Journal of Health Economics and Outcomes Research最新文献

{"title":"Central Nervous System-related Conditions and Associated Healthcare Resource Use Among Japanese nmCRPC Patients Based on Retrospective Claims Data.","authors":"Dianne A Ledesma, Jonathan L Chua, Susan S H Tang, Xiu W Lim","doi":"10.36469/001c.87550","DOIUrl":"10.36469/001c.87550","url":null,"abstract":"<p><p><b>Background:</b> Japanese patients with prostate cancer are typically treated with primary androgen deprivation therapy (ADT), most commonly administered as a combination of a luteinizing hormone-releasing hormone (LHRH) agonist and an antiandrogen (AA). Since LHRH agonists and AA therapy can be maintained for several years, the long-term effects of these treatments on patients must be carefully considered, including the risk of concomitant central nervous system (CNS) conditions which could affect treatment choices. <b>Objective:</b> To describe CNS-related concomitant conditions during ADT and/or AA treatment and the subsequent healthcare resource utilization in Japanese nonmetastatic castration-resistant prostate cancer (nmCRPC) patients. <b>Methods:</b> Patients diagnosed with nmCRPC and CNS-related conditions while on ADT and/or AA therapy between April 2009 and August 2017 were retrospectively followed up for a maximum of 2 years using a claims database. <b>Results:</b> A total of 455 patients (average age, 78.5 years), were included. The 3 most common concomitant CNS-related conditions were pain (~60% of events), insomnia (~30%), and headache (2%-3%). The frequency of CNS-related conditions in these patients increased approximately threefold after starting AA therapy (before, 969 events; after, 2802). On average, a patient had 10 episodes of concomitant CNS-related conditions in a year. Medical costs did not significantly increase due to CNS-related conditions. <b>Discussion:</b> The most frequently reported CNS-related conditions were pain, insomnia, and headaches. Furthermore, more concomitant CNS-related conditions 1 year after CRPC diagnosis and 1 year after starting AA treatment were recorded. <b>Conclusion:</b> Patients with nmCRPC experience an increase in the frequency of concomitant CNS-related conditions, including pain, insomnia, and headaches, after CRPC diagnosis or starting AA treatment. Future research should explore the causes of this increased frequency.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 2","pages":"91-99"},"PeriodicalIF":0.0,"publicationDate":"2023-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10621534/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71482387","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Value-based Reimbursement as a Mechanism to Achieve Social and Financial Impact in the Healthcare System.","authors":"Ana Paula Beck de Silva Etges, Harry H Liu, Porter Jones, Carisi A Polanczyk","doi":"10.36469/001c.89151","DOIUrl":"10.36469/001c.89151","url":null,"abstract":"<p><p>Value-based reimbursement strategies have been considered in the continuous search for establishing a sustainable healthcare system. For models that have been already implemented, success is demonstrated according to specific details of the patients' consumption profile based on their clinical condition and the risk balance among all the stakeholders. From fee-for-service to value-based bundled payment strategies, the manner in which accurate patient-level cost and outcome information are used varies, resulting in different risk agreements between stakeholders. A thorough understanding of value-based reimbursement agreements that views such agreements as a mechanism for risk management is critical to the task of ensuring that the healthcare system generates social impacts while ensuring financial sustainability. This perspective article focuses on a critical analysis of the impact of value-based reimbursement strategies on the healthcare system from a social and financial perspective. A critical analysis of the literature about value-based reimbursement was used to identify how these strategies impact healthcare systems. The literature analysis was followed by the conceptual description of value-based reimbursement agreements as mechanisms for achieving social and financial impacts on the healthcare system. There is no single successful path toward payment reform. Payment reform is used as a strategy to re-engineer the way in which the system is organized to provide care to patients, and its successful implementation leads to cultural, social, and financial changes. Stakeholders have reached consensus regarding the claim that the use of value reimbursement strategies and business models could increase efficiency and generate social impact by reducing healthcare inequity and improving population health. However, the successful implementation of such new strategies involves financial and social risks that require better management by all the stakeholders. The use of cutting-edge technologies are essential advances to manage these risks and must be paired with strong leadership focusing on the directive to improve population health and, consequently, value. Payment reform is used as a mechanism to re-engineer how the system is organized to deliver care to patients, and its successful implementation is expected to result in social and financial modifications to the healthcare system.