Carrie-Anne Ng, Richard De Abreu Lourenco, Rosalie Viney, Richard Norman, Madeleine T King, Nancy Kim, Brendan Mulhern
{"title":"Valuing quality of life for economic evaluations in cancer: navigating multiple methods.","authors":"Carrie-Anne Ng, Richard De Abreu Lourenco, Rosalie Viney, Richard Norman, Madeleine T King, Nancy Kim, Brendan Mulhern","doi":"10.1080/14737167.2024.2393332","DOIUrl":"10.1080/14737167.2024.2393332","url":null,"abstract":"<p><strong>Introduction: </strong>Utility values offer a quantitative means to evaluate the impact of novel cancer treatments on patients' quality of life (QoL). However, the multiple methods available for valuing QoL present challenges in selecting the most appropriate method across different contexts.</p><p><strong>Areas covered: </strong>This review provides cancer clinicians and researchers with an overview of methods to value QoL for economic evaluations, including standalone and derived preference-based measures (PBMs) and direct preference elicitation methods. Recent developments are described, including the comparative performance of cancer-specific PBMs versus generic PBMs, measurement of outcomes beyond health-related QoL, and increased use of discrete choice experiments to elicit preferences. Recommendations and considerations are provided to guide the choice of method for cancer research.</p><p><strong>Expert opinion: </strong>We foresee continued adoption of the QLU-C10D and FACT-8D in cancer clinical trials given the extensive use of the EORTC QLQ-C30 and FACT-G in cancer research. While these cancer-specific PBMs offer the convenience of eliciting utility values without needing a standalone PBM, researchers should consider potential limitations if they intend to substitute them for generic PBMs. As the field advances, there is a greater need for consensus on the approach to selection and integration of various methods in cancer clinical trials.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141999675","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}
Frank van der Meer, Jesper Jorgensen, Mickael Hiligsmann
{"title":"Burden of non-motor symptoms of Parkinson's disease: cost-of-illness and quality-of-life estimates through a scoping review.","authors":"Frank van der Meer, Jesper Jorgensen, Mickael Hiligsmann","doi":"10.1080/14737167.2024.2390042","DOIUrl":"10.1080/14737167.2024.2390042","url":null,"abstract":"<p><strong>Introduction: </strong>Parkinson's Disease (PD) is a progressive, chronic neurodegenerative disease, representing significant economic and social burdens. It is typically defined by motor symptoms (MSs), however, this does not reflect the full patient burden. Non-motor symptoms (NMSs) are increasingly recognized as central characteristics of PD. However, they still lack recognition in research. Therefore, this study aims to identify relevant NMSs, their prevalence, and the effect they have on Quality-of-Life (QoL) and Cost-of-Illness (COI). Secondly, it aims to identify gaps in the current body of knowledge and propose possible ways future research could bridge those gaps.</p><p><strong>Methods: </strong>The study employed a scoping review, identifying 60 records for inclusion, using PubMed and Web of Science. It included studies from Spain or Italy, including data on People with Parkinson's Disease. A comparative analysis was performed using Microsoft Excel.</p><p><strong>Results: </strong>It showed that the body of evidence relevant to NMSs, their prevalence, QoL, and COI is limited, or that estimates vary to an extent where interpretation is difficult.</p><p><strong>Conclusion: </strong>Most studies suffer from generalization, representation, and standardization issues, stemming from their designs and methodological decisions. Although the findings of this study should be interpreted with caution, several recommendations are made for future research.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141975487","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}
Emilie Batista Freire, Henry Maia Peixoto, Kênnea Martins Almeida Ayupe, Everton Nunes da Silva, Rodrigo Luiz Carregaro, Aline Martins de Toledo
