Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.08.012
Andrew H. Briggs DPhil , Alexis Doyle-Connolly MA , John Schneider PhD , Tanja Podkonjak PhD , Helen Taylor BA (Hons) , Emma Roffe PhD , Eric Low MSc , Sarah Davis MPhys , Martin Kaiser MD , Anthony J. Hatswell PhD , Neil Rabin MD
Objectives
The use of cost-effectiveness methods to support policy decisions has become well established, but difficulties can arise when evaluating a new treatment that is indicated to be used in combination with an established backbone treatment. If the latter has been priced close to the decision maker’s willingness-to-pay threshold, this may mean that there is no headroom for the new treatment to demonstrate value, at any price, even if the combination is clinically effective. Without a mechanism for attributing value to component treatments within a combination therapy, the health system risks generating negative funding decisions for combinations of proven clinical benefit to patients. The aim of this work was to define a value attribution methodology, which could be used to allocate value between the components of any combination treatment.
Methods
The framework is grounded in the standard decision rules of cost-effectiveness analysis and provides solutions according to key features of the problem: perfect/imperfect information about component treatment monotherapy effects and balanced/unbalanced market power between their manufacturers.
Results
The share of incremental value varies depending on whether there is perfect/imperfect information and balance/imbalance of market power, with some scenarios requiring the manufacturers to negotiate a share of the incremental value within a range defined by the framework.
Conclusions
It is possible to define a framework that is independent of price and focuses on benefits expressed as quality-adjusted life-year gains (and/or quality-adjusted life-year equivalents for cost savings), a standard metric used by many health technology assessment agencies to evaluate novel treatments.
{"title":"An Attribution of Value Framework for Combination Treatments","authors":"Andrew H. Briggs DPhil , Alexis Doyle-Connolly MA , John Schneider PhD , Tanja Podkonjak PhD , Helen Taylor BA (Hons) , Emma Roffe PhD , Eric Low MSc , Sarah Davis MPhys , Martin Kaiser MD , Anthony J. Hatswell PhD , Neil Rabin MD","doi":"10.1016/j.jval.2024.08.012","DOIUrl":"10.1016/j.jval.2024.08.012","url":null,"abstract":"<div><h3>Objectives</h3><div>The use of cost-effectiveness methods to support policy decisions has become well established, but difficulties can arise when evaluating a new treatment that is indicated to be used in combination with an established backbone treatment. If the latter has been priced close to the decision maker’s willingness-to-pay threshold, this may mean that there is no headroom for the new treatment to demonstrate value, at any price, even if the combination is clinically effective. Without a mechanism for attributing value to component treatments within a combination therapy, the health system risks generating negative funding decisions for combinations of proven clinical benefit to patients. The aim of this work was to define a value attribution methodology, which could be used to allocate value between the components of any combination treatment.</div></div><div><h3>Methods</h3><div>The framework is grounded in the standard decision rules of cost-effectiveness analysis and provides solutions according to key features of the problem: perfect/imperfect information about component treatment monotherapy effects and balanced/unbalanced market power between their manufacturers.</div></div><div><h3>Results</h3><div>The share of incremental value varies depending on whether there is perfect/imperfect information and balance/imbalance of market power, with some scenarios requiring the manufacturers to negotiate a share of the incremental value within a range defined by the framework.</div></div><div><h3>Conclusions</h3><div>It is possible to define a framework that is independent of price and focuses on benefits expressed as quality-adjusted life-year gains (and/or quality-adjusted life-year equivalents for cost savings), a standard metric used by many health technology assessment agencies to evaluate novel treatments.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 72-80"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142558801","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.08.006
Krishna Sabareesh Rajangom MS , F. Safa Erenay PhD , Qi-Ming He PhD , Rachel Figueiredo BA, MLIS , Kelvin K.W. Chan MD, MSc, PhD , Matthew C. Cheung MD, SM , Lauren F. Charbonneau BSc Pharm , Susan E. Horton PhD , Avram Denburg MD, MSc, PhD
Objectives
To systematically review published evidence on cancer drug wastage and the effectiveness of mitigation methods.
Methods
Search keywords for Scopus, PubMed, and EMBASE were developed using the Pearl Growing technique. Relevant articles were identified in a 2-step process: first, based on titles/abstracts, then on full article reviews. Among the identified English peer-reviewed articles, those considering adults ≥18 years and relevant cancer drug wastage outcomes were included. Key concepts and measures for drug wastage and its mitigation were tabulated. Trends in publication numbers were analyzed using Mann-Kendall tests. Costs were converted first to 2024 local currencies using country-wise consumer price indexes and then to 2024 USD using exchange rates.
