PharmacoEconomics最新文献

{"title":"Value-Based Healthcare in Practice: IDEATE, a Collaboration to Design and Test an Outcomes-Based Agreement for a Medicine in Wales.","authors":"Jessica R Burton, Kate Halsby, Graciela Sáinz de la Fuente, Jonathan Pearson-Stuttard, Rebecca Sloan, Thomas Porter, Gareth John, Andrew Warburton, Jennifer Selby, Gail Povey, Ruhe Chowdhury, Catherine Bale, Mark Davies, Emma Clifton-Brown, Hamish Laing","doi":"10.1007/s40273-024-01445-0","DOIUrl":"10.1007/s40273-024-01445-0","url":null,"abstract":"<p><strong>Objective: </strong>To develop a sustainable, scalable methodology for the design of outcome-based agreements (OBAs) that works on the ground and dynamically overcomes historical challenges.</p><p><strong>Methods: </strong>Project IDEATE co-created solutions to known (and emergent) challenges via iterative workshops and real-world data analysis to develop and refine a hypothetical model for an OBA in a trusted research environment. A cross-disciplinary collaboration between National Health Service (NHS) Wales, industry and academia was developed. Data were collected from Welsh national datasets and used to construct a novel linked dataset. OBA scenarios, with different contract parameters, were analysed to assess impact on the proportion of contract payment due and the volatility of payments.</p><p><strong>Results: </strong>An approved, in market, locally advanced and metastatic breast cancer treatment was selected as the test case. The total number of patients in the treatment cohort (2017-2020) was n = 99, and 286 in the control cohort (2014-2016). The final outcome variables selected were: (1) 1-year survival,( 2) intolerance to treatment (deferral), and (3) the total days disrupted by care. The primary scenario included all three outcomes measured at the population level and used a linear payment model. Volatility analyses demonstrated contract parameters can dramatically alter the contract output with greatest risk from a single, binary outcome contract design.</p><p><strong>Conclusions: </strong>The design of an OBA is a complex process that requires a multi-disciplinary approach. By assessing solutions to data, outcomes and contracting challenges, IDEATE provides a strong foundation for future success of OBAs in the UK. Outcome-based agreements (OBAs) are a way to pay for medicines if they help patient health in a specific way over time. These agreements can make it faster for people to get new medicines, but they also have challenges, like needing a lot of time and effort to manage them. A team from the NHS Wales, life sciences, and Swansea University created Project IDEATE to find a better way to design OBAs and solve some of these problems. Welsh datasets were used to create a new breast cancer dataset to test different OBAs and see how payments would change. A breast cancer treatment was used for the project. The project had 99 patients who got the medicine (2017-2020) and 286 patients who had breast cancer but did not get the medicine (2014-2016). Three health outcomes were measured: (1) living for one year after treatment, (2) patients needing to stop the medicine, and (3) days spent in care. The main OBA option we tested used all three health outcomes; the more the outcomes improved, the more the payments could go up until they hit the highest amount agreed. The analysis showed that the way an OBA is designed can make a big difference in how stable or risky it is, especially if one of the health outcomes has only two opti","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"191-207"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625499","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acceptance of Evidence Transfer Within German Early Benefit Assessment of New Drugs for Pediatric and Adolescents Target Populations.","authors":"Georgia Pick, Dirk Müller, Charalabos-Markos Dintsios","doi":"10.1007/s40273-024-01467-8","DOIUrl":"https://doi.org/10.1007/s40273-024-01467-8","url":null,"abstract":"<p><strong>Background and objective: </strong>In Germany, all new drugs undergo an early benefit assessment (EBA) by the decision-making body (G-BA). Due to limited access to clinical data in pediatric healthcare since 2017, evidence transfer has allowed for data from adult studies to be used in the EBA of pediatric drugs. This study examines the acceptance of evidence transfer, aiming to understand its correlation with granted added benefit.</p><p><strong>Methods: </strong>By searching the G-BA database, relevant EBAs were identified. In addition to descriptive statistics, agreement statistics regarding binary and ordinal extent of added benefit and binary logistic regression with and without intercept were performed to investigate acceptance of evidence transfer, juxtaposing it with manufacturers' claims, and to evaluate the impact of identified factors on evidence transfer.