Journal of Health Economics and Outcomes Research最新文献

{"title":"HTA and Reimbursement Status of Metastatic Hormone‑Sensitive Prostate Cancer, Non-Metastatic Castration-Resistant Prostate Cancer, and Metastatic Castration-Resistant Prostate Cancer Treatments in Europe: A Patient Access Landscape Review.","authors":"Goran Bencina, Elina Petrova, Demet Sönmez, Sonia Matos Pereira, Ioannis Dimitriadis, Stina Salomonsson","doi":"10.36469/001c.75208","DOIUrl":"10.36469/001c.75208","url":null,"abstract":"<p><p><b>Background:</b> Prostate cancer is the second most common cancer in men, with up to one-third of men being diagnosed in their lifetime. Recently, novel therapies have received regulatory approval with significant improvement in overall survival for metastatic castration-resistant prostate cancer, metastatic hormone-sensitive prostate cancer, and nonmetastatic castration-resistant prostate cancer. To improve decision-making regarding the value of anticancer therapies and support standardized assessment for use by health technology assessment (HTA) agencies, the European Society for Medical Oncology (ESMO) has developed a Magnitude of Clinical Benefit Scale (MCBS). <b>Objective:</b> This review aimed to map HTA status, reimbursement restrictions, and patient access for 3 advanced prostate cancer indications across 23 European countries during 2011-2021. <b>Methods:</b> HTA, country reimbursement lists, and ESMO-MCBS scorecards were reviewed for evidence and data across 26 European countries. <b>Results:</b> The analysis demonstrated that only in Greece, Germany, and Sweden was there full access across all included prostate cancer treatments. Treatments available for metastatic castration-resistant prostate cancer were widely reimbursed, with both abiraterone and enzalutamide accessible in all countries. In 3 countries (Hungary, the Netherlands, and Switzerland), there was a statistically significant difference (<i>P</i><.05) between status of reimbursement and ESMO-MCBS \"substantial benefit\" (score of 4 or 5) vs \"no substantial benefit\" (score <4). <b>Conclusion:</b> Overall, the impact of the ESMO-MCBS on reimbursement decisions in Europe is unclear, with significant variation across the countries included in this review.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 1","pages":"102-110"},"PeriodicalIF":2.3,"publicationDate":"2023-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10290826/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9721377","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":"HTA and Reimbursement Status of Metastatic Hormone‑Sensitive Prostate Cancer, Non-Metastatic Castration-Resistant Prostate Cancer, and Metastatic Castration-Resistant Prostate Cancer Treatments in Europe: A Patient Access Landscape Review","authors":"Goran Bencina, Elina Petrova, Demet Sönmez, Sonia Matos Pereira, Ioannis Dimitriadis, Stina Salomonsson","doi":"10.36469/jheor.2023.75208","DOIUrl":"https://doi.org/10.36469/jheor.2023.75208","url":null,"abstract":"Background: Prostate cancer is the second most common cancer in men, with up to one-third of men being diagnosed in their lifetime. Recently, novel therapies have received regulatory approval with significant improvement in overall survival for metastatic castration-resistant prostate cancer, metastatic hormone-sensitive prostate cancer, and nonmetastatic castration-resistant prostate cancer. To improve decision-making regarding the value of anticancer therapies and support standardized assessment for use by health technology assessment (HTA) agencies, the European Society for Medical Oncology (ESMO) has developed a Magnitude of Clinical Benefit Scale (MCBS). Objective: This review aimed to map HTA status, reimbursement restrictions, and patient access for 3 advanced prostate cancer indications across 23 European countries during 2011-2021. Methods: HTA, country reimbursement lists, and ESMO-MCBS scorecards were reviewed for evidence and data across 26 European countries. Results: The analysis demonstrated that only in Greece, Germany, and Sweden was there full access across all included prostate cancer treatments. Treatments available for metastatic castration-resistant prostate cancer were widely reimbursed, with both abiraterone and enzalutamide accessible in all countries. In 3 countries (Hungary, the Netherlands, and Switzerland), there was a statistically significant difference (P<.05) between status of reimbursement and ESMO-MCBS “substantial benefit” (score of 4 or 5) vs “no substantial benefit” (score <4). Conclusion: Overall, the impact of the ESMO-MCBS on reimbursement decisions in Europe is unclear, with significant variation across the countries included in this review.