using the terms \"impact effect Ebola outbreak system\", \"Management Ebola Poor Resources Settings\", \"Health Economic Challenges Ebola\" and \"Economic impact Ebola systems.\" Only studies focusing on epidemiology, diagnostics, sequencing, vaccination, therapeutics, ecology, work force, governance, healthcare provision and health system, and social, political, and economic aspects were considered. The search included the electronic archives of EVD outbreak reports from government and partners.</p><p><strong>Results: </strong>EVD outbreaks negatively impacts the functions of countries. The disruption in activities is proportional to the magnitude of the epidemic and slows down the transport of goods, decreases the region's tourist appeal, and increases 'brain drain'. Most low- and medium-income countries, such as the DRC, do not have a long-term holistic emergency plan for unexpected situations or sufficient resources to adequately implement countermeasures against EVD outbreaks. Although the DRC has acquired sufficient expertise in diagnostics, genomic sequencing, administration of vaccines and therapeutics, clinical trials, and research activities, deployment, operation, and maintenance of these expertise and associated tools remains a concern.</p><p><strong>Limitations: </strong>Despite the data search extension, additional reports addressing issues related to social aspects of EVD outbreaks in DRC were not retrieved.</p><p><strong>Conclusion: </strong>National leadership has not yet taken the lead in strategic, operational, or financial aspects. Therefore, national leaders should double their efforts and awareness to encourage local fundraising, sufficient budget al.location, infrastructure construction, equipment provision, and staff training, to effectively support a holistic approach in response to outbreaks, providing effective results, and all types of research activities.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"184-192"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139491396","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 of pembrolizumab plus lenvatinib compared with chemotherapy for treating previously treated advanced endometrial cancer in Sweden.","authors":"Lewis Ralph, Kate Young, Navneet Upadhyay, Vimalanand Shrikant Prabhu, Christina Ljungcrantz, Rachid Massaad, Ruifeng Xu, Anna Giertz, Adil Merchant, Robert Orlowski, Linda Duska","doi":"10.1080/13696998.2024.2329022","DOIUrl":"10.1080/13696998.2024.2329022","url":null,"abstract":"<p><strong>Objective: </strong>Pembrolizumab plus lenvatinib was recently approved for the treatment of advanced or recurrent endometrial carcinoma in women with disease progression on or following prior treatment with a platinum‑containing therapy in any setting, and who are not candidates for curative surgery or radiation (KEYNOTE-775/Study-309; NCT03517449). The objective was to assess the cost effectiveness of pembrolizumab plus lenvatinib compared with chemotherapy from a Swedish healthcare perspective.</p><p><strong>Materials and methods: </strong>A lifetime partitioned-survival model with three health states (progression free, progressed disease, death) was constructed. Chemotherapy was represented by paclitaxel or doxorubicin. Overall survival, progression-free survival, time on treatment, and utility data were obtained from KEYNOTE-775 (database lock: March 1, 2022). Costs (in 2020 Swedish Krona [SEK]) included drug acquisition and administration, health state, end of life, adverse event management, subsequent treatment, and societal (scenario analysis). Outcomes were calculated as quality-adjusted life-years (QALY) and life-years. Model results were presented as incremental cost-effectiveness ratios for all-comers, patients with proficient mismatch repair tumors, and deficient mismatch repair tumors. Deterministic and probabilistic sensitivity analyses were conducted.</p><p><strong>Results: </strong>Pembrolizumab plus lenvatinib is a cost-effective treatment when compared with chemotherapy, with estimated deterministic and probabilistic incremental cost-effectiveness ratios of SEK 795,712 and 819,757 per QALY gained. Pembrolizumab plus lenvatinib was associated with a large incremental QALY and life-year gain per person versus chemotherapy over the model time horizon (1.49 and 1.76).