</p><p><strong>Results: </strong>In 14 of 36 identified EBAs, the evidence transfer was accepted by the G-BA. They referred to four therapeutic areas, received a non-quantifiable added benefit and were subject to a pediatric investigation program. Non-quantifiable added benefit implies an added value in itself which can range from minor to major added benefit and is considering the genuine uncertainty mainly induced in theese EBAs due to evidence transfer, which is not allowing a quantification of an added benefit. The binary agreement between manufacturers' claims and G-BA's appraisals was less than by chance [kappa - 0.054 (- 0.158 to 0.050)] whereas the ordinal agreement became fair [kappa 0.333 (0.261-0.406)]. Congruence of the mechanism of action, alignment of disease pattern, transferability of efficacy and safety, and same comparator were fundamental for evidence transfer. Additionally, supportive evidence, therapeutic breakthroughs, and small-scale approval enhanced the acceptance of evidence transfer. The regression models yielded similar results showing different model fit and explained variance.</p><p><strong>Conclusions: </strong>Evidence transfer hinges upon fulfilling various minimum criteria and additional supportive evidence. Availability of study data from adult or older patients and the pediatric group under evaluation is crucial.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009453","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic Testing for Prostate Cancer-A Cost-Effectiveness Analysis Comparing a Prostatype P-Score Biomarker Approach to Standard Clinical Practice.","authors":"Adam Fridhammar, Oskar Frisell, Karin Wahlberg, Emelie Berglund, Pontus Röbeck, Sofie Persson","doi":"10.1007/s40273-024-01466-9","DOIUrl":"https://doi.org/10.1007/s40273-024-01466-9","url":null,"abstract":"<p><strong>Background: </strong>The Prostatype score (P-score) is a prognostic biomarker that integrates a three-gene (IGFBP3, F3, and VGLL3) signature derived from prostate biopsy samples, with key clinical parameters, including prostate-specific antigen (PSA) levels, Gleason grade, and tumor stage at diagnosis. The test has demonstrated superior predictive accuracy for prostate cancer outcomes compared with traditional risk categorization systems such as D'Amico. Notably, it reclassifies a higher proportion of patients into the low-risk category, making them eligible for active surveillance. This study assessed the cost-effectiveness of the P-score in comparison with D'Amico and the Swedish National Prostate Cancer Register (NPCR) risk categorization systems.</p><p><strong>Methods: </strong>A two-step decision analytic model was developed. The model consisted of a decision tree-informed Markov structure estimating the lifetime outcomes of 60-year-old men with diagnosed prostate cancer. Prostate cancer was classified as low-risk, intermediate-risk, or high-risk using either the P-score or D'Amico. Initial therapy was based on observed treatment patterns from the Swedish NPCR. Costs (SEK, year 2022) and quality-adjusted life years (QALYs) were estimated from a healthcare perspective and discounted at 3% per year; incremental cost-effectiveness ratio (ICER) was the primary outcome.</p><p><strong>Results: </strong>The P-score led to cost savings and generated an additional 0.19 QALYs compared with D'Amico. The added costs of the genetic test and higher costs of active surveillance and radiotherapy were counterbalanced by savings from reduced costs of surgery, treatment-related side-effects, and metastatic disease. The gain in QALYs was primarily due to the avoidance of metastatic disease and a reduction in treatment-related side-effects.</p><p><strong>Conclusions: </strong>The results of this study suggest that the P-score is likely to be a cost-effective alternative to D'Amico for prognostic evaluation of newly diagnosed prostate cancer in Sweden and compared with NPCR when health-related quality of life was included.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966116","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Estimating the Incremental Cost Per QALY Produced by the Spanish NHS: A Fixed-Effect Econometric Approach.","authors":"Laura Vallejo-Torres","doi":"10.1007/s40273-024-01441-4","DOIUrl":"10.1007/s40273-024-01441-4","url":null,"abstract":"<p><strong>Background: </strong>Knowing the health opportunity costs of funding decisions is crucial to assess whether the health gains associated with new interventions are larger than the health losses imposed by the displacement of resources. Empirical estimates based on the effect of health spending on health outcomes have been proposed in several countries, including Spain, as a proxy to capture these opportunity costs. However, there is a need to regularly update existing health opportunity cost estimates and to explore the role of omitted variable bias in these estimations.</p><p><strong>Objective: </strong>The aim of this paper is to provide an updated and refined estimate of the causal impact of health spending on health in Spain that can be translated into an estimate of the incremental cost per quality-adjusted life-year produced by the Spanish national health system.</p><p><strong>Methods: </strong>We applied fixed-effect models using data for 17 Spanish regions from 2002 until 2022 to estimate the impact of public health spending on health outcomes and explored the extent of omitted variable bias. Changes in these estimates over time were assessed and alternative specifications were tested.</p><p><strong>Results: </strong>Based on fixed-effect models with control variables, the estimated spending elasticity was 0.061, which translated into an incremental cost per quality-adjusted life-year of approximately €34,000. The bias-corrected elasticity was 0.075, with a corresponding incremental cost per quality-adjusted life-year of €27,000. We found that the estimated impact of spending on health decreases when recent years of data are added, and that the extent of omitted variable bias appears to increase, particularly when adding the COVID-19 pandemic period.