Results
Among 6298 unique articles, 94 met the inclusion criteria. Seventy-four (79%) of these were published since 2015, highlighting increasing attention to cancer drug wastage. Twenty-three articles (24%) explicitly reported drug wastage amounts, whereas 52 articles (55%) considered the mitigation methods. Most articles focused on high-income countries (n = 67), single-hospital settings (n = 45), and retrospective study designs (n = 55). Wastage mitigation techniques included vial sharing (n = 21), dose rounding (n = 17), closed-system transfer device (n = 9), centralized drug preparation (n = 7), and vial size optimization (n = 7). A trend toward higher median wastage cost was evident in US settings ($135.35/patient-month) compared with other countries ($37.71/patient-month), whereas mitigation methods across countries were not statistically significant.
Conclusions
High cancer drug costs highlight the importance of minimizing drug wastage to reduce healthcare expenditure. Our review demonstrates that wastage varies by healthcare setting and mitigation technique. Future studies would benefit from reporting standards for cancer drug wastage that include reporting wastage (both in mg and cost, preferably in terms of purchase power parity), as well as cohort size, considered vial sizes, considered dosages, and used mitigation methods separately for each drug. This approach would account for variability in cancer drug wastage and help identify optimal mitigation practices tailored to the health system context.
{"title":"Cancer Drug Wastage and Mitigation Methods: A Systematic Review","authors":"Krishna Sabareesh Rajangom MS , F. Safa Erenay PhD , Qi-Ming He PhD , Rachel Figueiredo BA, MLIS , Kelvin K.W. Chan MD, MSc, PhD , Matthew C. Cheung MD, SM , Lauren F. Charbonneau BSc Pharm , Susan E. Horton PhD , Avram Denburg MD, MSc, PhD","doi":"10.1016/j.jval.2024.08.006","DOIUrl":"10.1016/j.jval.2024.08.006","url":null,"abstract":"<div><h3>Objectives</h3><div>To systematically review published evidence on cancer drug wastage and the effectiveness of mitigation methods.</div></div><div><h3>Methods</h3><div>Search keywords for Scopus, PubMed, and EMBASE were developed using the Pearl Growing technique. Relevant articles were identified in a 2-step process: first, based on titles/abstracts, then on full article reviews. Among the identified English peer-reviewed articles, those considering adults ≥18 years and relevant cancer drug wastage outcomes were included. Key concepts and measures for drug wastage and its mitigation were tabulated. Trends in publication numbers were analyzed using Mann-Kendall tests. Costs were converted first to 2024 local currencies using country-wise consumer price indexes and then to 2024 USD using exchange rates.</div></div><div><h3>Results</h3><div>Among 6298 unique articles, 94 met the inclusion criteria. Seventy-four (79%) of these were published since 2015, highlighting increasing attention to cancer drug wastage. Twenty-three articles (24%) explicitly reported drug wastage amounts, whereas 52 articles (55%) considered the mitigation methods. Most articles focused on high-income countries (<em>n</em> = 67), single-hospital settings (<em>n</em> = 45), and retrospective study designs (<em>n</em> = 55). Wastage mitigation techniques included vial sharing (<em>n =</em> 21), dose rounding (<em>n</em> = 17), closed-system transfer device (<em>n</em> = 9), centralized drug preparation (<em>n</em> = 7), and vial size optimization (<em>n</em> = 7). A trend toward higher median wastage cost was evident in US settings ($135.35/patient-month) compared with other countries ($37.71/patient-month), whereas mitigation methods across countries were not statistically significant.</div></div><div><h3>Conclusions</h3><div>High cancer drug costs highlight the importance of minimizing drug wastage to reduce healthcare expenditure. Our review demonstrates that wastage varies by healthcare setting and mitigation technique. Future studies would benefit from reporting standards for cancer drug wastage that include reporting wastage (both in mg and cost, preferably in terms of purchase power parity), as well as cohort size, considered vial sizes, considered dosages, and used mitigation methods separately for each drug. This approach would account for variability in cancer drug wastage and help identify optimal mitigation practices tailored to the health system context.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 148-160"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354769","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.09.001
Siobhan Botwright MA , Manit Sittimart MSc , Kinanti Khansa Chavarina MPH , Diana Beatriz Bayani PhD , Tracy Merlin PhD , Gavin Surgey MCom , Christian Suharlim MD, MPH , Manuel A. Espinoza MD, PhD , Anthony J. Culyer Hon DEcon , Wija Oortwijn PhD , Yot Teerawattananon MD, PhD
Objectives
Health technology assessment (HTA) guidelines are intended to support successful implementation of HTA by enhancing consistency and transparency in concepts, methods, process, and use, thereby enhancing the legitimacy of the decision-making process. This report lays out good practices and practical recommendations for developing or updating HTA guidelines to ensure successful implementation.
