Value in Health最新文献

{"title":"It Takes 2 to Tango. Setting Out the Conditions in Which Performance-Based Risk-Sharing Arrangements Work for Both Parties","authors":"","doi":"10.1016/j.jval.2024.03.2196","DOIUrl":"10.1016/j.jval.2024.03.2196","url":null,"abstract":"<div><h3>Objectives</h3><p>Faster regulatory approval processes often fail to achieve faster patient access. We seek an approach, using performance-based risk-sharing arrangements, to address uncertainty for payers regarding the relative effectiveness and value for money of products launched through accelerated approval schemes.</p><p>One important reason for risk sharing is to resolve differences of opinion between innovators and payers about a technology’s underlying value. To date, there has been no formal attempt to set out the circumstances in which risk sharing can address these differences.</p></div><div><h3>Methods</h3><p>We use a value of information framework to understand what a performance-based risk-sharing arrangements can, in principle, add to a reimbursement scheme, separating payer perspectives on cost-effectiveness and the value of research from those of the innovator. We find 16 scenarios, developing 5 rules to analyze these 16 scenarios, identifying cases in which risk sharing adds value for both parties.</p></div><div><h3>Results</h3><p>We find that risk sharing provides an improved solution in 9 out of 16 combinations of payer and innovator expectations about treatment outcome and the value of further research. Among our assumptions, who pays for research and scheme administration costs are key.</p></div><div><h3>Conclusions</h3><p>Steps should be undertaken to make risk sharing more practical, ensuring that payers consider it an option. This requires additional costs to the health system falling on the innovator in an efficient way that aligns incentives for product development for global markets. Health systems benefits are earlier patient access to cost-effective treatments and payers with higher confidence of not wasting money. Innovators get greater returns while conducting research.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023325/pdfft?md5=823b232a278064ad1a3a7839bd80bdc0&pid=1-s2.0-S1098301524023325-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140776712","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}

{"title":"The Inflation Reduction Act: An Opportunity to Accelerate Confidence in Real-World Evidence in the United States","authors":"","doi":"10.1016/j.jval.2024.04.007","DOIUrl":"10.1016/j.jval.2024.04.007","url":null,"abstract":"<div><h3>Objectives</h3><p>The Inflation Reduction Act (IRA), enacted in 2022, brings substantial reforms to the US healthcare system, particularly regarding Medicare. A key aspect includes the introduction of Medicare price negotiation. The objective of this commentary is to explore the implications of the IRA for US pharmaceutical companies, with a specific focus on the role of real-world evidence (RWE) in the context of Medicare reforms.</p></div><div><h3>Methods</h3><p>This commentary uses a qualitative analysis of the IRA’s provisions related to healthcare and pharmaceutical regulation, focusing on how these reforms change the evidence requirements for pharmaceutical companies. It discusses the methodological aspects of generating and using RWE, including techniques such as target trial emulation and quantitative bias analysis methods to address biases inherent in RWE.</p></div><div><h3>Results</h3><p>This commentary highlights that the IRA introduces a unique approach to value assessment in the United States by evaluating drug value several years after launch, as opposed to at launch, similar to health technology assessments in other regions. It underscores the central role of RWE in comparing drug effectiveness across diverse clinical scenarios to improve the accuracy of real-world data comparisons. Furthermore, this article identifies key methodologies for managing the inherent biases in RWE, which are crucial for generating credible evidence for IRA price negotiations.</p></div><div><h3>Conclusions</h3><p>This article underscores the importance of these methodologies in ensuring credible evidence for IRA price negotiations. It advocates for an integrated approach in evidence generation, positioning RWE as pivotal for informed pricing discussions in the US healthcare landscape.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140763145","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}