</p><p><strong>Conclusions: </strong>This study provides an updated estimation of the incremental cost per quality-adjusted life-year produced by the Spanish national health system. The estimates provided can be easily updatable as new data become accessible, and the methods applied might be transferable to other settings with similar available data.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"109-122"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724785/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505503","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability.","authors":"Marcelien H E Callenbach, Rick A Vreman, Christine Leopold, Aukje K Mantel-Teeuwisse, Wim G Goettsch","doi":"10.1007/s40273-024-01433-4","DOIUrl":"10.1007/s40273-024-01433-4","url":null,"abstract":"<p><strong>Objective: </strong>To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions.</p><p><strong>Methods: </strong>A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population. To demonstrate the potential of the framework, three case studies were used: onasemnogene abeparvovec (Zolgensma^®), brexucabtagene autoleucel (Tecartus^®) and etranacogene dezaparvovec (Hemgenix^®). A hypothetical case study was used to illustrate the output of the calculation tool.</p><p><strong>Results: </strong>Part 1 of the framework presents steps for matching a suitable reimbursement and payment model with the disease and treatment characteristics. The reimbursement and payment models are further specified in Part 2. Part 3 guides end users through the setup of a calculation tool with which the financial impact can be calculated of two payment models: a price discount model and an outcome-based spread payment model with a discount. Part 4 concerns the output of the calculation tool, showing how different payment models lead to different financial consequences under three assumptions of longer term effectiveness.</p><p><strong>Conclusions: </strong>The presented framework provides decision makers with insight into the financial consequences of their chosen payment model under different assumptions. This can aid reimbursement negotiations by clarifying the optimal choice given a therapy's characteristics.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"53-66"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724790/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142378071","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Universal Health Coverage of Opioid Agonist Treatment in Norway: An Equity-Adjusted Economic Evaluation.","authors":"Prayash Chaudhary, Lars Thore Fadnes, Steinar Fosse, Fatemeh Chalabianloo, Kjell Arne Johansson","doi":"10.1007/s40273-024-01442-3","DOIUrl":"10.1007/s40273-024-01442-3","url":null,"abstract":"<p><strong>Background: </strong>Detailed information on the efficiency of health services targeting opioid use disorder (OUD) and treatment with opioid agonist treatment (OAT) is sparse. Many countries, including Norway, are still falling short of universal health coverage (UHC) of OAT. This study aims to evaluate the incremental lifetime costs and effects of treating OUD with OAT as compared to no OAT in Norway and scaling up the treatment to a universal coverage level using equity-adjusted health economic evaluations.</p><p><strong>Methods: </strong>We conducted cost-utility and budget impact analyses and constructed a two-state Markov model to compare the lifetime costs and outcomes among patients with OUD with and without OAT. Model inputs were derived from routine health information systems and the literature, with costs reported in 2023 Norwegian Kroner (NOK). The analyses were conducted from a Norwegian extended health-service and societal perspectives, with a lifetime time horizon. Quality-adjusted life years (QALYs) was the metric of health benefits. Outcomes were reported as incremental cost-effectiveness ratios (ICERs). The willingness-to-pay (WTP) threshold was equity-adjusted according to the future prognostic healthy life year loss method in Norway (severity of disease criterion), which is sensitive to the size of future undiscounted healthy life year loss due to the affected conditions. The WTP threshold is NOK 825,000 per QALY gained in Norwegian policy for conditions with undiscounted future QALY loss > 20. Uncertainty in the parameters and robustness of the results were assessed with one-way and probabilistic sensitivity analyses and scenario analyses.</p><p><strong>Findings: </strong>The mean results from probabilistic sensitivity analysis estimated that OAT was associated with 3.03 additional discounted QALYs gain and incremental lifetime discounted cost of NOK 1.45 million, leading to an ICER of NOK 479,099 per QALY gained when compared with not providing OAT, with the extended health-service perspective. From a societal perspective, OAT was cost-saving, i.e. OAT produced greater health benefits while resulting in lower overall societal costs compared to no OAT. The mean undiscounted future health loss was estimated to be 21.34 QALYs for the Norwegian patient group with OUD. A total 5-year budget increase of NOK 1.208 billion was estimated if OAT was going to be scaled up from the current coverage level of 70% to UHC. Compared with the current coverage, 100% coverage of OAT was associated with an additional lifetime cost of NOK 4.332 billion but also an additional 6760 QALYs gained.