Yan Chen, Ella X Du, Manasvi Sundar, Keith A Betts, Xin Yin, Samantha Eiffert, Karen Beauchamp, Andrew Delgado, Lisa Rosenblatt
{"title":"Costs of Adverse Events in Patients with Advanced or Metastatic Renal Cell Carcinoma with First-Line Treatment.","authors":"Yan Chen, Ella X Du, Manasvi Sundar, Keith A Betts, Xin Yin, Samantha Eiffert, Karen Beauchamp, Andrew Delgado, Lisa Rosenblatt","doi":"10.1007/s41669-024-00534-2","DOIUrl":"https://doi.org/10.1007/s41669-024-00534-2","url":null,"abstract":"<p><strong>Aim: </strong>This study evaluated costs associated with adverse events (AEs) in previously untreated real-world patients with advanced renal cell carcinoma (aRCC) in the USA.</p><p><strong>Materials and methods: </strong>This retrospective longitudinal cohort study analyzed data from the Merative MarketScan Research Database (1 January 2014-30 September 2021). Adult patients with aRCC receiving first-line systemic treatments for aRCC (tyrosine kinase inhibitors [TKIs], or combination therapies of TKIs and immunotherapy) on or after the date of aRCC diagnosis were included. A total of 27 AEs of interest were included based on a review of product labels of the first-line treatments included in the study and identified using International Classification of Diseases, Ninth/Tenth Revision, Clinical Modification codes. Incremental costs associated with AEs between cases and controls (unadjusted and adjusted for relevant baseline characteristics) were estimated by two-part modeling. Analyses were performed over three AE cost assessment periods (7, 14, and 30 days).</p><p><strong>Results: </strong>The study included 1681 patients with aRCC (mean [standard deviation; SD] age, 60.8 [10.6] years; 73.1% male), of which 1542 (91.7%) had at least one AE. AEs were mostly diagnosed in the outpatient (OP) setting. For most AEs, cases had significantly higher unadjusted and adjusted costs than controls. Costs associated with AEs ranged from < 300 US dollars (USD) for proteinuria to nearly 60,000 USD for hypophosphatemia. Seventeen AEs had adjusted 30-day costs exceeding 10,000 USD; of these, nine (pancreatitis, acute kidney injury, dyspnea, hypotension, hyperkalemia, hypomagnesemia, hyponatremia, hypophosphatemia, and neutrophil decreased/neutropenia) had 30-day costs exceeding 20,000 USD.</p><p><strong>Limitations: </strong>The study was subject to limitations of all observational analyses of claims data (e.g., residual confounding). Observed cost differences may not have been solely attributable to an AE of interest. Study findings may not be generalizable to aRCC patient populations outside the USA.</p><p><strong>Conclusion: </strong>Most patients experienced at least one AE after initiation of first-line treatment with a TKI or combination therapies of TKIs and immunotherapy. There were substantial costs associated with AEs. Considering both safety and efficacy profiles when selecting optimal treatments can potentially mitigate healthcare costs for aRCC.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590803","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Rachel Houten, Mohammad Iqbal Hussain, Antony P Martin, Nick Ainsworth, Claudia Lameirinhas, Alexander W Coombs, Simon Toh, Christopher Rao, Edward St John
{"title":"Digital Versus Paper-Based Consent from the UK NHS Perspective: A Micro-costing Analysis.","authors":"Rachel Houten, Mohammad Iqbal Hussain, Antony P Martin, Nick Ainsworth, Claudia Lameirinhas, Alexander W Coombs, Simon Toh, Christopher Rao, Edward St John","doi":"10.1007/s41669-024-00536-0","DOIUrl":"https://doi.org/10.1007/s41669-024-00536-0","url":null,"abstract":"<p><strong>Background: </strong>The paper-based consent pathway can be associated with missing information, error, and inadequate patient comprehension. Digital consent addresses some of these limitations. However, limited research has been conducted to understand relative costs and consequences associated with adopting digital consent pathways. The aim of this study was to compare the relative costs of digital consent pathways with paper-based consent pathways in UK National Health Service (NHS) clinical practice.</p><p><strong>Method: </strong>A micro-costing study was conducted from the UK NHS perspective. Multi-stakeholder involvement contributed to understanding how the paper-based consent pathway varies by department and hospital setting. Sensitivity analyses were conducted to identify the key cost drivers and scenario analyses explored the effect of consent timing and hospital digital readiness. Potential advantages and disadvantages of digital consent were also considered, such as possible impacts associated with consent-related litigation.