Methods
The task force was established in 2022 and comprised experts and academics from various geographical regions, each with substantial experience in developing HTA guidelines for national health policymaking. Literature reviews and key-informant interviews were conducted to inform these good practices. Stakeholder consultations, open peer reviews, and expert opinions validated the recommendations. A series of teleconferences among task force members was held to iteratively refine the report.
Results
The recommendations cover 6 key aspects throughout the guideline development cycle: (1) setting objectives, scope, and principles of the guideline, (2) building a team for a quality guideline, (3) defining a stakeholder engagement plan, (iv) developing content and utilizing available resources, (v) putting in place appropriate institutional arrangements, and (vi) monitoring and evaluating guideline success.
Conclusion
This report presents a set of resources and context-appropriate practices for developing or updating HTA guidelines. Across all contexts, the recommendations emphasize transparency, building trust among stakeholders, and fostering a culture of ongoing learning and improvement. The report recommends timing development and revision of guidelines according to the HTA landscape and pace of HTA institutionalization. Because HTA is increasingly used to inform different kinds of decision making in a variety of country contexts, it will be important to continue to monitor lessons learned to ensure the recommendations remain relevant and effective.
{"title":"Good Practices for Health Technology Assessment Guideline Development: A Report of the Health Technology Assessment International, HTAsiaLink, and ISPOR Special Task Force","authors":"Siobhan Botwright MA , Manit Sittimart MSc , Kinanti Khansa Chavarina MPH , Diana Beatriz Bayani PhD , Tracy Merlin PhD , Gavin Surgey MCom , Christian Suharlim MD, MPH , Manuel A. Espinoza MD, PhD , Anthony J. Culyer Hon DEcon , Wija Oortwijn PhD , Yot Teerawattananon MD, PhD","doi":"10.1016/j.jval.2024.09.001","DOIUrl":"10.1016/j.jval.2024.09.001","url":null,"abstract":"<div><h3>Objectives</h3><div>Health technology assessment (HTA) guidelines are intended to support successful implementation of HTA by enhancing consistency and transparency in concepts, methods, process, and use, thereby enhancing the legitimacy of the decision-making process. This report lays out good practices and practical recommendations for developing or updating HTA guidelines to ensure successful implementation.</div></div><div><h3>Methods</h3><div>The task force was established in 2022 and comprised experts and academics from various geographical regions, each with substantial experience in developing HTA guidelines for national health policymaking. Literature reviews and key-informant interviews were conducted to inform these good practices. Stakeholder consultations, open peer reviews, and expert opinions validated the recommendations. A series of teleconferences among task force members was held to iteratively refine the report.</div></div><div><h3>Results</h3><div>The recommendations cover 6 key aspects throughout the guideline development cycle: (1) setting objectives, scope, and principles of the guideline, (2) building a team for a quality guideline, (3) defining a stakeholder engagement plan, (iv) developing content and utilizing available resources, (v) putting in place appropriate institutional arrangements, and (vi) monitoring and evaluating guideline success.</div></div><div><h3>Conclusion</h3><div>This report presents a set of resources and context-appropriate practices for developing or updating HTA guidelines. Across all contexts, the recommendations emphasize transparency, building trust among stakeholders, and fostering a culture of ongoing learning and improvement. The report recommends timing development and revision of guidelines according to the HTA landscape and pace of HTA institutionalization. Because HTA is increasingly used to inform different kinds of decision making in a variety of country contexts, it will be important to continue to monitor lessons learned to ensure the recommendations remain relevant and effective.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 1-15"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143179174","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.08.007
Guilherme T. Arruda PhD , Maria Eduarda C.B. da Silva BSc , Barbara I. da Silva , Patricia Driusso PhD , Mariana A. Avila PhD
Objectives
To develop the Dysmenorrhea-related Impact on Functioning Scale (DIFS) to assess the impact of dysmenorrhea on functioning in cisgender women and transgender men and to evaluate its measurement properties.
