Pub Date : 2024-07-01Epub Date: 2024-05-16DOI: 10.1007/s40258-024-00887-z
Stuart J Wright, Ewan Gray, Gabriel Rogers, Anna Donten, Katherine Payne
Background: Decision-makers require knowledge of the strengths and weaknesses of decision-analytic models used to evaluate healthcare interventions to be able to confidently use the results of such models to inform policy. A number of aspects of model validity have previously been described, but no systematic approach to assessing the validity of a model has been proposed. This study aimed to consolidate the different aspects of model validity into a step-by-step approach to assessing the strengths and weaknesses of a decision-analytic model.
Methods: A pre-defined set of steps were used to conduct the validation process of an exemplar early decision-analytic-model-based cost-effectiveness analysis of a risk-stratified national breast cancer screening programme [UK healthcare perspective; lifetime horizon; costs (£; 2021)]. Internal validation was assessed in terms of descriptive validity, technical validity and face validity. External validation was assessed in terms of operational validation, convergent validity (or corroboration) and predictive validity.
Results: The results outline the findings of each step of internal and external validation of the early decision-analytic-model and present the validated model (called 'MANC-RISK-SCREEN'). The positive aspects in terms of meeting internal validation requirements are shown together with the remaining limitations of MANC-RISK-SCREEN.
Conclusion: Following a transparent and structured validation process, MANC-RISK-SCREEN has been shown to have satisfactory internal and external validity for use in informing resource allocation decision-making. We suggest that MANC-RISK-SCREEN can be used to assess the cost-effectiveness of exemplars of risk-stratified national breast cancer screening programmes (NBSP) from the UK perspective.
Implications: A step-by-step process for conducting the validation of a decision-analytic model was developed for future use by health economists. Using this approach may help researchers to fully demonstrate the strengths and limitations of their model to decision-makers.
{"title":"A structured process for the validation of a decision-analytic model: application to a cost-effectiveness model for risk-stratified national breast screening.","authors":"Stuart J Wright, Ewan Gray, Gabriel Rogers, Anna Donten, Katherine Payne","doi":"10.1007/s40258-024-00887-z","DOIUrl":"10.1007/s40258-024-00887-z","url":null,"abstract":"<p><strong>Background: </strong>Decision-makers require knowledge of the strengths and weaknesses of decision-analytic models used to evaluate healthcare interventions to be able to confidently use the results of such models to inform policy. A number of aspects of model validity have previously been described, but no systematic approach to assessing the validity of a model has been proposed. This study aimed to consolidate the different aspects of model validity into a step-by-step approach to assessing the strengths and weaknesses of a decision-analytic model.</p><p><strong>Methods: </strong>A pre-defined set of steps were used to conduct the validation process of an exemplar early decision-analytic-model-based cost-effectiveness analysis of a risk-stratified national breast cancer screening programme [UK healthcare perspective; lifetime horizon; costs (£; 2021)]. Internal validation was assessed in terms of descriptive validity, technical validity and face validity. External validation was assessed in terms of operational validation, convergent validity (or corroboration) and predictive validity.</p><p><strong>Results: </strong>The results outline the findings of each step of internal and external validation of the early decision-analytic-model and present the validated model (called 'MANC-RISK-SCREEN'). The positive aspects in terms of meeting internal validation requirements are shown together with the remaining limitations of MANC-RISK-SCREEN.</p><p><strong>Conclusion: </strong>Following a transparent and structured validation process, MANC-RISK-SCREEN has been shown to have satisfactory internal and external validity for use in informing resource allocation decision-making. We suggest that MANC-RISK-SCREEN can be used to assess the cost-effectiveness of exemplars of risk-stratified national breast cancer screening programmes (NBSP) from the UK perspective.</p><p><strong>Implications: </strong>A step-by-step process for conducting the validation of a decision-analytic model was developed for future use by health economists. Using this approach may help researchers to fully demonstrate the strengths and limitations of their model to decision-makers.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178649/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140954809","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-01Epub Date: 2024-03-26DOI: 10.1007/s40258-024-00879-z
Muhammad Iftikhar Ul Husnain, Mohammad Hajizadeh, Hasnat Ahmad, Rasheda Khanam
Background: Psychological distress (PD) is a major health problem that affects all aspects of health-related quality of life including physical, mental and social health, leading to a substantial human and economic burden. Studies have revealed a concerning rise in the prevalence of PD and various mental health conditions among Australians, particularly in female individuals. There is a scarcity of studies that estimate health state utilities (HSUs), which reflect the overall health-related quality of life in individuals with PD. No such studies have been conducted in Australia thus far.
Objective: We aimed to evaluate the age-specific, sex-specific and PD category-specific HSUs (disutilities) in Australian adults with PD to inform healthcare decision making in the management of PD.
Methods: Data on age, sex, SF-36/SF6D responses, Kessler psychological distress (K10) scale scores and other characteristics of N = 15,139 participants (n = 8149 female individuals) aged >15 years were derived from the latest wave (21) of the nationally representative Household, Income and Labor Dynamics in Australia survey. Participants were grouped into the severity categories of no (K10 score: 10-19), mild (K10: 20-24), moderate (K10: 25-29) and severe PD (K10: 30-50). Both crude and adjusted HSUs were calculated from participants' SF-36 profiles, considering potential confounders such as smoking, marital status, remoteness, education and income levels. The calculations were based on the SF-6D algorithm and aligned with Australian population norms. Additionally, the HSUs were stratified by age, sex and PD categories. Disutilities of PD, representing the mean difference between HSUs of people with PD and those without, were also calculated for each group.