</p><p><strong>Results: </strong>The cost per consent episode is approximately £0.90 more expensive when completed on paper. The ordering or printing of paper consent forms, and the transportation of forms to storage and back to clinic are process steps that would not be necessary with digital consent. Sensitivity and scenario analyses indicated consultation duration had the greatest impact on the relative costs of both pathways. Per litigation claim prevented, an average of £201,590 could be saved.</p><p><strong>Conclusions: </strong>Digital consent is potentially cost saving for the NHS. Consent for elective procedures is recommended in advance of the day of surgery, and digital consent used in this scenario demonstrated the greatest savings. Consultation duration was estimated to have the greatest impact on the relative costs of both pathways, which should be a focus of further investigation.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583260","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-11-01Epub Date: 2024-07-30DOI: 10.1007/s41669-024-00513-7
Matilde Slot, Thomas Bøjer Rasmussen, Mette Nørgaard, Carsten Schade Larsen, Lars Holger Ehlers
{"title":"Evaluating Cost-Effectiveness of Antiretroviral Therapy over Time: A Cohort and Cost-Effectiveness Study.","authors":"Matilde Slot, Thomas Bøjer Rasmussen, Mette Nørgaard, Carsten Schade Larsen, Lars Holger Ehlers","doi":"10.1007/s41669-024-00513-7","DOIUrl":"10.1007/s41669-024-00513-7","url":null,"abstract":"<p><strong>Objective: </strong>To estimate the costs and cost-effectiveness of introducing highly active antiretroviral therapy (HAART) in Denmark based on real-world evidence for the three treatment eras pre-HAART (1985-1995), early HAART (1996-2005), and late HAART (2006-2017).</p><p><strong>Methods: </strong>We performed a cohort study using Danish clinical and administrative registries to estimate costs, quality-adjusted life-years (QALYs), and life-years (LY) gained per person living with human immunodeficiency virus (PLHIV) in three treatment eras. The study utilized Markov modeling for a health economic evaluation, which summarized inputs from real-world evidence and estimated the cost-effectiveness in 2017 prices of the introduction of HAART in Denmark. We performed deterministic and probabilistic sensitivity analyses to assess the robustness of the results.</p><p><strong>Results: </strong>The total annual costs per PLHIV increased with the introduction of HAART for the index year but decreased in the incremental years and the last year of life. The total lifetime discounted (and undiscounted) cost for an average PLHIV was €91,010 (€128,981) in pre-HAART, €103,130 (€199,062) in early HAART, and €126,317 (€254,964) in late HAART. The estimated incremental cost-effectiveness ratios showed that early HAART was cost-effective compared with pre-HAART with an incremental cost-effectiveness ratio (ICER) of €1378 per QALY, and that late HAART was cost-effective compared with early HAART with an ICER of €7385 per QALY. Sensitivity analyses confirmed cost-effectiveness in all scenarios.</p><p><strong>Conclusions: </strong>The introduction and implementation of HAART in Danish healthcare was cost-effective, and in some scenarios, even disruptive, i.e., led to both cheaper and more effective care of PLHIV.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499550/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141856173","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Health Technology Assessment Reports for Non-Oncology Medications in Canada from 2018 to 2022: Methodological Critiques on Manufacturers' Submissions and a Comparison between Manufacturer and Canadian Agency for Drugs and Technologies in Health (CADTH) Analyses.","authors":"Fatemeh Mirzayeh Fashami, Jean-Eric Tarride, Behnam Sadeghirad, Kimia Hariri, Amirreza Peyrovinasab, Mitchell Levine","doi":"10.1007/s41669-024-00511-9","DOIUrl":"10.1007/s41669-024-00511-9","url":null,"abstract":"<p><strong>Introduction: </strong>Identifying key differences between manufacturers' submitted analysis and economic reanalysis by the Canadian Agency for Drugs and Technologies in Health (CADTH) is an important step toward understanding reimbursement recommendations. We compared economic values reported in manufacturers' analysis with the CADTH reanalysis and also assessed methodological critiques.</p><p><strong>Methods: </strong>Two reviewers extracted data from the clinical and economic reports in publicly available CADTH reports from 2018 to 2022. We used the Wilcoxon rank-sum test to assess the difference between mean economic values, and the Chi-square test to assess the association between the CADTH critique final recommendations.