Methods
Mixed and online design study conducted with adolescents and adult cisgender women and transgender men with dysmenorrhea. We developed the DIFS based on the International Classification of Functioning, Disability, and Health. Content validity was assessed with experts and people with dysmenorrhea. Item Response Theory developed the DIFS total score. Structural validity was assessed by exploratory and confirmatory factor analysis and internal consistency by Cronbach’s α and McDonald’s Ω. Construct validity and test-retest reliability were assessed by correlation between DIFS and World Health Organization Disability Assessment Schedule and intraclass correlation coefficient, respectively. Measurement error was also assessed.
Results
A total of 3335 people participated in the study. The DIFS is a 15-item instrument divided into “Bodily Functions” and “Daily Activities and Social Participation” sections and “Functioning” as a general factor. Internal consistency (α and Ω > 0.7) and test-retest reliability (intraclass correlation coefficient > 0.9) were adequate. No systematic error was found. Correlation was positive and strong between World Health Organization Disability Assessment Schedule and “Functioning” (r = 0.62, P ≤ .05). For the DIFS total score, higher scores indicate a greater impact of dysmenorrhea on functioning, and 44 points is the cutoff point for classifying the person with a significant impact of dysmenorrhea on functioning.
Conclusions
DIFS showed excellent measurement properties for assessing the impact of dysmenorrhea on functioning for cisgender women and transgender men.
{"title":"Dysmenorrhea-Related Impact on Functioning Scale: Development and Measurement Properties for Cisgender Women and Transgender Men","authors":"Guilherme T. Arruda PhD , Maria Eduarda C.B. da Silva BSc , Barbara I. da Silva , Patricia Driusso PhD , Mariana A. Avila PhD","doi":"10.1016/j.jval.2024.08.007","DOIUrl":"10.1016/j.jval.2024.08.007","url":null,"abstract":"<div><h3>Objectives</h3><div>To develop the Dysmenorrhea-related Impact on Functioning Scale (DIFS) to assess the impact of dysmenorrhea on functioning in cisgender women and transgender men and to evaluate its measurement properties.</div></div><div><h3>Methods</h3><div>Mixed and online design study conducted with adolescents and adult cisgender women and transgender men with dysmenorrhea. We developed the DIFS based on the International Classification of Functioning, Disability, and Health. Content validity was assessed with experts and people with dysmenorrhea. Item Response Theory developed the DIFS total score. Structural validity was assessed by exploratory and confirmatory factor analysis and internal consistency by Cronbach’s α and McDonald’s Ω. Construct validity and test-retest reliability were assessed by correlation between DIFS and World Health Organization Disability Assessment Schedule and intraclass correlation coefficient, respectively. Measurement error was also assessed.</div></div><div><h3>Results</h3><div>A total of 3335 people participated in the study. The DIFS is a 15-item instrument divided into “Bodily Functions” and “Daily Activities and Social Participation” sections and “Functioning” as a general factor. Internal consistency (α and Ω > 0.7) and test-retest reliability (intraclass correlation coefficient > 0.9) were adequate. No systematic error was found. Correlation was positive and strong between World Health Organization Disability Assessment Schedule and “Functioning” (r = 0.62, <em>P</em> ≤ .05). For the DIFS total score, higher scores indicate a greater impact of dysmenorrhea on functioning, and 44 points is the cutoff point for classifying the person with a significant impact of dysmenorrhea on functioning.</div></div><div><h3>Conclusions</h3><div>DIFS showed excellent measurement properties for assessing the impact of dysmenorrhea on functioning for cisgender women and transgender men.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 99-107"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354771","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.08.005
Joseph Kwon PhD , Rakhee Raghunandan PhD , Son Hong Nghiem PhD , Kirsten Howard PhD , Emily Lancsar PhD , Elisabeth Huynh PhD , Martin Howell PhD , Stavros Petrou PhD , Sarah Smith PhD
Objectives
Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales (PedsQL GCS), comprising 23 items covering 4 subscales (physical, emotional, social, and school functioning), is a widely applied generic measure of childhood health-related quality of life but does not provide health utilities for cost-effectiveness-based decision making. This study aimed to develop a reduced item version of PedsQL GCS amenable to health utility derivation in Australia.