{"title":"Direct healthcare costs for people with cerebral palsy in the Brazilian unified health system between 2015 and 2019.","authors":"Emilie Batista Freire, Henry Maia Peixoto, Kênnea Martins Almeida Ayupe, Everton Nunes da Silva, Rodrigo Luiz Carregaro, Aline Martins de Toledo","doi":"10.1080/14737167.2024.2390043","DOIUrl":"10.1080/14737167.2024.2390043","url":null,"abstract":"<p><strong>Background: </strong>Estimate the costs of inpatient and outpatient care for people with Cerebral Palsy (CP) in Brazil.</p><p><strong>Research design and methods: </strong>Health records of people with CP in the Hospital and Outpatient Information Systems of Brazil between 2015 and 2019 were analyzed. Variables analyzed were gender, age, ICD, Intensive Care Unit (ICU) use, total cost, and ICU cost. Costs were adjusted for inflation and converted to dollars. Linear regression analysis was performed to investigate the association between social and clinical variables and direct costs.</p><p><strong>Results: </strong>A total direct cost of approximately $166 million to the National Health System was identified, with $7.08 million/year and $26.1 million/year of inpatient and outpatient costs, respectively. The healthcare was primarily for children up to 14 years of age. The ICD 'spastic quadriplegic CP' received the most attendance. Rehabilitation was responsible for 75% of the outpatient care, with physiotherapy standing out. Increased age, use of ICU, and the types of CP are related to increased cost.</p><p><strong>Conclusions: </strong>Healthcare for people with CP produced expressive costs for the Brazilian public health system, mainly with outpatient procedures and rehabilitation, with children being the most attended. Estimating these costs assist in better resource allocation for more effective healthcare provision.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141906374","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}
{"title":"The utility and impact of digital endpoints for improving breast cancer outcomes.","authors":"Ismail Jatoi, Jonathan A L Gelfond","doi":"10.1080/14737167.2024.2390056","DOIUrl":"10.1080/14737167.2024.2390056","url":null,"abstract":"<p><strong>Introduction: </strong>In breast cancer clinical trials utilizing digital endpoints, wearable sensors record participants' health information during activities of daily living. These sensors are worn on the wrist or finger, placed as a skin patch or headband, or embedded on clothing. Data collected from wearable sensors form the basis of a digital endpoint, useful for determining effects of novel treatments on health outcomes, particularly quality-of-life outcomes.</p><p><strong>Areas covered: </strong>References for this article were selected from a PubMed search spanning from 1 January 2017,to 1 July 2024, using the terms 'wearable sensors,' 'digital endpoints,' 'virtualtrials,' 'breast cancer.' Additional articles from the authors' personal collection of papers and reviewers suggestions were also used.</p><p><strong>Expert opinion: </strong>Digital endpoints must be validated as proper surrogate measures for healthcare outcomes, prior to their use in breast cancer trials. Wearable sensors may introduce biases, such as 'missing not-at-random bias,' and perhaps even exacerbate disparities in healthcare outcomes if patients not comfortable with their use are excluded from clinical trials, or if the accuracy of sensors varies between racial and ethnic groups. Therefore, before embarking on trials with digital endpoints, validation studies are required, and limitations and risks of such trials need to be addressed.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141893291","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}
{"title":"Cost-effectiveness analysis of tofacitinib for the treatment of moderate to severe rheumatoid arthritis: a systematic review and meta-analysis.","authors":"Behzad Fatemi, Neda Yaghobi, Nikta Shobeiri, Razieh Ahmadi, Taraneh Mousavi, Fatemeh Soleymani, Soheila Rezaei","doi":"10.1080/14737167.2024.2390041","DOIUrl":"10.1080/14737167.2024.2390041","url":null,"abstract":"<p><strong>Background: </strong>As rheumatoid arthritis (RA) is a chronic and progressive disease that requires lifelong therapeutic intervention, it represents a considerable economic burden on those affected. This study investigated whether tofacitinib is a cost-effective therapeutic alternative to other DMARDs for treating moderate-to-severe RA.</p><p><strong>Research design and methods: </strong>All economic evaluation studies of tofacitinib compared to other DMARDs were identified. Using random-effects meta-analysis, we pooled incremental net benefit (INB) in (purchasing power parity) adjusted US$ with 95% confidence intervals. The modified economic evaluation bias checklist and Grading of Recommendation, Assessment, Development, and Evaluation (GRADE) instrument for quality appraisal were used. The subgroup analysis was done based on the comparator regimen.