Results: The average age of individuals was 46.130 years (46% male), and 31% experienced PD in the last 4 weeks. Overall, individuals with PD had significantly lower mean HSUs than those likely to be no PD, 0.637 (95% confidence interval [CI] 0.636, 0.640) vs 0.776 (95% CI 0.775, 0.777) i.e. disutility: -0.139 [95% CI -0.139, -0.138]). Mean disutilities of -0.108 (95% CI -0.110, -0.104), -0.140 (95% CI -0.142, -0.138), and -0.188 (95% CI -0.190, -0.187) were observed for mild PD, moderate PD and severe PD, respectively. Disutilities of PD also differed by age and sex groups. For instance, female individuals had up to 0.049 points lower mean HSUs than male individuals across the three classifications of PD. There was a clear decline in health-related quality of life with increasing age, demonstrated by lower mean HSUs in older population age groups, that ranged from 0.818 (95% CI 0.817, 0.818) for the 15-24 years age group with no PD to 0.496 (95% CI 0.491, 0.500) for the 65+ years age group with severe PD). Across all ages and genders, respondents were more likely to report issues in certain dimensions, notably vitality, and th
背景:心理困扰(PD)是一个重大的健康问题,它影响着与健康相关的生活质量的方方面面,包括身体、心理和社会健康,造成了巨大的人力和经济负担。研究表明,澳大利亚人,尤其是女性,心理困扰和各种心理健康问题的发病率呈上升趋势,令人担忧。很少有研究对健康状况效用(HSU)进行估算,而健康状况效用反映了帕金森病患者与健康相关的整体生活质量。迄今为止,澳大利亚尚未开展过此类研究:我们旨在评估澳大利亚帕金森氏症成人患者的年龄特异性、性别特异性和帕金森氏症类别特异性健康状态效用(disutilities),为帕金森氏症管理的医疗决策提供参考:年龄大于15岁的15139名参与者(女性8149人)的年龄、性别、SF-36/SF6D反应、凯斯勒心理压力量表(K10)评分和其他特征数据均来自最新一期(21)具有全国代表性的澳大利亚家庭、收入和劳动力动态调查。参与者被分为无(K10 评分:10-19 分)、轻度(K10:20-24 分)、中度(K10:25-29 分)和重度 PD(K10:30-50 分)等严重程度类别。根据参与者的 SF-36 资料计算出粗略和调整后的 HSU,并考虑到吸烟、婚姻状况、偏远地区、教育程度和收入水平等潜在混杂因素。计算以 SF-6D 算法为基础,并与澳大利亚人口标准保持一致。此外,HSU 还按年龄、性别和 PD 类别进行了分层。此外,还计算了每个群体的帕金森病差异,即帕金森病患者与非帕金森病患者的 HSUs 平均差异:患者的平均年龄为 46.130 岁(46% 为男性),31% 的患者在过去 4 周内曾患过帕金森病。总体而言,患有帕金森氏症的人的平均 HSUs 明显低于可能没有帕金森氏症的人,分别为 0.637(95% 置信区间 [CI] 0.636,0.640) vs 0.776(95% CI 0.775,0.777),即效用差:-0.139 [95% CI -0.139,-0.138])。轻度帕金森病、中度帕金森病和重度帕金森病的平均效用分别为-0.108(95% CI -0.110,-0.104)、-0.140(95% CI -0.142,-0.138)和-0.188(95% CI -0.190,-0.187)。不同年龄组和性别组的人患帕金森病的几率也不同。例如,在帕金森病的三种分类中,女性的平均健康相关指数比男性低 0.049 点。随着年龄的增长,与健康相关的生活质量明显下降,这表现在老年人群的平均 HSU 值较低,从 15-24 岁未患帕金森病年龄组的 0.818(95% CI 0.817,0.818)到 65 岁以上严重帕金森病年龄组的 0.496(95% CI 0.491,0.500)不等。)在所有年龄段和性别中,受访者更倾向于报告某些方面的问题,尤其是活力问题,而这些问题与年龄的关系并不一致:在澳大利亚,老年痴呆症造成的负担非常沉重,对女性和老年人的影响很大。实施针对不同年龄和性别的医疗保健干预措施来解决澳大利亚成年人的老年痴呆症问题,可以大大减轻这一负担。在我们的研究中计算出的针对各州的帕金森病 HSUs 可作为未来对澳大利亚和类似人群的帕金森病进行健康经济评估的重要依据。
{"title":"The Hidden Toll of Psychological Distress in Australian Adults and Its Impact on Health-Related Quality of Life Measured as Health State Utilities.","authors":"Muhammad Iftikhar Ul Husnain, Mohammad Hajizadeh, Hasnat Ahmad, Rasheda Khanam","doi":"10.1007/s40258-024-00879-z","DOIUrl":"10.1007/s40258-024-00879-z","url":null,"abstract":"<p><strong>Background: </strong>Psychological distress (PD) is a major health problem that affects all aspects of health-related quality of life including physical, mental and social health, leading to a substantial human and economic burden. Studies have revealed a concerning rise in the prevalence of PD and various mental health conditions among Australians, particularly in female individuals. There is a scarcity of studies that estimate health state utilities (HSUs), which reflect the overall health-related quality of life in individuals with PD. No such studies have been conducted in Australia thus far.</p><p><strong>Objective: </strong>We aimed to evaluate the age-specific, sex-specific and PD category-specific HSUs (disutilities) in Australian adults with PD to inform healthcare decision making in the management of PD.</p><p><strong>Methods: </strong>Data on age, sex, SF-36/SF6D responses, Kessler psychological distress (K10) scale scores and other characteristics of N = 15,139 participants (n = 8149 female individuals) aged >15 years were derived from the latest wave (21) of the nationally representative Household, Income and Labor Dynamics in Australia survey. Participants were grouped into the severity categories of no (K10 score: 10-19), mild (K10: 20-24), moderate (K10: 25-29) and severe PD (K10: 30-50). Both crude and adjusted HSUs were calculated from participants' SF-36 profiles, considering potential confounders such as smoking, marital status, remoteness, education and income levels. The calculations were based on the SF-6D algorithm and aligned with Australian population norms. Additionally, the HSUs were stratified by age, sex and PD categories. Disutilities of PD, representing the mean difference between HSUs of people with PD and those without, were also calculated for each group.</p><p><strong>Results: </strong>The average age of individuals was 46.130 years (46% male), and 31% experienced PD in the last 4 weeks. Overall, individuals with PD had significantly lower mean HSUs than those likely to be no PD, 0.637 (95% confidence interval [CI] 0.636, 0.640) vs 0.776 (95% CI 0.775, 0.777) i.e. disutility: -0.139 [95% CI -0.139, -0.138]). Mean disutilities of -0.108 (95% CI -0.110, -0.104), -0.140 (95% CI -0.142, -0.138), and -0.188 (95% CI -0.190, -0.187) were observed for mild PD, moderate PD and severe PD, respectively. Disutilities of PD also differed by age and sex groups. For instance, female individuals had up to 0.049 points lower mean HSUs than male individuals across the three classifications of PD. There was a clear decline in health-related quality of life with increasing age, demonstrated by lower mean HSUs in older population age groups, that ranged from 0.818 (95% CI 0.817, 0.818) for the 15-24 years age group with no PD to 0.496 (95% CI 0.491, 0.500) for the 65+ years age group with severe PD). Across all ages and genders, respondents were more likely to report issues in certain dimensions, notably vitality, and th","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178635/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140292570","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background: Survival outcomes for multiple myeloma have improved dramatically since the introduction of novel therapeutic agents. While these drugs are highly effective in improving survival outcomes and quality of life in patients with multiple myeloma, they come at a significant cost. We assessed the cost-effectiveness of bortezomib-based triplet or quadruplet drug regimens in isolation and followed by autologous hematopoietic stem cell transplantation (AHSCT) for the treatment of newly diagnosed multiple myeloma (NDMM) in the Indian context.