Methods
Data sources were 2 cohorts of the Longitudinal Study of Australian Children, including proxy responses for all PedsQL GCS versions (Toddlers, Young Children, Children, and Teens), and the CheckPoint sample containing child self-report to the Children version. Three analytic samples were CheckPoint sample (n = 1874); Mallinson sample containing 1 measurement per child from one of the Young Children, Children, or Teens versions (n = 7855); and Toddlers sample (n = 7401). Exploratory and confirmatory factor analyses assessed dimensionality. Psychometric analyses used Rasch and classical criteria on 3 randomly selected subsamples (n = 500) per sample. Item selection prioritized psychometric performance in the CheckPoint sample, also considering performance in other samples and conceptual content.
Results
Dimensionality assessments did not generate an alternative empirical structure for the measure, and psychometric analyses were conducted on the original 4 subscales. The selected items were: “Get aches and pains” for physical functioning; “Feel sad/blue” for emotional functioning; “Other kids not friends” for social functioning; and “Keeping up with school work” for school functioning.
Conclusions
The final 4-item set, pending further psychometric validation and valuation, can generate health utilities from the widely used PedsQL GCS to inform cost-effectiveness-based decision making.
目的：儿科生活质量量表（Pediatric Quality of Life InventoryTM Version 4.0 Generic Core Scales，简称 PedsQL GCS）由 23 个项目组成，涵盖四个分量表（身体、情绪、社交和学校功能），是一种广泛应用的儿童健康相关生活质量通用测量方法，但不能为基于成本效益的决策提供健康效用。本研究旨在开发一个可用于澳大利亚健康效用推导的 PedsQL GCS 简化项目版本：数据来源于《澳大利亚儿童纵向研究》的两个队列，包括所有 PedsQL GCS 版本（幼儿、幼儿、儿童、青少年）的代理回复，以及包含儿童版本自我报告的 CheckPoint 样本。三个分析样本分别是CheckPoint 样本（n=1,874）、Mallinson 样本（n=7,855）和幼儿样本（n=7,401）。探索性和确认性因素分析对维度进行了评估。心理测量分析对每个样本随机抽取的三个子样本（样本数=500）采用了 Rasch 和经典标准。项目选择优先考虑在 CheckPoint 样本中的心理测量表现，同时也考虑在其他样本中的表现和概念内容：维度评估没有为测量结果生成其他经验结构，因此对原有的四个子量表进行了心理测量分析。选定的项目有身体机能："疼痛"；情绪机能："悲伤/忧郁"；社会功能："其他孩子不是朋友"；学校功能："跟上学校功课"：最终的四项目集（有待进一步的心理计量验证和评估）可以从广泛使用的儿童生活质量量表 GCS 中生成健康效用，为基于成本效益的决策提供依据。
{"title":"Development of a Health-State Classification System for the Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales for Preference-Based Valuation in Australia","authors":"Joseph Kwon PhD , Rakhee Raghunandan PhD , Son Hong Nghiem PhD , Kirsten Howard PhD , Emily Lancsar PhD , Elisabeth Huynh PhD , Martin Howell PhD , Stavros Petrou PhD , Sarah Smith PhD","doi":"10.1016/j.jval.2024.08.005","DOIUrl":"10.1016/j.jval.2024.08.005","url":null,"abstract":"<div><h3>Objectives</h3><div>Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales (PedsQL GCS), comprising 23 items covering 4 subscales (physical, emotional, social, and school functioning), is a widely applied generic measure of childhood health-related quality of life but does not provide health utilities for cost-effectiveness-based decision making. This study aimed to develop a reduced item version of PedsQL GCS amenable to health utility derivation in Australia.</div></div><div><h3>Methods</h3><div>Data sources were 2 cohorts of the Longitudinal Study of Australian Children, including proxy responses for all PedsQL GCS versions (Toddlers, Young Children, Children, and Teens), and the CheckPoint sample containing child self-report to the Children version. Three analytic samples were CheckPoint sample (<em>n</em> = 1874); Mallinson sample containing 1 measurement per child from one of the Young Children, Children, or Teens versions (<em>n</em> = 7855); and Toddlers sample (<em>n</em> = 7401). Exploratory and confirmatory factor analyses assessed dimensionality. Psychometric analyses used Rasch and classical criteria on 3 randomly selected subsamples (<em>n</em> = 500) per sample. Item selection prioritized psychometric performance in the CheckPoint sample, also considering performance in other samples and conceptual content.</div></div><div><h3>Results</h3><div>Dimensionality assessments did not generate an alternative empirical structure for the measure, and psychometric analyses were conducted on the original 4 subscales. The selected items were: “Get aches and pains” for physical functioning; “Feel sad/blue” for emotional functioning; “Other kids not friends” for social functioning; and “Keeping up with school work” for school functioning.</div></div><div><h3>Conclusions</h3><div>The final 4-item set, pending further psychometric validation and valuation, can generate health utilities from the widely used PedsQL GCS to inform cost-effectiveness-based decision making.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 88-98"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354770","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.11.002
Oriana Ciani PhD, MSc , Claudio Jommi MSc