</p><p><strong>Results: </strong>Of the selected 11 studies, the number of studies from high-, upper-middle- and lower-middle-income countries was 7, 3, and 1, respectively. The subgroup analysis showed that tofacitinib with an INB of 19,180 US$ [95% CI, -34520 to -3840; p-value = 0.01] was not statistically cost-effective compared with cDMARDs (p-value > 0.0001). Compared to other DMARDs, the estimated pooled INB of tofacitinib was US$ 7260 [95% CI, 3030 to 11,480; p-value < 0.001], but there was substantial heterogeneity among the included studies, and the observed publication bias.</p><p><strong>Conclusion: </strong>While tofacitinib shows potential as a cost-effective treatment, tailored economic evaluations are needed to account for the diverse and evolving contexts of RA treatment.</p><p><strong>Registration: </strong>PROSPERO: CRD42023405970.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141893290","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}
Morgan Ehman, Jesman Punian, Deirdre Weymann, Dean A Regier
{"title":"Next-generation sequencing in oncology: challenges in economic evaluations.","authors":"Morgan Ehman, Jesman Punian, Deirdre Weymann, Dean A Regier","doi":"10.1080/14737167.2024.2388814","DOIUrl":"10.1080/14737167.2024.2388814","url":null,"abstract":"<p><strong>Introduction: </strong>Next-generation sequencing (NGS) identifies genetic variants to inform personalized treatment plans. Insufficient evidence of cost-effectiveness impedes the integration of NGS into routine cancer care. The complexity of personalized treatment challenges conventional economic evaluation. Clearly delineating challenges informs future cost-effectiveness analyses to better value and contextualize health, preference-, and equity-based outcomes.</p><p><strong>Areas covered: </strong>We conducted a scoping review to characterize the applied methods and outcomes of economic evaluations of NGS in oncology and identify existing challenges. We included 27 articles published since 2016 from a search of PubMed, Embase, and Web of Science. Identified challenges included defining the evaluative scope, managing evidentiary limitations including lack of causal evidence, incorporating preference-based utility, and assessing distributional and equity-based impacts. These challenges reflect the difficulty of generating high-quality clinical effectiveness and real-world evidence (RWE) for NGS-guided interventions.</p><p><strong>Expert opinion: </strong>Adapting methodological approaches and developing life-cycle health technology assessment (HTA) guidance using RWE is crucial for implementing NGS in oncology. Healthcare systems, decision-makers, and HTA organizations are facing a pivotal opportunity to adapt to an evolving clinical paradigm and create innovative regulatory and reimbursement processes that will enable more sustainable, equitable, and patient-oriented healthcare.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141878554","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}
Ali Darvishi, Ramin Abdi Dezfouli, Zohreh Ghasemian, Ali Akbari Sari, Mohammad Hossein Harirchian, Rajabali Daroudi
{"title":"The cost-utility analysis of adding ocrelizumab to the treatment plan for primary progressive multiple sclerosis in Iran.","authors":"Ali Darvishi, Ramin Abdi Dezfouli, Zohreh Ghasemian, Ali Akbari Sari, Mohammad Hossein Harirchian, Rajabali Daroudi","doi":"10.1080/14737167.2024.2388818","DOIUrl":"10.1080/14737167.2024.2388818","url":null,"abstract":"<p><strong>Background: </strong>This study evaluates the cost-effectiveness of adding ocrelizumab to supportive care for primary progressive multiple sclerosis (PPMS) in Iran.</p><p><strong>Research design and methods: </strong>Using a lifetime horizon from the payer's perspective, we developed a decision analytic model with Expanded Disability Status Scales (EDSS) as Markov health states while taking transition probabilities and treatment effects into account. Data were sourced from clinical trials and other literature. The target population was PPMS patients receiving either supportive care or ocrelizumab. We assessed cost- effectiveness through total costs, quality-adjusted life-years (QALYs), and the incremental cost- effectiveness ratio (ICER). Sensitivity analyses addressed uncertainties.</p><p><strong>Results: </strong>The addition of ocrelizumab to supportive care provided an incremental gain of 0.89 QALYs and an additional cost of US$76,771.34, resulting in an ICER of US$86,220.35 compared to supportive care, which is 5.2 times Iran's GDP per capita (US$16,557). Thus, ocrelizumab is not cost-effective at the threshold of one time GDP per capita. However, the probability of cost-effectiveness increases at higher thresholds. Sensitivity analyses confirmed the robustness of the results.</p><p><strong>Conclusion: </strong>While ocrelizumab is not cost-effective at the threshold of one-time GDP per capita, its clinical benefits are significant. Formulating healthcare policies for high-cost medications with low alternatives like ocrelizumab is essential.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141878555","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}