{"title":"Economic Evaluations of Chimeric Antigen Receptor T-Cell Therapies for Hematologic and Solid Malignancies: A Systematic Review","authors":"","doi":"10.1016/j.jval.2024.04.004","DOIUrl":"10.1016/j.jval.2024.04.004","url":null,"abstract":"<div><h3>Objectives</h3><p>This study aimed to systematically review evidence on the cost-effectiveness of chimeric antigen receptor T-cell (CAR-T) therapies for patients with cancer.</p></div><div><h3>Methods</h3><p>Electronic databases were searched in October 2022 and updated in September 2023. Systematic reviews, health technology assessments, and economic evaluations that compared costs and effects of CAR-T therapy in patients with cancer were included. Two reviewers independently screened studies, extracted data, synthesized results, and critically appraised studies using the Philips checklist. Cost data were presented in 2022 US dollars.</p></div><div><h3>Results</h3><p>Our search yielded 1809 records, 47 of which were included. Most of included studies were cost-utility analysis, published between 2018 and 2023, and conducted in the United States. Tisagenlecleucel, axicabtagene ciloleucel, idecabtagene vicleucel, ciltacabtagene autoleucel, lisocabtagene maraleucel, brexucabtagene autoleucel, and relmacabtagene autoleucel were compared with various standard of care chemotherapies. The incremental cost-effectiveness ratio (ICER) for CAR-T therapies ranged from $9424 to $4 124 105 per quality-adjusted life-year (QALY) in adults and from $20 784 to $243 177 per QALY in pediatric patients. Incremental cost-effectiveness ratios were found to improve over longer time horizons or when an earlier cure point was assumed. Most studies failed to meet the Philips checklist due to a lack of head-to-head comparisons and uncertainty surrounding CAR-T costs and curative effects.</p></div><div><h3>Conclusions</h3><p>CAR-T therapies were more expensive and generated more QALYs than comparators, but their cost-effectiveness was uncertain and dependent on patient population, cancer type, and model assumptions. This highlights the need for more nuanced economic evaluations and continued research to better understand the value of CAR-T therapies in diverse patient populations.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023362/pdfft?md5=4d4873fcfc4e50860b406c6a66f9df2f&pid=1-s2.0-S1098301524023362-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140770549","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}

{"title":"An Exploratory Study of Alternative Time Frames and Descriptors for EQ-5D-5L in Obstructive Airway Diseases Using Mixed Methods.","authors":"Ai-Ping Chua, Mathieu F Janssen, Ling Jie Cheng, Jan Busschbach, Nan Luo","doi":"10.1016/j.jval.2024.07.004","DOIUrl":"10.1016/j.jval.2024.07.004","url":null,"abstract":"<p><strong>Objectives: </strong>EQ-5D-5L with its recall time of \"today\" may limit its ability to capture episodic symptoms and exacerbations in chronic obstructive airway diseases (OAD). We examined whether longer time frames and changing the intensity response scales to frequency scales could improve the measurement properties of EQ-5D-5L.</p><p><strong>Methods: </strong>We used a mixed-method design starting with in-depth interviews with 20 patients and clinicians to elicit preferred time frames using concept elicitation techniques and content analyses. We then administered the top 4 preferred variants using 1- and 4-weeks' time frames with the original intensity or an alternative frequency response scale alongside EQ-5D-5L and St George Respiratory Questionnaire to OAD patients during 2 different visits. We compared the ceiling effects and construct validity by testing a priori hypotheses in relation to St George Respiratory Questionnaire and clinical outcomes via correlation and receiver operating characteristic (ROC) analyses, respectively. Follow-up patients were categorized into \"better,\" \"stable,\" and \"worse\" groups to assess reliability using intraclass correlation coefficient (ICC) or Cohen's Kappa (k) and responsiveness using ROC analysis.</p><p><strong>Results: </strong>A total of 184 patients (mean [SD] age: 54[18]; female: 37.0%) completed baseline assessments. A total of 120 patients also completed follow-up assessments (mean [SD] interval: 2.8 [1.7] months). The ceilings were lower in the variants compared with EQ-5D-5L (P < .001). Reliability of the variants were comparable to or higher than EQ-5D-5L. The c-statistic values derived from ROC analyses of the variants were consistently higher than EQ-5D-5L.</p><p><strong>Conclusions: </strong>Use of longer time frames with the original intensity or the frequency response scales may improve EQ-5D-5L's psychometric properties in OAD patients.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879479","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}

{"title":"A Causal Model for Primary Prevention of Cardiovascular Disease: The Health Economic Model for the Primary Prevention of Cardiovascular Disease.","authors":"Jedidiah I Morton, Danny Liew, Zanfina Ademi","doi":"10.1016/j.jval.2024.07.010","DOIUrl":"10.1016/j.jval.2024.07.010","url":null,"abstract":"<p><strong>Objectives: </strong>Our objective was to design and develop an open-source model capable of simulating interventions for primary prevention of cardiovascular disease (CVD) that incorporated the cumulative effects of risk factors (eg, cholesterol years or blood-pressure years) to enhance health economic modeling in settings which clinical trials are not possible.</p><p><strong>Methods: </strong>We reviewed the literature to design the model structure by selecting the most important causal risk factors for CVD-low-density lipoprotein-cholesterol (LDL-C), systolic blood pressure (SBP), smoking, diabetes, and lipoprotein (a) (Lp(a))-and most common CVDs-myocardial infarction and stroke. The epidemiological basis of the model involves the simulation of risk factor trajectories, which are used to modify CVD risk via causal effect estimates derived from Mendelian randomization. LDL-C, SBP, Lp(a), and smoking all have cumulative impacts on CVD risk, which were incorporated into the health economic model. The data for the model were primarily sourced from the UK Biobank study. We calibrated the model using clinical trial data and validated the model against the observed UK Biobank data. Finally, we performed an example health economic analysis to demonstrate the utility of the model. The model is open source.</p><p><strong>Results: </strong>The model performed well in all validation tests. It was able to produce interpretable and plausible (consistent with expectations of the existing literature) results from an example health economic analysis.</p><p><strong>Conclusions: </strong>We have constructed an open-source health economic model capable of incorporating the cumulative effect of LDL-C (ie, cholesterol years), SBP (SBP-years), Lp(a), and smoking on lifetime CVD risk.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879478","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}