{"title":"Value Attribution for Combination Treatments: Two Potential Solutions for an Insoluble Problem","authors":"Oriana Ciani PhD, MSc , Claudio Jommi MSc","doi":"10.1016/j.jval.2024.11.002","DOIUrl":"10.1016/j.jval.2024.11.002","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 70-71"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142711057","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.09.014
William Rioux BKin , Dylan Viste BHSc , Stephanie Robertson MBA , Linzi Williamson PhD , Anne Miller MPPA , Evan Poncelet MA , S. Monty Ghosh MD, MSc, MPH, FRCPC
Objectives
The overdose epidemic continues to be one of the leading causes of death in North America and continues to contribute to high healthcare costs. Although harm reduction initiatives have significantly reduced the aforementioned costs, there is a dearth of evidence regarding overdose response hotlines and applications. We aim to evaluate the social return on investment from a payer perspective of one such overdose response hotline, Canada’s National Overdose Response Service, and its implications for service users, service operators, the Canadian healthcare system, and program funders.
Methods
Outcome variables determined from theory of change models were developed in consultation with the aforementioned vested interest groups. Proxy values were attributed to each variable identified through values present within existing literature and databases. These values were then compared with operational costs accounting for deadweight, attribution, and displacement to determine a final social return on investment ratio. A discount rate was then applied based on the influence of risk on the outcome achieved.
Results
The ratio illustrating the value created for all stakeholders, resulting from the $1 592 000 investment made over 2 years, is $15.84 per single dollar invested. The value generated stems primarily from overdose prevention, mental health support, staff employment, reductions in emergency service utilization, service referrals, and volunteer well-being, which outweigh costs including operational funding, work-related stressors, compassion fatigue, and false calls.
Conclusions
The results of our study demonstrate that the National Overdose Response Service provides a social value that far outweighs the costs attributed to the program’s operation.
{"title":"Virtual/Mobile Overdose Response in Canada: A Social Return on Investment Analysis","authors":"William Rioux BKin , Dylan Viste BHSc , Stephanie Robertson MBA , Linzi Williamson PhD , Anne Miller MPPA , Evan Poncelet MA , S. Monty Ghosh MD, MSc, MPH, FRCPC","doi":"10.1016/j.jval.2024.09.014","DOIUrl":"10.1016/j.jval.2024.09.014","url":null,"abstract":"<div><h3>Objectives</h3><div>The overdose epidemic continues to be one of the leading causes of death in North America and continues to contribute to high healthcare costs. Although harm reduction initiatives have significantly reduced the aforementioned costs, there is a dearth of evidence regarding overdose response hotlines and applications. We aim to evaluate the social return on investment from a payer perspective of one such overdose response hotline, Canada’s National Overdose Response Service, and its implications for service users, service operators, the Canadian healthcare system, and program funders.</div></div><div><h3>Methods</h3><div>Outcome variables determined from theory of change models were developed in consultation with the aforementioned vested interest groups. Proxy values were attributed to each variable identified through values present within existing literature and databases. These values were then compared with operational costs accounting for deadweight, attribution, and displacement to determine a final social return on investment ratio. A discount rate was then applied based on the influence of risk on the outcome achieved.</div></div><div><h3>Results</h3><div>The ratio illustrating the value created for all stakeholders, resulting from the $1 592 000 investment made over 2 years, is $15.84 per single dollar invested. The value generated stems primarily from overdose prevention, mental health support, staff employment, reductions in emergency service utilization, service referrals, and volunteer well-being, which outweigh costs including operational funding, work-related stressors, compassion fatigue, and false calls.</div></div><div><h3>Conclusions</h3><div>The results of our study demonstrate that the National Overdose Response Service provides a social value that far outweighs the costs attributed to the program’s operation.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 42-50"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475920","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
To investigate heterogeneity in the cost-effectiveness of high-flow nasal cannula (HFNC) therapy compared with continuous positive airway pressure (CPAP) for acutely ill children requiring noninvasive respiratory support.