Methods: A Markov model was developed to assess the health and economic outcomes of novel drug regimens with and without AHSCT for the treatment of NDMM in India. We estimated the lifetime quality-adjusted life-years (QALYs) and costs in each scenario. The incremental cost-effectiveness ratios (ICERs) were computed and compared against the current willingness-to-pay threshold of a one-time per capita gross domestic product of ₹146,890 (US$1,927.70) for India. Parameter uncertainty was assessed through Monte Carlo probabilistic sensitivity analysis.
Results: Among seven treatment sequences, the VCd (bortezomib, cyclophosphamide, dexamethasone) alone arm has the lowest cost and health benefits as compared to four treatment sequences, namely VTd (bortezomib, thalidomide, dexamethasone) alone, VRd (bortezomib, lenalidomide, dexamethasone) alone, VRd plus AHSCT and DVRd (Daratumumab, bortezomib, lenalidomide, dexamethasone) plus AHSCT. It was found that VTd plus AHSCT and VCd plus AHSCT arms were extendedly dominated (ED) by combination of two alternative treatments. Among the five non-dominated strategies, VRd has a lowest incremental cost of ₹ 2,20,093 (US$2,888) per QALY gained compared to VTd alone followed by VRd plus AHSCT [₹3,14,530 (US$4,128) per QALY gained] in comparison to VRd alone. None of the novel treatment sequences were found to be cost-effective at the current WTP threshold of ₹1,46,890 (US$1,927.7).
Conclusion: At the current WTP threshold of one-time per capita GDP (₹ 146,890) of India, VRd alone and VRd plus AHSCT has 38.1% and 6.9% probability to be cost-effective, respectively. Reduction in current reimbursement rates of novel drugs, namely VRd, lenalidomide, and pomalidomide plus dexamethasone under national insurance program and societal cost of transplant by 50%, would make VRd plus AHSCT and VTd plus AHSCT cost-effective at an incremental cost of ₹40,671 (US$34) and ₹97,639 (US$1,281) per QALY gained, respectively.
{"title":"Cost-Effectiveness of Novel Agent Regimens for Transplant-Eligible Newly Diagnosed Multiple Myeloma Patients in India.","authors":"Jyoti Dixit, Pankaj Malhotra, Nikita Mehra, Anisha Mathew, Lalit Kumar, Ashish Singh, Nidhi Gupta, Manjunath Nookala Krishnamurthy, Partha Sarathi Roy, Amal Chandra Kataki, Sudeep Gupta, Shankar Prinja","doi":"10.1007/s40258-024-00877-1","DOIUrl":"10.1007/s40258-024-00877-1","url":null,"abstract":"<p><strong>Background: </strong>Survival outcomes for multiple myeloma have improved dramatically since the introduction of novel therapeutic agents. While these drugs are highly effective in improving survival outcomes and quality of life in patients with multiple myeloma, they come at a significant cost. We assessed the cost-effectiveness of bortezomib-based triplet or quadruplet drug regimens in isolation and followed by autologous hematopoietic stem cell transplantation (AHSCT) for the treatment of newly diagnosed multiple myeloma (NDMM) in the Indian context.</p><p><strong>Methods: </strong>A Markov model was developed to assess the health and economic outcomes of novel drug regimens with and without AHSCT for the treatment of NDMM in India. We estimated the lifetime quality-adjusted life-years (QALYs) and costs in each scenario. The incremental cost-effectiveness ratios (ICERs) were computed and compared against the current willingness-to-pay threshold of a one-time per capita gross domestic product of ₹146,890 (US$1,927.70) for India. Parameter uncertainty was assessed through Monte Carlo probabilistic sensitivity analysis.</p><p><strong>Results: </strong>Among seven treatment sequences, the VCd (bortezomib, cyclophosphamide, dexamethasone) alone arm has the lowest cost and health benefits as compared to four treatment sequences, namely VTd (bortezomib, thalidomide, dexamethasone) alone, VRd (bortezomib, lenalidomide, dexamethasone) alone, VRd plus AHSCT and DVRd (Daratumumab, bortezomib, lenalidomide, dexamethasone) plus AHSCT. It was found that VTd plus AHSCT and VCd plus AHSCT arms were extendedly dominated (ED) by combination of two alternative treatments. Among the five non-dominated strategies, VRd has a lowest incremental cost of ₹ 2,20,093 (US$2,888) per QALY gained compared to VTd alone followed by VRd plus AHSCT [₹3,14,530 (US$4,128) per QALY gained] in comparison to VRd alone. None of the novel treatment sequences were found to be cost-effective at the current WTP threshold of ₹1,46,890 (US$1,927.7).</p><p><strong>Conclusion: </strong>At the current WTP threshold of one-time per capita GDP (₹ 146,890) of India, VRd alone and VRd plus AHSCT has 38.1% and 6.9% probability to be cost-effective, respectively. Reduction in current reimbursement rates of novel drugs, namely VRd, lenalidomide, and pomalidomide plus dexamethasone under national insurance program and societal cost of transplant by 50%, would make VRd plus AHSCT and VTd plus AHSCT cost-effective at an incremental cost of ₹40,671 (US$34) and ₹97,639 (US$1,281) per QALY gained, respectively.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140048587","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Introduction: Rheumatoid arthritis (RA) is a progressive and debilitating disease, causing persistent joint pain that limits daily activities requiring long-term treatment. Newer targeted therapies expand RA treatment options, but their high cost necessitates a focus on cost effectiveness. To address this, we aim to conduct a cost-utility analysis of these newer RA pharmacotherapies to support evidence-based policy decision-making.