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"195 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135188176","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 Clinical and Economic Impact of Employees Who Are Care Partners of Patients with Multiple Sclerosis by Disease Severity.","authors":"Barry Hendin, Richard A Brook, Ian A Beren, Nathan Kleinman, Cindy Fink, Amy L Phillips, Carroline Lobo","doi":"10.36469/001c.57593","DOIUrl":"10.36469/001c.57593","url":null,"abstract":"<p><p><b>Background:</b> Research on employee care partners of patients with multiple sclerosis (MS) is limited. <b>Objectives:</b> The clinical and economic impact on employee care partners was evaluated by MS disease severity. <b>Methods:</b> Employees with spouses/domestic partners with MS from the Workpartners database (Jan. 1, 2010-Dec. 31, 2019) were eligible if: spouse/partner had at least 3 MS-related (ICD-9-CM/ICD-10-CM:340.xx/G35) inpatient/outpatient/disease-modifying therapy claims within 1 year (latest claim = index date); 6-month pre-index/1-year post-index enrollment; and age 18 to 64 years. Employee care partners' demographic/clinical characteristics and direct/indirect costs were compared across predetermined MS severity categories. Logistic and generalized linear regression modeled the costs. <b>Results:</b> Among 1041 employee care partners of patients with MS, 358 (34.4%) patients had mild MS, 491 (47.2%) moderate, and 192 (18.4%) severe. Mean (standard error [SE]) employee care partner age was 49.0 (0.5) for patients with mild disease, 50.5 (0.4) for moderate, 51.7 (0.6) for severe; percent female care partners was 24.6% [2.3%] mild, 19.8% [1.8%] moderate, 27.6% [3.2%] severe; and mean care partner Charlson Comorbidity Index scores 0.28 (0.05) mild, 0.30 (0.04) moderate, 0.27 (0.06) severe. More care partners of patients with moderate/severe vs mild MS had hyperlipidemia (32.6%/31.8% vs 21.2%), hypertension (29.5%/29.7% vs 19.3%), gastrointestinal disease (20.8%/22.9% vs 13.1%), depression (9.2%/10.9% vs 3.9%), and anxiety 10.6%/8.9% vs 4.2%). Adjusted mean medical costs were greater for employee care partners of patients with moderate vs mild/severe disease (<i>P</i><.001). Pharmacy costs (SE) were lower for employee care partners of mild vs severe/moderate patients (<i>P</i><.005). Sick leave costs (SE) were greater for employee care partners of mild/severe vs moderate patients (<i>P</i><.05). <b>Discussion:</b> Employee care partners of patients with moderate/severe vs mild MS had more comorbidities (ie, hypertension, gastrointestinal disease, depression, and anxiety) and higher pharmacy costs. Employee care partners of patients with moderate vs mild/severe MS had higher medical and lower sick leave costs. Treatment strategies that improve patient outcomes may reduce employee care partner burden and lower costs for employers in some instances. <b>Conclusions:</b> Comorbidities and direct/indirect costs of employees whose spouses/partners have MS were considerable and varied with MS severity.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 1","pages":"91-101"},"PeriodicalIF":0.0,"publicationDate":"2023-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10105615/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9693634","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":"The Clinical and Economic Impact of Employees Who Are Care Partners of Patients with Multiple Sclerosis by Disease Severity","authors":"Barry Hendin, Richard Brook, Ian Beren, Nathan Kleinman, Cindy Fink, Amy Phillips, Carroline Lobo","doi":"10.36469/jheor.2023.57593","DOIUrl":"https://doi.org/10.36469/jheor.2023.57593","url":null,"abstract":"Background: Research on employee care partners of patients with multiple sclerosis (MS) is limited. Objectives: The clinical and economic impact on employee care partners was evaluated by MS disease severity. Methods: Employees with spouses/domestic partners with MS from the Workpartners database (Jan. 1, 2010–Dec. 31, 2019) were eligible if: spouse/partner had at least 3 MS-related (ICD-9-CM/ICD-10-CM:340.xx/G35) inpatient/outpatient/disease-modifying therapy claims within 1 year (latest claim = index date); 6-month pre-index/1-year post-index enrollment; and age 18 to 64 years. Employee care partners’ demographic/clinical characteristics and direct/indirect costs were compared across predetermined MS severity categories. Logistic and generalized linear regression modeled the costs. Results: Among 1041 employee care partners of patients with MS, 358 (34.4%) patients had mild MS, 491 (47.2%) moderate, and 192 (18.4%) severe. Mean (standard error [SE]) employee care partner age was 49.0 (0.5) for patients with mild disease, 50.5 (0.4) for moderate, 51.7 (0.6) for severe; percent female care partners was 24.6% [2.3%] mild, 19.8% [1.8%] moderate, 27.6% [3.2%] severe; and mean care partner Charlson Comorbidity Index scores 0.28 (0.05) mild, 