Methods
Using data from the First-line Support for Assistance in Breathing in Children trial, we explore heterogeneity at the patient and subgroup levels using 2 causal forest approaches and a seemingly unrelated regression approach for comparison. First-line Support for Assistance in Breathing in Children is a noninferiority randomized controlled trial (ISRCTN60048867) involving 24 UK pediatric intensive care units. The Step-up trial focuses on acutely ill children aged 0 to 15 years, requiring noninvasive respiratory support. A total of 600 children were randomly assigned to HFNC and CPAP groups in a 1:1 allocation ratio, with 94 patients excluded because of data unavailability.
Results
The primary outcome is the incremental net monetary benefit (INB) of HFNC compared with CPAP, using a willingness-to-pay threshold of £20 000 per quality-adjusted life year gain. INB is derived from total costs and quality-adjusted life years at 6 months. Subgroup analysis showed that some subgroups, such as male children, those aged less than 12 months, and those without severe respiratory distress at randomization, had more favorable INB results. Patient-level analysis revealed heterogeneity in INB estimates, particularly driven by the cost component, with greater uncertainty for those with higher INBs.
Conclusions
The estimated overall INB of HFNC is significantly larger for specific patient subgroups, suggesting that the cost-effectiveness of HFNC can be heterogeneous, which highlights the importance of considering patient characteristics in evaluating the cost-effectiveness of HFNC.
{"title":"Exploring Heterogeneity in the Cost-Effectiveness of High-Flow Nasal Cannula Therapy in Acutely Ill Children—Insights From the Step-Up First-line Support for Assistance in Breathing in Children Trial Using a Machine Learning Method","authors":"Zaid Hattab MS , Silvia Moler-Zapata PhD , Edel Doherty PhD , Zia Sadique PhD , Padmanabhan Ramnarayan MD , Stephen O’Neill PhD","doi":"10.1016/j.jval.2024.08.008","DOIUrl":"10.1016/j.jval.2024.08.008","url":null,"abstract":"<div><h3>Objectives</h3><div>To investigate heterogeneity in the cost-effectiveness of high-flow nasal cannula (HFNC) therapy compared with continuous positive airway pressure (CPAP) for acutely ill children requiring noninvasive respiratory support.</div></div><div><h3>Methods</h3><div>Using data from the First-line Support for Assistance in Breathing in Children trial, we explore heterogeneity at the patient and subgroup levels using 2 causal forest approaches and a seemingly unrelated regression approach for comparison. First-line Support for Assistance in Breathing in Children is a noninferiority randomized controlled trial (ISRCTN60048867) involving 24 UK pediatric intensive care units. The Step-up trial focuses on acutely ill children aged 0 to 15 years, requiring noninvasive respiratory support. A total of 600 children were randomly assigned to HFNC and CPAP groups in a 1:1 allocation ratio, with 94 patients excluded because of data unavailability.</div></div><div><h3>Results</h3><div>The primary outcome is the incremental net monetary benefit (INB) of HFNC compared with CPAP, using a willingness-to-pay threshold of £20 000 per quality-adjusted life year gain. INB is derived from total costs and quality-adjusted life years at 6 months. Subgroup analysis showed that some subgroups, such as male children, those aged less than 12 months, and those without severe respiratory distress at randomization, had more favorable INB results. Patient-level analysis revealed heterogeneity in INB estimates, particularly driven by the cost component, with greater uncertainty for those with higher INBs.</div></div><div><h3>Conclusions</h3><div>The estimated overall INB of HFNC is significantly larger for specific patient subgroups, suggesting that the cost-effectiveness of HFNC can be heterogeneous, which highlights the importance of considering patient characteristics in evaluating the cost-effectiveness of HFNC.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 60-69"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.08.009
Jake Hitch MRes , Tom Denee MScBA , Simon Brassel MSc , Jennifer Lee MBA , Michel Michaelides MD, FRCOphth , Jacob Petersen MSc , Sarah Alulis MPH , Lotte Steuten PhD
Objectives
X-linked retinitis pigmentosa (XLRP) is a rare inherited retinal disease with no available treatment. Gene therapies in clinical trials will pose challenges for health technology assessment (HTA) if found to be safe and effective. We evaluated 2 of these challenges, namely acceptability and difficulties in assessing value beyond short-term patient health and healthcare savings and discounting in economic evaluation.