</p><p><strong>Limitations: </strong>Time-to-event data were incomplete and semiparametric and parametric curves were utilized for lifetime extrapolation. Willingness-to-pay thresholds, costs, and utility weights vary by country, which would vary the treatment's cost effectiveness in different countries.</p><p><strong>Conclusions: </strong>This partitioned survival analysis suggests that pembrolizumab plus lenvatinib is cost effective compared with chemotherapy in Sweden for women with advanced or recurrent endometrial carcinoma following previous systemic therapy. Results were robust to mismatch repair status and to changes in parameters/assumptions.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"483-491"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140101739","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 adherence of macitentan versus ambrisentan and bosentan in Australian patients with pulmonary arterial hypertension: a retrospective real-world database study.","authors":"Edmund Lau, Eugene Kotlyar, Yogeshwar Makanji, Dae Young Yu, Jin Yu Tan, Jeremy Casorso, Mahsa H Kouhkamari, Sooyeol Lim, David Bin-Chia Wu, Paul Bloomfield","doi":"10.1080/13696998.2024.2328483","DOIUrl":"10.1080/13696998.2024.2328483","url":null,"abstract":"<p><strong>Aim: </strong>Bosentan, ambrisentan, and macitentan are endothelin receptor antagonists (ERAs), currently available in Australia for treatment of pulmonary arterial hypertension (PAH). This study assessed the comparative adherence of these ERAs for PAH in Australian patients.</p><p><strong>Methods: </strong>This retrospective, observational study used data for adults with PAH from the Services Australia 10% Pharmaceuticals Benefits Scheme (PBS) dataset (01/2006-10/2020). The primary outcome was treatment adherence (i.e. receiving ≥80% of ERA doses over 12 months). Secondary outcomes were time to treatment change (add-on or switch) and overall survival.</p><p><strong>Results: </strong>The study included 436 patients who took bosentan (<i>n</i> = 200), ambrisentan (<i>n</i> = 69), or macitentan (<i>n</i> = 167). Treatment adherence was significantly greater in patients who received macitentan (65.3%) versus ambrisentan (56.5%) and bosentan (58.0%), with odds ratios (ORs; 95% CI) of 0.51 (0.30-0.88; <i>p</i> = 0.016) for bosentan versus macitentan and 0.48 (0.24-0.96; <i>p</i> = 0.037) for ambrisentan versus macitentan. The median time to treatment change was 47.2 and 43.4 months for bosentan and ambrisentan, respectively (not calculated for macitentan because of insufficient duration of data).</p><p><strong>Limitations and conclusions: </strong>Real-world data for Australian patients with PAH showed that treatment adherence for ERAs was suboptimal. Adherence was higher for macitentan compared with ambrisentan and bosentan.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"596-604"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140131743","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":"Quantifying preferences for urea cycle disorder treatments using a discrete choice experiment.","authors":"Josiah Edelblut, Jeffrey R Skaar, John Hilton, Matthew Seibt, Kyle Martin, Nandini Hadker, Adrian Quartel, Robert D Steiner","doi":"10.1080/13696998.2024.2330846","DOIUrl":"10.1080/13696998.2024.2330846","url":null,"abstract":"<p><strong>Aims: </strong>Urea cycle disorders (UCDs) can cause ammonia accumulation and central nervous system toxicity. Nitrogen-binding medications can be efficacious, but certain attributes may negatively impact adherence. This study sought to quantify the administration-related attributes influencing overall prescription selection and patient adherence.</p><p><strong>Methods: </strong>A web-based, quantitative survey including discrete choice experiment (DCE) methodology captured responses from health care providers for patients with UCDs. A series of hypothetical treatment profile sets with attributes such as route of administration, taste/odor, preparation instructions, packaging, dose measurement, and weight use restrictions were presented. From 16 sets of 3 hypothetical product profiles, respondents evaluated attributes most preferred for prescription selection or patient adherence. Attributes assumed a higher overall preference if relative importance (RI) scores were >16.67% (the value if all attributes were of equal importance). Preference weight scores were assessed. A nine-point Likert scale assessed respondent attitudes, such as satisfaction.