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 2","pages":"100-103"},"PeriodicalIF":2.3,"publicationDate":"2023-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10621730/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71482396","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment Patterns and Outcomes Among Patients With Advanced or Recurrent Endometrial Cancer Initiating First-Line Therapy in the United States.","authors":"Robert L Coleman, Jamie Garside, Jean Hurteau, Joehl Nguyen, Monica Kobayashi","doi":"10.36469/001c.87853","DOIUrl":"10.36469/001c.87853","url":null,"abstract":"<p><p><b>Background:</b> Patients with advanced or recurrent endometrial cancer (EC) typically have limited treatment options and poor long-term survival outcomes following first-line therapy. Real-world treatment patterns and survival outcomes data are limited for patients in this setting. <b>Objectives:</b> The objective of this retrospective study was to describe real-world demographics, clinical characteristics, treatment patterns, and overall survival among patients in the United States with primary advanced or recurrent EC who initiated at least 1 line of therapy (LOT). <b>Methods:</b> Patients with a diagnosis of primary advanced or recurrent EC in a real-world database from January 1, 2013, to July 31, 2021, were included. The date for inclusion was the date of EC diagnosis documentation; patients were indexed for treatment patterns and outcomes at the start of the first LOT and at the start of each subsequent LOT they initiated. Data were stratified by subgroups of patients who had mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) tumors. <b>Results:</b> A total of 1961 patients who received at least 1 LOT were included. Most patients in this cohort, and the dMMR/MSI-H subgroup, received a platinum combination as first-line treatment, with carboplatin-paclitaxel being the most common regimen. Only 53% of patients who received first-line treatment subsequently received second-line therapy. Of the patients who received at least 1 LOT, use of immunotherapy in the second-line setting was more common in the dMMR/MSI-H subgroup. Median overall survival ranged from 14.1 to 31.8 months across the 5 most frequently used first-line treatment regimens in the ≥1 LOT cohort and became shorter with each subsequent LOT. <b>Discussion:</b> The use of platinum-based chemotherapy for first-line treatment of advanced or recurrent EC predominates in the real-world setting, despite the poor long-term survival outcomes associated with most of these regimens. <b>Conclusions:</b> Patients with recurrent/advanced EC have a poor prognosis, highlighting the need for therapies with more durable benefits.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 2","pages":"82-90"},"PeriodicalIF":0.0,"publicationDate":"2023-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10613433/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71412536","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment Patterns and Outcomes Among Patients With Advanced or Recurrent Endometrial Cancer Initiating First-Line Therapy in the United States","authors":"Robert Coleman, Jamie Garside, Jean Hurteau, Joehl Nguyen, Monica Kobayashi","doi":"10.36469/jheor.2023.87853","DOIUrl":"https://doi.org/10.36469/jheor.2023.87853","url":null,"abstract":"Background: Patients with advanced or recurrent endometrial cancer (EC) typically have limited treatment options and poor long-term survival outcomes following first-line therapy. Real-world treatment patterns and survival outcomes data are limited for patients in this setting. Objectives: The objective of this retrospective study was to describe real-world demographics, clinical characteristics, treatment patterns, and overall survival among patients in the United States with primary advanced or recurrent EC who initiated at least 1 line of therapy (LOT). Methods: Patients with a diagnosis of primary advanced or recurrent EC in a real-world database from January 1, 2013, to July 31, 2021, were included. The date for inclusion was the date of EC diagnosis documentation; patients were indexed for treatment patterns and outcomes at the start of the first LOT and at the start of each subsequent LOT they initiated. Data were stratified by subgroups of patients who had mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) tumors. Results: A total of 1961 patients who received at least 1 LOT were included. Most patients in this cohort, and the dMMR/MSI-H subgroup, received a platinum combination as first-line treatment, with carboplatin-paclitaxel being the most common regimen. Only 53% of patients who received first-line treatment subsequently received second-line therapy. Of the patients who received at least 1 LOT, use of immunotherapy in the second-line setting was more common in the dMMR/MSI-H subgroup. Median overall survival ranged from 14.1 to 31.8 months across the 5 most frequently used first-line treatment regimens in the ≥1 LOT cohort and became shorter with each subsequent LOT. Discussion: The use of platinum-based chemotherapy for first-line treatment of advanced or recurrent EC predominates in the real-world setting, despite the poor long-term survival outcomes associated with most of these regimens. Conclusions: Patients with recurrent/advanced EC have a poor prognosis, highlighting the need for therapies with more durable benefits.