Methods: We analyzed the cost-utility of sequential treatment with TNF-α, B cell and JAK-inhibitors compared with conventional synthetic disease-modifying antirheumatic drugs (csDMARDs) for RA treatment in methotrexate (MTX) nonresponders. We used a Markov model with lifetime horizon and 6-month cycles from an Indian health system perspective. Costs (INR 2022) and quality-adjusted life years (QALYs) were used to determine the incremental cost-effectiveness ratios (ICERs) at a cost-effectiveness threshold of India's gross domestic product (GDP) per capita (2022). We assessed uncertainty using univariate, probabilistic sensitivity, and scenario analyses.
Results: Despite additional QALYs, TNF-α, B cell, and JAK inhibitors were not cost-effective for treating moderate-to-severe patients with RA unresponsive to csDMARDs (including MTX) in India, as increased costs outweighed their clinical benefits. ICERs ranged from 10,46,206 to 31,09,207 Indian Rupees in the base case analysis, exceeding three times India's GDP per-capita [approximately USD $13,287 to $39,487 and GBP £10,776 to £32,025]. Sensitivity analyses confirmed the results' robustness. Scenario analysis suggested that a cost reduction of over 75% in drug prices could make most of the interventions cost effective compared with csDMARDs.
Conclusions: TNF-α, B cell, and JAK-inhibitors are not cost-effective compared with csDMARDs for patients with RA who have not responded to MTX in India at the current prices. Cost-effectiveness estimates were highly influenced by drug pricing variations. Therefore, reducing the prices of these interventions could enhance affordability, potentially leading to their inclusion in publicly funded health programs.
导言:类风湿性关节炎(RA)是一种渐进性衰弱疾病,会引起持续性关节疼痛,限制日常活动,需要长期治疗。较新的靶向疗法扩大了类风湿关节炎的治疗选择,但其高昂的费用使人们必须关注其成本效益。为此,我们旨在对这些较新的 RA 药物疗法进行成本效用分析,以支持循证政策决策:我们分析了 TNF-α、B 细胞和 JAK 抑制剂与传统合成改善病情抗风湿药(csDMARDs)序贯治疗甲氨蝶呤(MTX)无应答者 RA 的成本效用比较。我们从印度卫生系统的角度出发,使用了一个终身视角和 6 个月周期的马尔可夫模型。成本(2022 年印度卢比)和质量调整生命年(QALYs)用于确定印度人均国内生产总值(GDP)成本效益阈值(2022 年)下的增量成本效益比(ICERs)。我们通过单变量分析、概率敏感性分析和情景分析评估了不确定性:尽管增加了QALY,但在印度,TNF-α、B细胞和JAK抑制剂治疗对csDMARDs(包括MTX)无反应的中重度RA患者并不划算,因为增加的成本超过了其临床疗效。在基础病例分析中,ICER 为 10,46,206 至 31,09,207 印度卢比,超过印度人均 GDP 的三倍[约为 13,287 美元至 39,487 美元,10,776 英镑至 32,025 英镑]。敏感性分析证实了结果的稳健性。情景分析表明,与csDMARDs相比,药品价格降低75%以上可使大多数干预措施具有成本效益:结论:在印度,对于对MTX治疗无效的RA患者,以目前的价格计算,TNF-α、B细胞和JAK抑制剂与csDMARDs相比不具成本效益。成本效益估计值受药物价格变化的影响很大。因此,降低这些干预措施的价格可以提高患者的负担能力,从而有可能将其纳入公共资助的医疗计划。
{"title":"Cost-Utility Analysis of TNF-α Inhibitors, B Cell Inhibitors, and JAK Inhibitors Versus csDMARDs for Rheumatoid Arthritis Treatment.","authors":"Madhumitha Haridoss, Akhil Sasidharan, Sajith Kumar, Kavitha Rajsekar, Krishnamurthy Venkataraman, Bhavani Shankara Bagepally","doi":"10.1007/s40258-024-00898-w","DOIUrl":"https://doi.org/10.1007/s40258-024-00898-w","url":null,"abstract":"<p><strong>Introduction: </strong>Rheumatoid arthritis (RA) is a progressive and debilitating disease, causing persistent joint pain that limits daily activities requiring long-term treatment. Newer targeted therapies expand RA treatment options, but their high cost necessitates a focus on cost effectiveness. To address this, we aim to conduct a cost-utility analysis of these newer RA pharmacotherapies to support evidence-based policy decision-making.</p><p><strong>Methods: </strong>We analyzed the cost-utility of sequential treatment with TNF-α, B cell and JAK-inhibitors compared with conventional synthetic disease-modifying antirheumatic drugs (csDMARDs) for RA treatment in methotrexate (MTX) nonresponders. We used a Markov model with lifetime horizon and 6-month cycles from an Indian health system perspective. Costs (INR 2022) and quality-adjusted life years (QALYs) were used to determine the incremental cost-effectiveness ratios (ICERs) at a cost-effectiveness threshold of India's gross domestic product (GDP) per capita (2022). We assessed uncertainty using univariate, probabilistic sensitivity, and scenario analyses.</p><p><strong>Results: </strong>Despite additional QALYs, TNF-α, B cell, and JAK inhibitors were not cost-effective for treating moderate-to-severe patients with RA unresponsive to csDMARDs (including MTX) in India, as increased costs outweighed their clinical benefits. ICERs ranged from 10,46,206 to 31,09,207 Indian Rupees in the base case analysis, exceeding three times India's GDP per-capita [approximately USD $13,287 to $39,487 and GBP £10,776 to £32,025]. Sensitivity analyses confirmed the results' robustness. Scenario analysis suggested that a cost reduction of over 75% in drug prices could make most of the interventions cost effective compared with csDMARDs.</p><p><strong>Conclusions: </strong>TNF-α, B cell, and JAK-inhibitors are not cost-effective compared with csDMARDs for patients with RA who have not responded to MTX in India at the current prices. Cost-effectiveness estimates were highly influenced by drug pricing variations. Therefore, reducing the prices of these interventions could enhance affordability, potentially leading to their inclusion in publicly funded health programs.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141475768","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Background and objective: Imaging with low or no benefit for the patient undermines the quality of care and amounts to vast opportunity costs. More than 3.6 billion imaging examinations are performed annually, and about 20-50% of these are of low value. This study aimed to synthesize knowledge of the costs of low-value imaging worldwide.