0.30 (0.04) moderate, 0.27 (0.06) severe. More care partners of patients with moderate/severe vs mild MS had hyperlipidemia (32.6%/31.8% vs 21.2%), hypertension (29.5%/29.7% vs 19.3%), gastrointestinal disease (20.8%/22.9% vs 13.1%), depression (9.2%/10.9% vs 3.9%), and anxiety 10.6%/8.9% vs 4.2%). Adjusted mean medical costs were greater for employee care partners of patients with moderate vs mild/severe disease (P<.001). Pharmacy costs (SE) were lower for employee care partners of mild vs severe/moderate patients (P<.005). Sick leave costs (SE) were greater for employee care partners of mild/severe vs moderate patients (P<.05). Discussion: Employee care partners of patients with moderate/severe vs mild MS had more comorbidities (ie, hypertension, gastrointestinal disease, depression, and anxiety) and higher pharmacy costs. Employee care partners of patients with moderate vs mild/severe MS had higher medical and lower sick leave costs. Treatment strategies that improve patient outcomes may reduce employee care partner burden and lower costs for employers in some instances. Conclusions: Comorbidities and direct/indirect costs of employees whose spouses/partners have MS were considerable and varied with MS severity.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135189507","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":"Response to Letter to the Editor","authors":"Folashayo Adeniji, Taiwo Obembe","doi":"10.36469/jheor.2023.74215","DOIUrl":"https://doi.org/10.36469/jheor.2023.74215","url":null,"abstract":"The authors respond to the comments raised in the letter regarding Adeniji and Obembe’s article on catastrophic health expenditures in sub-Saharan Africa.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"32 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135289949","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":"Letter to the Editor.","authors":"Ishir Narayan","doi":"10.36469/001c.74185","DOIUrl":"10.36469/001c.74185","url":null,"abstract":"<p><p>Highlighting other risk factors for cardiovascular disease could enhance Adeniji and Obembe's study.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 1","pages":"89"},"PeriodicalIF":2.3,"publicationDate":"2023-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10103749/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9671619","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":"Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation","authors":"Derralynn Hughes, Andrew Lenny, Koonal Shah, Louise Longworth, Giovanna Devercelli, Olulade Ayodele","doi":"10.36469/jheor.2023.71344","DOIUrl":"https://doi.org/10.36469/jheor.2023.71344","url":null,"abstract":"Background: Health state utilities are measures of health-related quality of life that reflect the value placed on improvements in patients’ health status and are necessary for estimation of quality-adjusted life-years. Health state utility data on Fabry disease (FD) are limited. In this study we used vignette (scenario) construction and valuation to develop health state utilities. Objectives: The aim of this study was to use vignette construction and valuation to estimate health state utility values suitable for inclusion in economic models of FD treatments. Methods: Health state vignettes were developed from semistructured qualitative telephone interviews with patients with FD and informed by published literature and input from an expert. Each vignette was valued in an online survey by members of the United Kingdom (UK) general population using the composite time trade-off (TTO) method, which aims to determine the time the respondent would trade to live in full health compared with each impaired health state. Results: Eight adults (50% women) with FD from the UK were interviewed. They were recruited via various approaches, including patient organizations and social media. The interviewees’ responses, evidence from published literature, and input from a clinical expert informed the development of 6 health state vignettes (pain, moderate clinically evident FD [CEFD], severe CEFD, end-stage renal disease [ESRD], stroke, and cardiovascular disease [CVD]) and 3 combined health states (severe CEFD + ESRD, severe CEFD + CVD, and severe CEFD + stroke). A vignette valuation survey was administered to 1222 participants from the UK general population who were members of an external surveying organization and agreed to participate in this study; 1175 surveys were successfully completed and included in the analysis. Responses to TTO questions were converted into utility values for each health state. Pain was the highest valued health state (0.465), and severe CEFD + ESRD was the lowest (0.033). Discussion: Overall, mean utility values declined as the severity of the vignettes increased, indicating that respondents were more willing to trade life-years to avoid a severe health state. Conclusions: Health state vignettes reflect the effects of FD on all major health-related quality-of-life domains and may help to support economic modeling for treatment of FD.