</p><p><strong>Conclusion: </strong>Our analysis suggests that OAT is cost-effective in Norway and has the potential to be cost-saving from a societal perspective. Therefore, Norwegian policy should consider scaling up treatment to extend the coverage of OAT.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"93-107"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724781/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142505504","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Design and Features of Pricing and Payment Schemes for Health Technologies: A Scoping Review and a Proposal for a Flexible Need-Driven Classification.","authors":"Vittoria Ardito, Oriana Ciani, Michael Drummond","doi":"10.1007/s40273-024-01435-2","DOIUrl":"10.1007/s40273-024-01435-2","url":null,"abstract":"<p><strong>Background and objective: </strong>In a context of growing clinical and financial uncertainty, pricing and payment schemes can act as possible solutions to the problems of affordability and access to health technologies. However, a comprehensive categorization of the available schemes to help decision makers tackle these challenges is lacking. This work aims at mapping existing types of pricing and payment schemes, and proposes a new approach for their classification, in order to help decision makers and other stakeholders select the best type of scheme to meet their needs.</p><p><strong>Methods: </strong>A Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR)-compliant scoping literature review was performed between 2010 and 2023 in three databases (PubMed, Web of Science, Scopus). The search strategy was developed around two groups of keywords, \"pricing/payment schemes\" and \"scheme innovativeness\". Eligible studies were those illustrating the unique design and features of each scheme type, which were extracted by two independent reviewers, and synthesized using a narrative format, including a detailed tabular description of each type of scheme.</p><p><strong>Results: </strong>A total of 70 unique types of pricing and payment schemes were identified. Around one third (33%) was only specified in principle, while two thirds (67%) had been implemented in practice. About half of the scheme types were proposed for drugs (34/70, 49%), and the vast majority were not designed for a specific therapeutic area (55/70, 79%). Each scheme type was categorized based on distinctive characteristics: the objectives, the outcome component, the timing/modalities of payments, and the evidence collection requirements.</p><p><strong>Conclusions: </strong>Instead of trying to fit the retrieved schemes into a rigid taxonomy, we propose a new approach that suggests a flexible need-driven use of the available scheme types, driven primarily by the specific objective that one might have, and allows leveraging of the other key characteristics of each type of scheme.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"5-29"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724778/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142471985","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Economic Burden Associated with Pulmonary Arterial Hypertension in the United States.","authors":"Anna Watzker, Adnan Alsumali, Christine Ferro, Gabriela Dieguez, Clare Park, Dominik Lautsch, Karim El-Kersh","doi":"10.1007/s40273-024-01427-2","DOIUrl":"10.1007/s40273-024-01427-2","url":null,"abstract":"<p><strong>Background: </strong>Pulmonary arterial hypertension (PAH) is a progressive disease characterized by elevated pressure in the pulmonary arteries, commonly resulting in right heart failure. PAH is associated with a high economic burden throughout the duration of the disease.</p><p><strong>Methods: </strong>This retrospective cohort study of the Milliman Contributor Health Source Data, the Medicare 100% Research Identifiable Files, and the Merative Marketscan^® Commercial dataset between 2018 and 2020 identified adult patients with prevalent PAH based on the earliest qualifying diagnosis date or medication date ('index date') between January 1, 2019 and November 30, 2020. Outcomes were assessed using patient data from index date through the earliest of end of enrollment, end of data, or death (Medicare fee-for-service [FFS] only). All-cause and PAH-related medical and pharmacy costs per-patient per-month (PPPM) and healthcare resource utilization per 1000 patients were summarized.</p><p><strong>Results: </strong>The study included 11,670 Medicare FFS, 1021 Medicare Advantage, 274 Medicaid, and 1174 commercially insured patients in the US. The annual national burden to payers was estimated to be US$3.1 billion. The PPPM payer costs ranged from US$6500 to US$14,742; out-of-pocket (OOP) costs ranged from US$341 to US$907 PPPM. Inpatient utilization rate ranged from 435 to 770 per 1000 patients for all-cause admissions and from 15 to 58 per 1000 patients for PAH-related admissions.</p><p><strong>Conclusions: </strong>This study demonstrates that PAH continues to be associated with a high economic burden and healthcare resource utilization across all payer types within the US healthcare system.