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"8 5","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136317061","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Hidden Economic Consequences of Migraine to the UK Government: Burden-of-Disease Analysis Using a Fiscal Framework","authors":"Rui Martins, Samuel Large, Rachel Russell, Gary Surmay, Mark Connolly","doi":"10.36469/jheor.2023.87790","DOIUrl":"https://doi.org/10.36469/jheor.2023.87790","url":null,"abstract":"Background: Migraine is a highly prevalent and incapacitating neurological disorder associated with the highest global disability burden in people aged 15 to 49 years. Europe has the fourth-highest prevalence of migraine, after North America, South America, and Central America, and above Asia and Africa. Migraine leads to relatively modest direct healthcare expenditure but has substantial indirect costs due to reduced productivity. Methods: The economic burden of migraine was estimated in comparison with the general population of the United Kingdom (UK) using an analytical fiscal modeling framework applying the government cost perspective. Published measures of migraine’s impact on labor participation were applied to rates of economic activity/inactivity of the general population. The model estimates lifetime changes to earnings from employment, direct and indirect taxes paid, and financial support requirements over the life course. Incremental differences between those affected and unaffected by migraine are reported as net fiscal consequences to public accounts. Fiscal costs are reported as the discounted average per capita over a 20-year time horizon and for the entire annual UK cohort with prevalent migraine. Results: People affected by migraine are more likely to be absent from work, unemployed, and disabled, and to retire early. A 44-year-old individual affected by migraine was associated with £19 823 in excess fiscal costs to the UK government, £1379 per year living with the condition, compared with someone not affected by the disease. Annually, migraine was estimated to represent £12.20 billion to the public economy, approximately £130.63 per migraine episode. The model predicted annual productivity losses in the health and social care workforce to be £2.05 billion and total annual productivity losses to be over £5.81 billion. Conclusions: This fiscal analysis monetizes the occupational consequences of migraine to the UK government, both in terms of lost tax revenue and transfer payments. The findings are substantial and useful to characterize disease severity and to inform the body of evidence considered by decision makers appraising the cost-effectiveness of health technologies.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"120 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135695758","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Hidden Economic Consequences of Migraine to the UK Government: Burden-of-Disease Analysis Using a Fiscal Framework.","authors":"Rui Martins, Samuel Large, Rachel Russell, Gary Surmay, Mark P Connolly","doi":"10.36469/001c.87790","DOIUrl":"10.36469/001c.87790","url":null,"abstract":"<p><p><b>Background:</b> Migraine is a highly prevalent and incapacitating neurological disorder associated with the highest global disability burden in people aged 15 to 49 years. Europe has the fourth-highest prevalence of migraine, after North America, South America, and Central America, and above Asia and Africa. Migraine leads to relatively modest direct healthcare expenditure but has substantial indirect costs due to reduced productivity. <b>Methods:</b> The economic burden of migraine was estimated in comparison with the general population of the United Kingdom (UK) using an analytical fiscal modeling framework applying the government cost perspective. Published measures of migraine's impact on labor participation were applied to rates of economic activity/inactivity of the general population. The model estimates lifetime changes to earnings from employment, direct and indirect taxes paid, and financial support requirements over the life course. Incremental differences between those affected and unaffected by migraine are reported as net fiscal consequences to public accounts. Fiscal costs are reported as the discounted average per capita over a 20-year time horizon and for the entire annual UK cohort with prevalent migraine. <b>Results:</b> People affected by migraine are more likely to be absent from work, unemployed, and disabled, and to retire early. A 44-year-old individual affected by migraine was associated with £19 823 in excess fiscal costs to the UK government, £1379 per year living with the condition, compared with someone not affected by the disease. Annually, migraine was estimated to represent £12.20 billion to the public economy, approximately £130.63 per migraine episode. The model predicted annual productivity losses in the health and social care workforce to be £2.05 billion and total annual productivity losses to be over £5.81 billion. <b>Conclusions:</b> This fiscal analysis monetizes the occupational consequences of migraine to the UK government, both in terms of lost tax revenue and transfer payments. The findings are substantial and useful to characterize disease severity and to inform the body of evidence considered by decision makers appraising the cost-effectiveness of health technologies.