Methods: This systematic review was based on the PRISMA statement. The database search was developed in Medline and further adapted to Embase-Ovid, Cochrane Library, and Scopus. Primary empirical studies assessing the costs of low-value diagnostic imaging were included if published between 2012 and March 2022. Studies designed as randomized controlled trials, non-randomized trials, cohort studies, cross-sectional studies, descriptive studies, cost analysis, cost-effectiveness analysis, and mixed-methods studies were eligible. The analysis was descriptive.
Results: Of 5,567 records identified, 106 were included. Most of the studies included were conducted in the USA (n = 76), and a hospital or medical center was the most common setting (n = 82). Thirty-eight of the included studies calculated the costs of multiple imaging modalities; in studies with only one imaging modality included, conventional radiography was the most common (n = 32). Aggregated costs for low-value examinations amounts to billions of dollars per year globally. Initiatives to reduce low-value imaging may reduce costs by up to 95% without harming patients.
Conclusions: This study is the first systematic review of the cost of low-value imaging worldwide, documenting a high potential for cost reduction. Given the universal challenges with resource allocation, the large amount used for low-value imaging represents a vast opportunity cost and offers great potential to improve the quality and efficiency of care.
{"title":"Cost of Low-Value Imaging Worldwide: A Systematic Review.","authors":"Elin Kjelle, Ingrid Øfsti Brandsæter, Eivind Richter Andersen, Bjørn Morten Hofmann","doi":"10.1007/s40258-024-00876-2","DOIUrl":"10.1007/s40258-024-00876-2","url":null,"abstract":"<p><strong>Background and objective: </strong>Imaging with low or no benefit for the patient undermines the quality of care and amounts to vast opportunity costs. More than 3.6 billion imaging examinations are performed annually, and about 20-50% of these are of low value. This study aimed to synthesize knowledge of the costs of low-value imaging worldwide.</p><p><strong>Methods: </strong>This systematic review was based on the PRISMA statement. The database search was developed in Medline and further adapted to Embase-Ovid, Cochrane Library, and Scopus. Primary empirical studies assessing the costs of low-value diagnostic imaging were included if published between 2012 and March 2022. Studies designed as randomized controlled trials, non-randomized trials, cohort studies, cross-sectional studies, descriptive studies, cost analysis, cost-effectiveness analysis, and mixed-methods studies were eligible. The analysis was descriptive.</p><p><strong>Results: </strong>Of 5,567 records identified, 106 were included. Most of the studies included were conducted in the USA (n = 76), and a hospital or medical center was the most common setting (n = 82). Thirty-eight of the included studies calculated the costs of multiple imaging modalities; in studies with only one imaging modality included, conventional radiography was the most common (n = 32). Aggregated costs for low-value examinations amounts to billions of dollars per year globally. Initiatives to reduce low-value imaging may reduce costs by up to 95% without harming patients.</p><p><strong>Conclusions: </strong>This study is the first systematic review of the cost of low-value imaging worldwide, documenting a high potential for cost reduction. Given the universal challenges with resource allocation, the large amount used for low-value imaging represents a vast opportunity cost and offers great potential to improve the quality and efficiency of care.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178636/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139995345","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-01Epub Date: 2024-05-17DOI: 10.1007/s40258-024-00886-0
Chiara Malmberg, Magnus Värendh, Patric Berling, Mata Charokopou, Erik Eklund
Objective: This study evaluated, in a Swedish setting, the cost effectiveness of fenfluramine (FFA) as an add-on to standard of care (SoC) for reducing seizure frequency in Dravet syndrome, a severe developmental epileptic encephalopathy.
Methods: Cost effectiveness of FFA+SoC compared with SoC only was evaluated using a patient-level simulation model with a lifetime horizon. Patient characteristics and treatment effects, including convulsive seizures, seizure-free days and mortality, were derived from FFA clinical trials. Resource use and costs included cost of drug acquisition, routine care and monitoring, as well as ongoing and emergency resources. Quality of life (QoL) estimates for patients and their caregivers were derived from clinical trial data. Robustness was evaluated by one-way sensitivity analysis, probabilistic sensitivity analysis and scenario analyses.