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"100 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135543672","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":"Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation.","authors":"Derralynn Hughes, Andrew Lenny, Koonal Shah, Louise Longworth, Giovanna Devercelli, Olulade Ayodele","doi":"10.36469/001c.71344","DOIUrl":"10.36469/001c.71344","url":null,"abstract":"<p><p><b>Background:</b> Health state utilities are measures of health-related quality of life that reflect the value placed on improvements in patients' health status and are necessary for estimation of quality-adjusted life-years. Health state utility data on Fabry disease (FD) are limited. In this study we used vignette (scenario) construction and valuation to develop health state utilities. <b>Objectives:</b> The aim of this study was to use vignette construction and valuation to estimate health state utility values suitable for inclusion in economic models of FD treatments. <b>Methods:</b> Health state vignettes were developed from semistructured qualitative telephone interviews with patients with FD and informed by published literature and input from an expert. Each vignette was valued in an online survey by members of the United Kingdom (UK) general population using the composite time trade-off (TTO) method, which aims to determine the time the respondent would trade to live in full health compared with each impaired health state. <b>Results:</b> Eight adults (50% women) with FD from the UK were interviewed. They were recruited via various approaches, including patient organizations and social media. The interviewees' responses, evidence from published literature, and input from a clinical expert informed the development of 6 health state vignettes (pain, moderate clinically evident FD [CEFD], severe CEFD, end-stage renal disease [ESRD], stroke, and cardiovascular disease [CVD]) and 3 combined health states (severe CEFD + ESRD, severe CEFD + CVD, and severe CEFD + stroke). A vignette valuation survey was administered to 1222 participants from the UK general population who were members of an external surveying organization and agreed to participate in this study; 1175 surveys were successfully completed and included in the analysis. Responses to TTO questions were converted into utility values for each health state. Pain was the highest valued health state (0.465), and severe CEFD + ESRD was the lowest (0.033). <b>Discussion:</b> Overall, mean utility values declined as the severity of the vignettes increased, indicating that respondents were more willing to trade life-years to avoid a severe health state. <b>Conclusions:</b> Health state vignettes reflect the effects of FD on all major health-related quality-of-life domains and may help to support economic modeling for treatment of FD.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 1","pages":"80-88"},"PeriodicalIF":0.0,"publicationDate":"2023-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10095067/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9671618","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":"Regarding \"Burden of Comorbidities and Healthcare Resource Utilization Among Medicaid-Enrolled Extremely Premature Infants\".","authors":"Ava Tsapatsaris, Miran Bhima, Tejas C Sekhar","doi":"10.36469/001c.73989","DOIUrl":"10.36469/001c.73989","url":null,"abstract":"<p><p>In their article, Mowitz et al investigated the burden of comorbidities and healthcare resource utilization among extremely premature infants enrolled in Medicaid, laying a foundation for further policy action.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"10 1","pages":"77-79"},"PeriodicalIF":2.3,"publicationDate":"2023-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10076213/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9641124","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":"Regarding “Burden of Comorbidities and Healthcare Resource Utilization Among Medicaid-Enrolled Extremely Premature Infants.”","authors":"Ava Tsapatsaris, Miran Brim, Tejas Sekhar","doi":"10.36469/jheor.2023.73989","DOIUrl":"https://doi.org/10.36469/jheor.2023.73989","url":null,"abstract":"In their article, Mowitz et al investigated the burden of comorbidities and healthcare resource utilization among extremely premature infants enrolled in Medicaid, laying a foundation for further policy action.","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"36 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135466528","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}