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 2","pages":"72-81"},"PeriodicalIF":0.0,"publicationDate":"2023-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10552723/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41116710","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global Surgeon Opinion on the Impact of Surgical Access When Using Endocutters Across Specialties","authors":"Marina Gutierrez, Nadine Jamous, William Petraiuolo, Sanjoy Roy","doi":"10.36469/jheor.2023.87644","DOIUrl":"https://doi.org/10.36469/jheor.2023.87644","url":null,"abstract":"Background: Despite design enhancements in endocutters, key challenges related to limited surgical access and space can impact stapling and, potentially, surgical outcomes. Objectives: This study aimed to develop consensus statements outlining the clinical value of precise articulation and greater anatomical access in minimally invasive surgery performed by bariatric, colorectal, and thoracic surgeons. Methods: Colorectal, bariatric, and thoracic surgeons from Japan, the United States, United Kingdom, and France participated in a 2-round modified Delphi panel. Round 1 included binary, Likert scale–type, multiple-response, and open-ended questions. These were converted to affirmative statements for round 2 if sufficient agreement was reached. Consensus was set at a predefined threshold of at least 90% of panelists across all surgical specialties and regions selecting the same option (“agree” or “disagree”) for the affirmative statements. Results: Of the 49 statements in the round 2 questionnaire, panelists (n=135) reached consensus that (1) tissue slippage outside stapler jaws can occur due to limited access and space; (2) greater jaw aperture could help to manipulate thick or fragile tissue more easily; (3) articulation of an endocutter is clinically important in laparoscopic surgeries; (4) improved access to hard-to-reach targets and in limited space would improve safety; and (5) an endocutter with improved access through greater articulation would become common use. Discussion: By understanding user-specific challenges and needs from both specialty- and region-wide perspectives, endoscopic stapling devices can continue to be refined. In this study, improved articulation and greater jaw aperture were the key design features examined. Improved articulation and greater jaw aperture were key stapler design features identified in this study that may mitigate the risk of instrument clashes and intraoperative complications such as anastomotic leaks. Conclusions: This study gained insights into surgeons’ perspective across a variety of specialties and from 3 distinct geographies. Participating surgeons reached consensus that an endocutter with greater jaw aperture and articulation may improve surgical access and has potential to improve surgical outcomes.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"79 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136308653","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global Surgeon Opinion on the Impact of Surgical Access When Using Endocutters Across Specialties.","authors":"Marina Gutierrez, Nadine Jamous, William Petraiuolo, Sanjoy Roy","doi":"10.36469/001c.87644","DOIUrl":"10.36469/001c.87644","url":null,"abstract":"<p><p><b>Background:</b> Despite design enhancements in endocutters, key challenges related to limited surgical access and space can impact stapling and, potentially, surgical outcomes. <b>Objectives:</b> This study aimed to develop consensus statements outlining the clinical value of precise articulation and greater anatomical access in minimally invasive surgery performed by bariatric, colorectal, and thoracic surgeons. <b>Methods:</b> Colorectal, bariatric, and thoracic surgeons from Japan, the United States, United Kingdom, and France participated in a 2-round modified Delphi panel. Round 1 included binary, Likert scale-type, multiple-response, and open-ended questions. These were converted to affirmative statements for round 2 if sufficient agreement was reached. Consensus was set at a predefined threshold of at least 90% of panelists across all surgical specialties and regions selecting the same option (\"agree\" or \"disagree\") for the affirmative statements. <b>Results:</b> Of the 49 statements in the round 2 questionnaire, panelists (n=135) reached consensus that (1) tissue slippage outside stapler jaws can occur due to limited access and space; (2) greater jaw aperture could help to manipulate thick or fragile tissue more easily; (3) articulation of an endocutter is clinically important in laparoscopic surgeries; (4) improved access to hard-to-reach targets and in limited space would improve safety; and (5) an endocutter with improved access through greater articulation would become common use. <b>Discussion:</b> By understanding user-specific challenges and needs from both specialty- and region-wide perspectives, endoscopic stapling devices can continue to be refined. In this study, improved articulation and greater jaw aperture were the key design features examined. Improved articulation and greater jaw aperture were key stapler design features identified in this study that may mitigate the risk of instrument clashes and intraoperative complications such as anastomotic leaks. <b>Conclusions:</b> This study gained insights into surgeons' perspective across a variety of specialties and from 3 distinct geographies. Participating surgeons reached consensus that an endocutter with greater jaw aperture and articulation may improve surgical access and has potential to improve surgical outcomes.