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"83-91"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724771/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142471986","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Analysis of Traditional and Pharmacometric-Based Pharmacoeconomic Modeling in the Cost-Utility Evaluation of Sunitinib Therapy.","authors":"Maddalena Centanni, Janine Nijhuis, Mats O Karlsson, Lena E Friberg","doi":"10.1007/s40273-024-01438-z","DOIUrl":"10.1007/s40273-024-01438-z","url":null,"abstract":"<p><strong>Background: </strong>Cost-utility analyses (CUAs) increasingly use models to predict long-term outcomes and translate trial data to real-world settings. Model structure uncertainty affects these predictions. This study compares pharmacometric against traditional pharmacoeconomic model evaluations for CUAs of sunitinib in gastrointestinal stromal tumors (GIST).</p><p><strong>Methods: </strong>A two-arm trial comparing sunitinib 37.5 mg daily with no treatment was simulated using a pharmacometric-based pharmacoeconomic model framework. Overall, four existing models [time-to-event (TTE) and Markov models] were re-estimated to the survival data and linked to logistic regression models describing the toxicity data [neutropenia, thrombocytopenia, hypertension, fatigue, and hand-foot syndrome (HFS)] to create traditional pharmacoeconomic model frameworks. All five frameworks were used to simulate clinical outcomes and sunitinib treatment costs, including a therapeutic drug monitoring (TDM) scenario.</p><p><strong>Results: </strong>The pharmacometric model framework predicted that sunitinib treatment costs an additional 142,756 euros per quality adjusted life year (QALY) compared with no treatment, with deviations - 21.2% (discrete Markov), - 15.1% (continuous Markov), + 7.2% (TTE Weibull), and + 39.6% (TTE exponential) from the traditional model frameworks. The pharmacometric framework captured the change in toxicity over treatment cycles (e.g., increased HFS incidence until cycle 4 with a decrease thereafter), a pattern not observed in the pharmacoeconomic frameworks (e.g., stable HFS incidence over all treatment cycles). Furthermore, the pharmacoeconomic frameworks excessively forecasted the percentage of patients encountering subtherapeutic concentrations of sunitinib over the course of time (pharmacoeconomic: 24.6% at cycle 2 to 98.7% at cycle 16, versus pharmacometric: 13.7% at cycle 2 to 34.1% at cycle 16).</p><p><strong>Conclusions: </strong>Model structure significantly influences CUA predictions. The pharmacometric-based model framework more closely represented real-world toxicity trends and drug exposure changes. The relevance of these findings depends on the specific question a CUA seeks to address.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"31-43"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724784/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142351615","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How Much Better is Faster? Empirical Tests of QALY Assumptions in Health-Outcome Sequences.","authors":"F Reed Johnson, John J Sheehan, Semra Ozdemir, Matthew Wallace, Jui-Chen Yang","doi":"10.1007/s40273-024-01437-0","DOIUrl":"10.1007/s40273-024-01437-0","url":null,"abstract":"<p><strong>Objectives: </strong>This study was designed to test hypotheses regarding the path dependence of health-outcome values in the form of linear additivity of health-state utilities and diminishing marginal utility of health outcomes.</p><p><strong>Methods: </strong>We employed a discrete-choice experiment to quantify patient treatment preferences for major depressive disorder. In a series of choice questions, participants evaluated seven symptom-improvement sequences and out-of-pocket costs over 6-week durations. Money-equivalent values were derived from a deductive latent-class mixed-logit analysis.</p><p><strong>Results: </strong>The discrete-choice experiment was completed by 751 respondents with self-reported major depressive disorder recruited from an online commercial panel. The class-membership probability was 0.83 for latent-class preferences consistent with supporting relative importance weights for all symptom-improvement sequences in the study design. First, we found strong support for diminishing marginal utility in symptom-improvement sequences. The money-equivalent value of an initial week of normal mood was $147 (95% confidence interval: $128, $166) and a second week of normal mood was $70 ($49, $91). Furthermore, for short treatment durations where conventional discounting was not a factor, equivalent changes in health status were valued more highly for an earlier onset of effect: holding subsequent symptom patterns constant, $338 (211, 454) versus $70 (49, 91) for improvements starting in week 2 versus week 3 and $147 ($128, $166) versus $29 (-$4, $64) for improvements starting in week 3 versus week 4.</p><p><strong>Conclusions: </strong>Our findings imply that conventional quality-adjusted life-year calculations in which health values are assumed to be path independent can understate the value of health improvements that appear earlier in a sequence.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"45-52"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142375715","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}