{"title":"New International Pharmaco-Economic Collaboration on Alzheimer's Disease (IPECAD) Open-Source Model Framework for the Health Technology Assessment of Early Alzheimer's Disease Treatment: Development and Use Cases.","authors":"Ron Handels, William L Herring, Sabine Grimm, Anders Sköldunger, Bengt Winblad, Anders Wimo, Linus Jönsson","doi":"10.1016/j.jval.2024.07.009","DOIUrl":"10.1016/j.jval.2024.07.009","url":null,"abstract":"<p><strong>Objectives: </strong>Reimbursement decisions for new Alzheimer's disease (AD) treatments are informed by economic evaluations. An open-source model with intuitive structure for model cross-validation can support the transparency and credibility of such evaluations. We describe the new International Pharmaco-Economic Collaboration on Alzheimer's Disease (IPECAD) open-source model framework (version 2) for the health-economic evaluation of early AD treatment and use it for cross-validation and addressing uncertainty.</p><p><strong>Methods: </strong>A cohort state-transition model using a categorized composite domain (cognition and function) was developed by replicating an existing reference model and testing it for internal validity. Then, features of existing Institute for Clinical and Economic Review (ICER) and Alzheimer's Disease Archimedes Condition-Event Simulator (AD-ACE) models assessing lecanemab treatment were implemented for model cross-validation. Additional uncertainty scenarios were performed on choice of efficacy outcome from trial, natural disease progression, treatment effect waning and stopping rules, and other methodological choices. The model is available open-source as R code, spreadsheet, and web-based version via https://github.com/ronhandels/IPECAD.</p><p><strong>Results: </strong>In the IPECAD model incremental life-years, quality-adjusted life-years (QALY) gains and cost savings were 21% to 31% smaller compared with the ICER model and 36% to 56% smaller compared with the AD-ACE model. IPECAD model results were particularly sensitive to assumptions on treatment effect waning and stopping rules and choice of efficacy outcome from trial.</p><p><strong>Conclusions: </strong>We demonstrated the ability of a new IPECAD open-source model framework for researchers and decision makers to cross-validate other (Health Technology Assessment submission) models and perform additional uncertainty analyses, setting an example for open science in AD decision modeling and supporting important reimbursement decisions.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879525","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}

{"title":"Development of a Prioritization Framework to Aid Healthcare Funding Decision Making in Health Technology Assessment in Australia: Application of Multicriteria Decision Analysis.","authors":"Saval Khanal, Son Nghiem, Mel Miller, Paul Scuffham, Joshua Byrnes","doi":"10.1016/j.jval.2024.07.003","DOIUrl":"10.1016/j.jval.2024.07.003","url":null,"abstract":"<p><strong>Objectives: </strong>This study develops a prioritization framework to aid healthcare funding decision making in health technology assessment (HTA) in Australia using a multiple criteria decision analysis (MCDA) approach.</p><p><strong>Methods: </strong>MCDA frameworks for HTAs were reviewed through literature survey to identify the initial criteria and levels within each criterion. Key stakeholders and experts were consulted to confirm these criteria and levels. A conjoint analysis using 1000Minds was undertaken with policy makers from the Department of Health to establish ranking criteria and weighting scores. Monte Carlo simulations were used to examine the sensitivity of findings to factors affecting the ranking and weighting scores. The MCDA was then applied to 6 examples of chronic care models or technologies projects to demonstrate the performance of this approach.</p><p><strong>Results: </strong>Five criteria (clinical efficacy/effectiveness, safety and tolerability, severity of the condition, quality/uncertainty, and direct impact on healthcare costs) were consistently ranked highest by healthcare decision makers. Among the criteria, patient-level health outcomes were considered the most important, followed by social and ethical values. The analyses were robust to inform the uncertainty in the parameter.</p><p><strong>Conclusions: </strong>This study has developed an MCDA tool that effectively integrates key priorities for HTA reviews, reflecting the values and preferences of healthcare stakeholders in Australia. Although this tool aims to align the assessment process more closely with health benefits, it also highlights the importance of considering other criteria.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879520","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}