Results: Lifetime cost of FFA+SoC was ~3 million SEK per patient compared with ~1.5 million SEK for SoC only. FFA+SoC generated 15% more QALYs than SoC only (21.2 vs 18.5 over a lifetime), resulting in an incremental cost-effectiveness ratio (ICER) of ~540,000 SEK. Moreover, FFA+SoC had a higher probability of being cost effective than SoC only from a willingness-to-pay threshold of 710,000 SEK. Results remained generally consistent across scenario analyses, with only few exceptions (exclusions of carer utility or FFA effect on sudden unexpected death in epilepsy).
Conclusion: Due to better seizure control, FFA is a clinically meaningful add-on therapy and was estimated to be a cost-effective addition to current SoC for patients with this rare disease in Sweden at a willingness-to-pay threshold of 1,000,000 SEK.
{"title":"Cost Effectiveness of Adding Fenfluramine to Standard of Care for Patients with Dravet Syndrome in Sweden.","authors":"Chiara Malmberg, Magnus Värendh, Patric Berling, Mata Charokopou, Erik Eklund","doi":"10.1007/s40258-024-00886-0","DOIUrl":"10.1007/s40258-024-00886-0","url":null,"abstract":"<p><strong>Objective: </strong>This study evaluated, in a Swedish setting, the cost effectiveness of fenfluramine (FFA) as an add-on to standard of care (SoC) for reducing seizure frequency in Dravet syndrome, a severe developmental epileptic encephalopathy.</p><p><strong>Methods: </strong>Cost effectiveness of FFA+SoC compared with SoC only was evaluated using a patient-level simulation model with a lifetime horizon. Patient characteristics and treatment effects, including convulsive seizures, seizure-free days and mortality, were derived from FFA clinical trials. Resource use and costs included cost of drug acquisition, routine care and monitoring, as well as ongoing and emergency resources. Quality of life (QoL) estimates for patients and their caregivers were derived from clinical trial data. Robustness was evaluated by one-way sensitivity analysis, probabilistic sensitivity analysis and scenario analyses.</p><p><strong>Results: </strong>Lifetime cost of FFA+SoC was ~3 million SEK per patient compared with ~1.5 million SEK for SoC only. FFA+SoC generated 15% more QALYs than SoC only (21.2 vs 18.5 over a lifetime), resulting in an incremental cost-effectiveness ratio (ICER) of ~540,000 SEK. Moreover, FFA+SoC had a higher probability of being cost effective than SoC only from a willingness-to-pay threshold of 710,000 SEK. Results remained generally consistent across scenario analyses, with only few exceptions (exclusions of carer utility or FFA effect on sudden unexpected death in epilepsy).</p><p><strong>Conclusion: </strong>Due to better seizure control, FFA is a clinically meaningful add-on therapy and was estimated to be a cost-effective addition to current SoC for patients with this rare disease in Sweden at a willingness-to-pay threshold of 1,000,000 SEK.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140954995","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-01Epub Date: 2024-03-01DOI: 10.1007/s40258-023-00863-z
Davene R Wright, Mikaela Batista, Tim Wrightson
Questions regarding the effectiveness and safety of health interventions and allocation of health care resources are frequently discussed in mainstream and social media. Additionally, government and foundation funders are increasingly mandating that results be disseminated to the lay public and patients may benefit from being able to digest scientific research regarding their health conditions. Therefore, it is important to widely disseminate and clearly communication health economics and outcomes research (HEOR) findings to a range of interested parties. Digital media features such as graphical or visual abstracts, infographics and videos are informative and add value to research articles by improving reader engagement with articles, potentially increasing their impact, and allowing results to be more widely disseminated. However, use of novel digital media for research dissemination has been relatively limited to date. In this article, we discuss the rationale for developing novel media to communicate and disseminate research findings and offer practical advice for doing so. We conclude by outlining a future agenda for research regarding HEOR communication and dissemination.
{"title":"#SharingHEOR: Developing Modern Media for Communication and Dissemination of Health Economics and Outcomes Research.","authors":"Davene R Wright, Mikaela Batista, Tim Wrightson","doi":"10.1007/s40258-023-00863-z","DOIUrl":"10.1007/s40258-023-00863-z","url":null,"abstract":"<p><p>Questions regarding the effectiveness and safety of health interventions and allocation of health care resources are frequently discussed in mainstream and social media. Additionally, government and foundation funders are increasingly mandating that results be disseminated to the lay public and patients may benefit from being able to digest scientific research regarding their health conditions. Therefore, it is important to widely disseminate and clearly communication health economics and outcomes research (HEOR) findings to a range of interested parties. Digital media features such as graphical or visual abstracts, infographics and videos are informative and add value to research articles by improving reader engagement with articles, potentially increasing their impact, and allowing results to be more widely disseminated. However, use of novel digital media for research dissemination has been relatively limited to date. In this article, we discuss the rationale for developing novel media to communicate and disseminate research findings and offer practical advice for doing so. We conclude by outlining a future agenda for research regarding HEOR communication and dissemination.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139995344","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-01Epub Date: 2024-03-11DOI: 10.1007/s40258-024-00875-3
Matthew Franklin, Sebastian Hinde, Rachael Maree Hunter, Gerry Richardson, William Whittaker
Commissioning describes the process of contracting appropriate care services to address pre-identified needs through pre-agreed payment structures. Outcomes-based commissioning (i.e., paying services for pre-agreed outcomes) shares a common goal with economic evaluation: achieving value for money for relevant outcomes (e.g., health) achieved from a finite budget. We describe considerations and challenges as to the practical role of relevant outcomes for evaluation and commissioning, seeking to bridge a gap between economic evaluation evidence and care commissioning. We describe conceptual (e.g., what are 'relevant' outcomes) alongside practical considerations (e.g., quantifying and using relevant endpoint or surrogate outcomes) and pertinent issues when linking outcomes to commissioning-based payment mechanisms, using England as a case study. Economic evaluation often focuses on a single endpoint health-focused maximand, e.g., quality-adjusted life-years (QALYs), whereas commissioning often focuses on activity-based surrogate outcomes (e.g., health monitoring), as easier-to-measure key performance indicators that are more acceptable (e.g., by clinicians) and amenable to being linked with payment structures. However, payments linked to endpoint and/or surrogate outcomes can lead to market inefficiencies; for example, when surrogates do not have the intended causal effect on endpoint outcomes or when service activity focuses on only people who can achieve prespecified payment-linked outcomes. Accounting for and explaining direct links from commissioners' payment structures to surrogate and then endpoint economic outcomes is a vital step to bridging a gap between economic evaluation approaches and commissioning. Decision-analytic models could aid this but they must be designed to account for relevant surrogate and endpoint outcomes, the payments assigned to such outcomes, and their interaction with the system commissioners purport to influence.