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 2","pages":"62-71"},"PeriodicalIF":0.0,"publicationDate":"2023-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10515882/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41135499","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes for Dostarlimab and Real-World Treatments in Post-platinum Patients With Advanced/Recurrent Endometrial Cancer: The GARNET Trial Versus a US Electronic Health Record-Based External Control Arm","authors":"Scott Goulden, Qin Shen, Robert Coleman, Cara Mathews, Matthias Hunger, Ankit Pahwa, Rene Schade","doi":"10.36469/jheor.2023.77484","DOIUrl":"https://doi.org/10.36469/jheor.2023.77484","url":null,"abstract":"Background: Patients with advanced or recurrent endometrial cancer (EC) have limited treatment options following platinum-based chemotherapy and poor prognosis. The single-arm, Phase I GARNET trial (NCT02715284) previously reported dostarlimab efficacy in mismatch repair–deficient/microsatellite instability–high advanced or recurrent EC. Objectives: The objective of this study was to compare overall survival (OS) and describe time to treatment discontinuation (TTD) for dostarlimab (GARNET Cohort A1 safety population) with an equivalent real-world external control arm receiving non-anti-programmed death (PD)-1/PD-ligand (L)1/2 treatments (constructed using data from a nationwide electronic health record–derived de-identified database and applied GARNET eligibility criteria). Methods: Propensity scores constructed from prognostic factors, identified by literature review and clinical experts, were used for inverse probability of treatment weighting (IPTW). Kaplan-Meier curves were constructed and OS/TTD was estimated (Cox regression model was used to estimate the OS-adjusted hazard ratio). Results: Dostarlimab was associated with a 52% lower risk of death vs real-world treatments (hazard ratio, 0.48; 95% confidence interval [CI], 0.35-0.66). IPTW-adjusted median OS for dostarlimab (N=143) was not estimable (95% CI, 19.4–not estimable) versus 13.1 months (95% CI, 8.3-15.9) for real-world treatments (N = 185). Median TTD was 11.7 months (95% CI, 6.0-38.7) for dostarlimab and 5.3 months (95% CI, 4.1-6.0) for the real-world cohort. Discussion: Consistent with previous analyses, patients treated with dostarlimab had significantly longer OS than patients in the US real-world cohort after adjusting for the lack of randomization using stabilized IPTW. Additionally, patients had a long TTD when treated with dostarlimab, suggesting a favorable tolerability profile. Conclusion: Patients with advanced or recurrent EC receiving dostarlimab in GARNET had significantly lower risk of death than those receiving real-world non-anti-PD-(L)1/2 treatments.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"94 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136361220","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes for Dostarlimab and Real-World Treatments in Post-platinum Patients With Advanced/Recurrent Endometrial Cancer: The GARNET Trial Versus a US Electronic Health Record-Based External Control Arm.","authors":"Scott Goulden, Qin Shen, Robert L Coleman, Cara Mathews, Matthias Hunger, Ankit Pahwa, Rene Schade","doi":"10.36469/001c.77484","DOIUrl":"10.36469/001c.77484","url":null,"abstract":"<p><p><b>Background:</b> Patients with advanced or recurrent endometrial cancer (EC) have limited treatment options following platinum-based chemotherapy and poor prognosis. The single-arm, Phase I GARNET trial (NCT02715284) previously reported dostarlimab efficacy in mismatch repair-deficient/microsatellite instability-high advanced or recurrent EC. <b>Objectives:</b> The objective of this study was to compare overall survival (OS) and describe time to treatment discontinuation (TTD) for dostarlimab (GARNET Cohort A1 safety population) with an equivalent real-world external control arm receiving non-anti-programmed death (PD)-1/PD-ligand (L)1/2 treatments (constructed using data from a nationwide electronic health record-derived de-identified database and applied GARNET eligibility criteria). <b>Methods:</b> Propensity scores constructed from prognostic factors, identified by literature review and clinical experts, were used for inverse probability of treatment weighting (IPTW). Kaplan-Meier curves were constructed and OS/TTD was estimated (Cox regression model was used to estimate the OS-adjusted hazard ratio). <b>Results:</b> Dostarlimab was associated with a 52% lower risk of death vs real-world treatments (hazard ratio, 0.48; 95% confidence interval [CI], 0.35-0.66). IPTW-adjusted median OS for dostarlimab (N=143) was not estimable (95% CI, 19.4-not estimable) versus 13.1 months (95% CI, 8.3-15.9) for real-world treatments (N = 185). Median TTD was 11.7 months (95% CI, 6.0-38.7) for dostarlimab and 5.3 months (95% CI, 4.1-6.0) for the real-world cohort. <b>Discussion:</b> Consistent with previous analyses, patients treated with dostarlimab had significantly longer OS than patients in the US real-world cohort after adjusting for the lack of randomization using stabilized IPTW. Additionally, patients had a long TTD when treated with dostarlimab, suggesting a favorable tolerability profile. <b>Conclusion:</b> Patients with advanced or recurrent EC receiving dostarlimab in GARNET had significantly lower risk of death than those receiving real-world non-anti-PD-(L)1/2 treatments.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 2","pages":"53-61"},"PeriodicalIF":0.0,"publicationDate":"2023-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10493167/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10589111","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}