{"title":"Public value judgments about the criteria for reimbursement of medicines in South Korea.","authors":"Kyung-Bok Son","doi":"10.1080/14737167.2024.2388815","DOIUrl":"10.1080/14737167.2024.2388815","url":null,"abstract":"<p><strong>Objectives: </strong>This study quantified the public value (PV) of the criteria and sub-criteria in the current drug reimbursement systems in South Korea and examined sociodemographic factors that associated with PV.</p><p><strong>Methods: </strong>The Analytic Hierarchy Process (AHP) was used to quantify the PVs of criteria and sub-criteria. We developed a questionnaire to generate pairwise comparison matrices among criteria and sub-criteria. From 27 March to 1 April 2023, we recruited 1,000 study participants using a quota sampling method stratified by age, sex, and region based on Korean census data.</p><p><strong>Results: </strong>The PVs for the criteria were highest for clinical usefulness (28.5%), followed by cost-effectiveness (27.1%), budget impact (24.3%), and reimbursement in other countries (20.1%). The sociodemographic characteristics of the participants had a significant impact on the PVs of the criteria. Willingness to pay additional premiums for national health insurance was negatively associated with PV for clinical usefulness and cost-effectiveness and positively associated with PV for reimbursement in other countries.</p><p><strong>Conclusions: </strong>The public prioritized clinical usefulness and cost-effectiveness as the main criteria. However, the PVs of the criteria were divergent and associated with sociodemographic factors. Divergent public interests require an evidence-informed deliberative process for reimbursement decisions.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141874564","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}
Charlotte Sauter, Jacob Diemar, Viktorija Terebaite
{"title":"Usability of digital health devices in clinical trials.","authors":"Charlotte Sauter, Jacob Diemar, Viktorija Terebaite","doi":"10.1080/14737167.2024.2384545","DOIUrl":"10.1080/14737167.2024.2384545","url":null,"abstract":"","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141792325","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}
Pieralessandro Lasalvia, Eliana C Vásquez M, Jose Javier Arango Álvarez, Paola Garcia-Padilla
{"title":"Cost-effectiveness of dapagliflozin plus standard treatment compared to standard therapy for the management of chronic kidney disease in Colombia.","authors":"Pieralessandro Lasalvia, Eliana C Vásquez M, Jose Javier Arango Álvarez, Paola Garcia-Padilla","doi":"10.1080/14737167.2024.2382976","DOIUrl":"https://doi.org/10.1080/14737167.2024.2382976","url":null,"abstract":"<p><strong>Background: </strong>The DAPA-CKD study showed that dapagliflozin added to standard treatment reduced the risk of chronic kidney disease progression, and death from renal or cardiovascular causes compared to placebo.</p><p><strong>Objective: </strong>Assess the cost-effectiveness of dapagliflozin and standard treatment versus standard treatment alone for chronic kidney disease within the Colombian health system.</p><p><strong>Methods: </strong>We employed a Markov model based on the DAPA-CKD study, tailored to the Colombian scenario. The model forecasted hospitalizations for heart failure, overall and cardiovascular mortality, and chronic kidney disease progression over a 10-year horizon with a 5% discount rate.</p><p><strong>Results: </strong>Dapagliflozin combined with standard treatment is a cost-effective intervention in treating stage 2-4 CKD. In the base case, the ICER was US $5,366, below 1 GDP (US $6.558) per capita. This was consistent in the sensitivity analyses.</p><p><strong>Conclusion: </strong>Our study showed that dapagliflozin, when combined with standard treatment, is cost-effective against standard treatment alone, aligning with Colombia's willingness-to-pay threshold.</p>","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":null,"pages":null},"PeriodicalIF":1.8,"publicationDate":"2024-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141751486","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}