{"title":"Is Economic Evaluation and Care Commissioning Focused on Achieving the Same Outcomes? Resource-Allocation Considerations and Challenges Using England as a Case Study.","authors":"Matthew Franklin, Sebastian Hinde, Rachael Maree Hunter, Gerry Richardson, William Whittaker","doi":"10.1007/s40258-024-00875-3","DOIUrl":"10.1007/s40258-024-00875-3","url":null,"abstract":"<p><p>Commissioning describes the process of contracting appropriate care services to address pre-identified needs through pre-agreed payment structures. Outcomes-based commissioning (i.e., paying services for pre-agreed outcomes) shares a common goal with economic evaluation: achieving value for money for relevant outcomes (e.g., health) achieved from a finite budget. We describe considerations and challenges as to the practical role of relevant outcomes for evaluation and commissioning, seeking to bridge a gap between economic evaluation evidence and care commissioning. We describe conceptual (e.g., what are 'relevant' outcomes) alongside practical considerations (e.g., quantifying and using relevant endpoint or surrogate outcomes) and pertinent issues when linking outcomes to commissioning-based payment mechanisms, using England as a case study. Economic evaluation often focuses on a single endpoint health-focused maximand, e.g., quality-adjusted life-years (QALYs), whereas commissioning often focuses on activity-based surrogate outcomes (e.g., health monitoring), as easier-to-measure key performance indicators that are more acceptable (e.g., by clinicians) and amenable to being linked with payment structures. However, payments linked to endpoint and/or surrogate outcomes can lead to market inefficiencies; for example, when surrogates do not have the intended causal effect on endpoint outcomes or when service activity focuses on only people who can achieve prespecified payment-linked outcomes. Accounting for and explaining direct links from commissioners' payment structures to surrogate and then endpoint economic outcomes is a vital step to bridging a gap between economic evaluation approaches and commissioning. Decision-analytic models could aid this but they must be designed to account for relevant surrogate and endpoint outcomes, the payments assigned to such outcomes, and their interaction with the system commissioners purport to influence.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178631/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140100872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-01Epub Date: 2024-03-30DOI: 10.1007/s40258-024-00878-0
Mohsen Ghaffari Darab, Lidia Engel, Dennis Henzler, Michael Lauerer, Eckhard Nagel, Vicki Brown, Cathrine Mihalopoulos
Background: There has been an increase in model-based economic evaluations of interventions for dementia. The most recent systematic review of economic evaluations for dementia highlighted weaknesses in studies, including lack of justification for model assumptions and data inputs.
Objective: This study aimed to update the last published systematic review of model-based economic evaluations of interventions for dementia, including Alzheimer's disease, with a focus on any methodological improvements and quality assessment of the studies.
Methods: Systematic searches in eight databases, including PubMed, Cochrane, Embase, CINAHL, PsycINFO, EconLit, international HTA database, and the Tufts Cost-Effectiveness Analysis Registry were undertaken from February 2018 until August 2022. The quality of the included studies was assessed using the Philips checklist and the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist. The findings were summarized through narrative analysis.
Results: This review included 23 studies, comprising cost-utility analyses (87%), cost-benefit analyses (9%) and cost-effectiveness analyses (4%). The studies covered various interventions, including pharmacological (n = 10, 43%), non-pharmacological (n = 4, 17%), prevention (n = 4, 17%), diagnostic (n = 4, 17%) and integrated (n = 1, 4%) [diagnostics-pharmacologic] strategies. Markov transition models were commonly employed (65%), followed by decision trees (13%) and discrete-event simulation (9%). Several interventions from all categories were reported as being cost effective. The quality of reporting was suboptimal for the Methods and Results sections in almost all studies, although the majority of studies adequately addressed the decision problem, scope, and model-type selection in their economic evaluations. Regarding the quality of methodology, only a minority of studies addressed competing theories or clearly explained the rationale for model structure. Furthermore, few studies systematically identified key parameters or assessed data quality, and uncertainty was mostly addressed partially.
Conclusions: This review informs future research and resource allocation by providing insights into model-based economic evaluations for dementia interventions and highlighting areas for improvement.