</p><p><strong>Results: </strong>A total of 51 respondents completed the survey. Respondents reported dissatisfaction with current treatments (mean [SD] = 5.4 [1.7]). For prescription selection, four attributes achieved RI >16.67%: taste/odor (24%), weight restrictions (21%), preparation instructions (18%), and route of administration (17%). For adherence, three attributes related to administration achieved RI >16.67%: taste/odor (28%), preparation instructions (21%), and route of administration (17%). Preference weights for \"taste/odor masked\" were higher than \"not taste/odor masked\" for prescription selection (mean [SD]; 1.52 [1.10] vs -1.52 [1.10]) and treatment adherence (73.8 [55.2] vs -73.8 [55.2]).</p><p><strong>Limitations: </strong>This study contained a relatively small sample size. Survey respondent selection, the use of hypothetical product profiles, and exclusion of non-pharmacologic treatment options could have contributed to potential biases.</p><p><strong>Conclusions: </strong>Among attributes tested, taste/odor was the most important attribute influencing overall preference for both prescribing and patient adherence, with taste/odor masking preferred. Optimizing nitrogen-binding medications through masking taste/odor may support improved patient adherence and outcomes in UCDs.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"506-517"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140140375","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 analysis of mosunetuzumab for treatment of relapsed or refractory follicular lymphoma after two or more lines of systemic therapy in the United States.","authors":"Matthew Matasar, Javier Sanchez Alvarez, Hélène Parisé, Eric Zuk, Danilo Di Maio, Sheila Shapouri, Eunice Kim, Shih-Wen Lin","doi":"10.1080/13696998.2024.2352820","DOIUrl":"10.1080/13696998.2024.2352820","url":null,"abstract":"<p><strong>Aims: </strong>Mosunetuzumab has received accelerated approval by the US Food and Drug Administration for adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy. We evaluated the cost-effectiveness of mosunetuzumab for the treatment of R/R FL from a US private payer perspective.</p><p><strong>Materials and methods: </strong>A partitioned survival model simulated lifetime costs and outcomes of mosunetuzumab against seven comparators: axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), tazemetostat (taz, EZH2 wild-type only), rituximab plus lenalidomide (R-Len) or bendamustine (R-Benda), obinutuzumab plus bendamustine (O-Benda), and a retrospective real-world cohort (RW) based on current patterns of care derived from US electronic health records (Flatiron Health). Efficacy data for mosunetuzumab were from the pivotal Phase II GO29781 trial (NCT02500407). Relative treatment efficacy was estimated from indirect treatment comparisons (ITCs). Costs included were related to treatment, adverse events, routine care, and terminal care. Except for drug costs (March 2023), all costs were inflated to 2022 US dollars. Costs and quality-adjusted life-years (QALYs) were used to calculate incremental cost-effectiveness ratios (ICERs). Net monetary benefit (NMB) was calculated using a willingness-to-pay (WTP) threshold of $150,000/QALY.</p><p><strong>Results: </strong>Mosunetuzumab dominated taz, tisa-cel, and axi-cel with greater QALYs and lower costs. Mosunetuzumab was projected to be cost-effective against R-Benda, O-Benda, and RW with ICERs of $78,607, $42,731, and $21,434, respectively. Mosunetuzumab incurred lower costs but lower QALYs vs. R-Len. NMBs showed that mosunetuzumab was cost-effective against comparators except R-Len.</p><p><strong>Limitations: </strong>Without head-to-head comparative data, the model had to rely on ITCs, some of which were affected by residual bias. Model inputs were obtained from multiple sources. Extensive sensitivity analyses assessed the importance of these uncertainties.</p><p><strong>Conclusion: </strong>Mosunetuzumab is estimated to be cost-effective compared with approved regimens except R-Len for the treatment of adults with R/R FL.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"766-776"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140858551","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}