{"title":"Clinical Outcome Assessments are Never \"Validated\".","authors":"Christina K Zigler, Theresa Coles","doi":"10.1016/j.jval.2024.07.006","DOIUrl":"10.1016/j.jval.2024.07.006","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879519","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}

{"title":"Discriminatory Properties of Quality-Adjusted Life Year Based Cost-Effectiveness Analyses for Patients With Disabilities: A Duchenne Muscular Dystrophy Case Study.","authors":"Alexa C Klimchak, Lauren E Sedita, Eleanor M Perfetto, Katherine L Gooch, Daniel C Malone","doi":"10.1016/j.jval.2024.07.008","DOIUrl":"10.1016/j.jval.2024.07.008","url":null,"abstract":"<p><strong>Objectives: </strong>Quality-adjusted life years (QALYs) have been challenged as a measure of benefit for people with disabilities, particularly for those in low-utility health states or with irreversible disability. This study examined the impact of a QALY-based assessment on the price for a hypothetical treatment for Duchenne muscular dystrophy (DMD), a progressive, genetic neuromuscular disease.</p><p><strong>Methods: </strong>A previously published, 5-state model, which analyzed treatments for early ambulatory (EA) DMD patients, was replicated, validated, and adapted to include early nonambulatory (ENA) DMD patients. The model was used to assess a QALY-based threshold price (maximum cost-effective price) for a hypothetical treatment for 13-year-old ENA and 5-year-old EA patients (initial health states with lower and higher utility, respectively). All inputs were replicated including willingness-to-pay thresholds of $50 000 to $200 000/QALY.</p><p><strong>Results: </strong>In contrast to EA patients, ENA patients had a 98% modeled decline in QALY-based threshold price at a willingness-to-pay of $150 000/QALY or higher, despite equal treatment benefit (delayed progression/death). At $100 000/QALY or lower, net nontreatment costs exceeded health benefits, implying any treatment for ENA patients would not be considered cost-effective, even at $0 price, including an indefinite pause in disease progression.</p><p><strong>Conclusions: </strong>For certain severe, disabling conditions, traditional approaches are likely to conclude that treatments are not cost-effective at any price once a patient progresses to a disabled health state with low utility value. These findings elucidate theoretical/ethical concerns regarding potential discriminatory properties of traditional QALY assessments for people with disabilities, particularly those who have lost ambulation or have other physical limitations.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879521","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}

{"title":"When are You Recovered? Defining Cutoff Points Using the Acute Respiratory Tract Infection Questionnaire.","authors":"Eskild Johansen, Volkert Siersma, Malene P Hansen, Rune M Aabenhus","doi":"10.1016/j.jval.2024.07.002","DOIUrl":"10.1016/j.jval.2024.07.002","url":null,"abstract":"<p><strong>Objectives: </strong>Acute respiratory tract infections (ARTIs) are a heterogenous group of diseases. Often, it is difficult to obtain a precise diagnosis in general practice but also difficult to determine when the patient is recovered. The lack of a precise definition of recovery after ARTI complicates scientific research aiming to optimize diagnostics and compare treatments. The study aimed to define cutoff points to determine the end of an ARTI as a proxy for recovery in patients diagnosed with ARTI in general practice using a validated patient-reported outcome instrument; The ARTI Questionnaire (ARTIQ).</p><p><strong>Methods: </strong>A total of 259 participants was divided in 2 groups-1 with ARTI and 1 without. Histograms and area under the curve were calculated for each of the 5 dimensions within the ARTIQ to evaluate the discriminative effect. For the most discriminative dimensions receiver operating comparison curves were performed to determine relevant cutoff points for having or not having ARTI symptoms and serve as a proxy for recovery in clinical research.</p><p><strong>Results: </strong>The highest discriminative effect was found in 2 dimensions: \"physical-upper airways\" and \"physical-lower airways.\" When combining these dimensions, the area under the curve was 0.97. Sensitivity, specificity, and predictive values were calculated for selected cutoff points.</p><p><strong>Conclusion: </strong>Cutoff points serving as proxy for recovery from ARTI using a patient-reported outcome were identified. The specific cutoff point for a certain research project must be selected considering the specific clinical situation of interest.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879539","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}