{"title":"Model-Based Economic Evaluations of Interventions for Dementia: An Updated Systematic Review and Quality Assessment.","authors":"Mohsen Ghaffari Darab, Lidia Engel, Dennis Henzler, Michael Lauerer, Eckhard Nagel, Vicki Brown, Cathrine Mihalopoulos","doi":"10.1007/s40258-024-00878-0","DOIUrl":"10.1007/s40258-024-00878-0","url":null,"abstract":"<p><strong>Background: </strong>There has been an increase in model-based economic evaluations of interventions for dementia. The most recent systematic review of economic evaluations for dementia highlighted weaknesses in studies, including lack of justification for model assumptions and data inputs.</p><p><strong>Objective: </strong>This study aimed to update the last published systematic review of model-based economic evaluations of interventions for dementia, including Alzheimer's disease, with a focus on any methodological improvements and quality assessment of the studies.</p><p><strong>Methods: </strong>Systematic searches in eight databases, including PubMed, Cochrane, Embase, CINAHL, PsycINFO, EconLit, international HTA database, and the Tufts Cost-Effectiveness Analysis Registry were undertaken from February 2018 until August 2022. The quality of the included studies was assessed using the Philips checklist and the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist. The findings were summarized through narrative analysis.</p><p><strong>Results: </strong>This review included 23 studies, comprising cost-utility analyses (87%), cost-benefit analyses (9%) and cost-effectiveness analyses (4%). The studies covered various interventions, including pharmacological (n = 10, 43%), non-pharmacological (n = 4, 17%), prevention (n = 4, 17%), diagnostic (n = 4, 17%) and integrated (n = 1, 4%) [diagnostics-pharmacologic] strategies. Markov transition models were commonly employed (65%), followed by decision trees (13%) and discrete-event simulation (9%). Several interventions from all categories were reported as being cost effective. The quality of reporting was suboptimal for the Methods and Results sections in almost all studies, although the majority of studies adequately addressed the decision problem, scope, and model-type selection in their economic evaluations. Regarding the quality of methodology, only a minority of studies addressed competing theories or clearly explained the rationale for model structure. Furthermore, few studies systematically identified key parameters or assessed data quality, and uncertainty was mostly addressed partially.</p><p><strong>Conclusions: </strong>This review informs future research and resource allocation by providing insights into model-based economic evaluations for dementia interventions and highlighting areas for improvement.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178626/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140329611","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-06-25DOI: 10.1007/s40258-024-00893-1
Amber Salisbury, Joshua Ciardi, Richard Norman, Amelia K Smit, Anne E Cust, Cynthia Low, Michael Caruana, Louisa Gordon, Karen Canfell, Julia Steinberg, Alison Pearce
Purpose: Genetic and genomic testing can provide valuable information on individuals' risk of chronic diseases, presenting an opportunity for risk-tailored disease screening to improve early detection and health outcomes. The acceptability, uptake and effectiveness of such programmes is dependent on public preferences for the programme features. This study aims to conduct a systematic review of discrete choice experiments assessing preferences for genetic/genomic risk-tailored chronic disease screening.
Methods: PubMed, Embase, EconLit and Cochrane Library were searched in October 2023 for discrete choice experiment studies assessing preferences for genetic or genomic risk-tailored chronic disease screening. Eligible studies were double screened, extracted and synthesised through descriptive statistics and content analysis of themes. Bias was assessed using an existing quality checklist.
Results: Twelve studies were included. Most studies focused on cancer screening (n = 10) and explored preferences for testing of rare, high-risk variants (n = 10), largely within a targeted population (e.g. subgroups with family history of disease). Two studies explored preferences for the use of polygenic risk scores (PRS) at a population level. Twenty-six programme attributes were identified, with most significantly impacting preferences. Survival, test accuracy and screening impact were most frequently reported as most important. Depending on the clinical context and programme attributes and levels, estimated uptake of hypothetical programmes varied from no participation to almost full participation (97%).
Conclusion: The uptake of potential programmes would strongly depend on specific programme features and the disease context. In particular, careful communication of potential survival benefits and likely genetic/genomic test accuracy might encourage uptake of genetic and genomic risk-tailored disease screening programmes. As the majority of the literature focused on high-risk variants and cancer screening, further research is required to understand preferences specific to PRS testing at a population level and targeted genomic testing for different disease contexts.
{"title":"Public Preferences for Genetic and Genomic Risk-Informed Chronic Disease Screening and Early Detection: A Systematic Review of Discrete Choice Experiments.","authors":"Amber Salisbury, Joshua Ciardi, Richard Norman, Amelia K Smit, Anne E Cust, Cynthia Low, Michael Caruana, Louisa Gordon, Karen Canfell, Julia Steinberg, Alison Pearce","doi":"10.1007/s40258-024-00893-1","DOIUrl":"https://doi.org/10.1007/s40258-024-00893-1","url":null,"abstract":"<p><strong>Purpose: </strong>Genetic and genomic testing can provide valuable information on individuals' risk of chronic diseases, presenting an opportunity for risk-tailored disease screening to improve early detection and health outcomes. The acceptability, uptake and effectiveness of such programmes is dependent on public preferences for the programme features. This study aims to conduct a systematic review of discrete choice experiments assessing preferences for genetic/genomic risk-tailored chronic disease screening.</p><p><strong>Methods: </strong>PubMed, Embase, EconLit and Cochrane Library were searched in October 2023 for discrete choice experiment studies assessing preferences for genetic or genomic risk-tailored chronic disease screening. Eligible studies were double screened, extracted and synthesised through descriptive statistics and content analysis of themes. Bias was assessed using an existing quality checklist.</p><p><strong>Results: </strong>Twelve studies were included. Most studies focused on cancer screening (n = 10) and explored preferences for testing of rare, high-risk variants (n = 10), largely within a targeted population (e.g. subgroups with family history of disease). Two studies explored preferences for the use of polygenic risk scores (PRS) at a population level. Twenty-six programme attributes were identified, with most significantly impacting preferences. Survival, test accuracy and screening impact were most frequently reported as most important. Depending on the clinical context and programme attributes and levels, estimated uptake of hypothetical programmes varied from no participation to almost full participation (97%).</p><p><strong>Conclusion: </strong>The uptake of potential programmes would strongly depend on specific programme features and the disease context. In particular, careful communication of potential survival benefits and likely genetic/genomic test accuracy might encourage uptake of genetic and genomic risk-tailored disease screening programmes. As the majority of the literature focused on high-risk variants and cancer screening, further research is required to understand preferences specific to PRS testing at a population level and targeted genomic testing for different disease contexts.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141445336","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}