Methods
We conducted a narrative literature review on the socioeconomic burden of XLRP to identify relevant components of value for a hypothetical gene therapy from a societal perspective and to assess their relative importance. We compared the resulting value profile against the value frameworks of three European HTA agencies. We also reviewed their guidelines on discounting and potential discounting issues specific to XLRP.
Results
Much of the societal value of an XLRP gene therapy is likely to originate from productivity effects, carer spillovers, and value elements related to patient uncertainty. The evidence on these effects, however, is often limited, making it difficult for HTA agencies to assess them. Cost-effectiveness results are likely to be highly sensitive to the discount rate, and discounting will compound the effects of omitting important sources of value.
Conclusions
We have identified and detailed important components of societal value, key evidence gaps, and potential discounting issues for an XLRP gene therapy, which can inform future value assessments. Many of these may apply to gene therapies in other disease areas. Revisiting existing HTA approaches is recommended to ensure these are fit for purpose for such new classes of treatment.
{"title":"Challenges in Value Assessment for One-Time Gene Therapies for Inherited Retinal Diseases: Are We Turning a Blind Eye?","authors":"Jake Hitch MRes , Tom Denee MScBA , Simon Brassel MSc , Jennifer Lee MBA , Michel Michaelides MD, FRCOphth , Jacob Petersen MSc , Sarah Alulis MPH , Lotte Steuten PhD","doi":"10.1016/j.jval.2024.08.009","DOIUrl":"10.1016/j.jval.2024.08.009","url":null,"abstract":"<div><h3>Objectives</h3><div>X-linked retinitis pigmentosa (XLRP) is a rare inherited retinal disease with no available treatment. Gene therapies in clinical trials will pose challenges for health technology assessment (HTA) if found to be safe and effective. We evaluated 2 of these challenges, namely acceptability and difficulties in assessing value beyond short-term patient health and healthcare savings and discounting in economic evaluation.</div></div><div><h3>Methods</h3><div>We conducted a narrative literature review on the socioeconomic burden of XLRP to identify relevant components of value for a hypothetical gene therapy from a societal perspective and to assess their relative importance. We compared the resulting value profile against the value frameworks of three European HTA agencies. We also reviewed their guidelines on discounting and potential discounting issues specific to XLRP.</div></div><div><h3>Results</h3><div>Much of the societal value of an XLRP gene therapy is likely to originate from productivity effects, carer spillovers, and value elements related to patient uncertainty. The evidence on these effects, however, is often limited, making it difficult for HTA agencies to assess them. Cost-effectiveness results are likely to be highly sensitive to the discount rate, and discounting will compound the effects of omitting important sources of value.</div></div><div><h3>Conclusions</h3><div>We have identified and detailed important components of societal value, key evidence gaps, and potential discounting issues for an XLRP gene therapy, which can inform future value assessments. Many of these may apply to gene therapies in other disease areas. Revisiting existing HTA approaches is recommended to ensure these are fit for purpose for such new classes of treatment.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 116-124"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142393660","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-01DOI: 10.1016/j.jval.2024.08.002
Milou A. Hogervorst PharmD, PhD , Kanaka V. Soman MSc , Helga Gardarsdottir PharmD, PhD , Wim G. Goettsch PhD , Lourens T. Bloem PharmD, PhD
Objectives
This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.
Methods
A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).
Results
Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD–based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.
Conclusions
Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.
{"title":"Analytical Methods for Comparing Uncontrolled Trials With External Controls From Real-World Data: A Systematic Literature Review and Comparison With European Regulatory and Health Technology Assessment Practice","authors":"Milou A. Hogervorst PharmD, PhD , Kanaka V. Soman MSc , Helga Gardarsdottir PharmD, PhD , Wim G. Goettsch PhD , Lourens T. Bloem PharmD, PhD","doi":"10.1016/j.jval.2024.08.002","DOIUrl":"10.1016/j.jval.2024.08.002","url":null,"abstract":"<div><h3>Objectives</h3><div>This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.</div></div><div><h3>Methods</h3><div>A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).</div></div><div><h3>Results</h3><div>Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD–based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.</div></div><div><h3>Conclusions</h3><div>Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 1","pages":"Pages 161-174"},"PeriodicalIF":4.9,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142146419","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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