</p><p><strong>Results: </strong>Of the total submissions, 99.4% included effectiveness critiques, 88.8% included model structure critiques, 69.1% included utility score critiques, and 78.7% included cost critiques. The median incremental cost-utility ratio (ICUR) in the manufacturers' analyses was $138,658/quality-adjusted life-year (QALY), 2.5-fold lower than the CADTH's reanalysis at $380,251/QALY (p < 0.001). The median CADTH reanalysis for 3-year budget impact analysis (BIA) was $4,575,102, which was 27% higher than the manufacturers' submitted 3-year BIA (p < 0.001). CADTH requested a price reduction for 95% of all submissions, and the median price reduction request was 63.5%. In 2021 and 2022, the willingness-to-pay threshold identified in CADTH reports remained constant at $50,000 per QALY gained for all medications.</p><p><strong>Conclusion: </strong>There was high frequency of CADTH critiques on manufacturers' submissions in all four aspects of economic submissions: effectiveness, cost, utility score and structure. We observed a higher median incremental cost and lower median incremental QALYs in the CADTH reanalysis compared with the manufacturers' submissions. The resulting higher ICUR in the CADTH reanalysis often leads to a recommendation that the manufacturer needs to reduce its price. The 3-year budget impact was higher in the CADTH reanalyses compared with manufacturers' submissions.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499573/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141893993","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-11-01Epub Date: 2024-08-05DOI: 10.1007/s41669-024-00515-5
Maria Farris, Stephen Goodall, Richard De Abreu Lourenco, Brendan Mulhern, Kathleen Manipis, Elena Meshcheriakova, Milena Lewandowska
{"title":"Estimating Australian Population Utilities for Inherited Retinal Disease Using Time Trade-Off.","authors":"Maria Farris, Stephen Goodall, Richard De Abreu Lourenco, Brendan Mulhern, Kathleen Manipis, Elena Meshcheriakova, Milena Lewandowska","doi":"10.1007/s41669-024-00515-5","DOIUrl":"10.1007/s41669-024-00515-5","url":null,"abstract":"<p><strong>Purpose: </strong>Inherited retinal disease (IRD) causes progressive loss of visual function, degenerating towards complete blindness. Economic evaluation of gene therapies for rare forms of genetic IRDs have had to rely on health-related quality of life (HR-QoL) estimates from other diseases because there is limited data available for such a rare condition. This study aimed to estimate Australian societal-based utility values for IRD health states that can be used in cost-utility analyses (CUA) using a time trade-off (TTO) protocol adapted from a UK study.</p><p><strong>Methods: </strong>The EuroQol Valuation Technology (EQVT) protocol composite TTO (cTTO) framework was followed, which includes worse-than-death (WTD) states and quality control (QC) measures. Preferences were collected from a general population sample of 110 Australian adult participants. Five health state vignettes from the UK study which had been validated with patients and clinicians were presented randomly to participants during videoconferencing (VC) interviews with one of four interviewers. Technical and protocol feasibility were assessed in a pilot of 10 interviews. QC measures were used to monitor interviewers' performance during the study.</p><p><strong>Results: </strong>One participant withdrew consent. The final analysis was conducted on 109 respondents (including 4 non-traders). The average time to complete the interview was 44.2 minutes (SD 8.7). Participants reported mean visual analogue scale (VAS) scores between 63.15 for 'moderate impairment' and 17.98 for 'hand motion' to 'no light perception'. Mean health state utilities (HSU) varied between 0.76 (SD 0.26) in 'moderate impairment', and 0.20 (SD 0.58) in 'hand motion' to 'no light perception'. Of all HSU evaluations, 14% were considered WTD which most commonly occurred in the most severe visually impaired health state.</p><p><strong>Conclusion: </strong>This study provides valuable information on HSUs across a range of IRD health states from the Australian general population perspective. The utilities obtained in this study can be used as inputs into CUA of IRD therapies.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499549/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141889886","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-11-01Epub Date: 2024-08-16DOI: 10.1007/s41669-024-00517-3
Modou Diop, David Epstein
{"title":"A Systematic Review of the Impact of Spinal Cord Injury on Costs and Health-Related Quality of Life.","authors":"Modou Diop, David Epstein","doi":"10.1007/s41669-024-00517-3","DOIUrl":"10.1007/s41669-024-00517-3","url":null,"abstract":"<p><strong>Objective: </strong>To systematically review the health-related quality of life (HRQoL) burden and costs of spinal cord injury (SCI) on health services, patients and wider society.</p><p><strong>Methods: </strong>A systematic review guided by the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Statement was conducted in March 2021 through Scopus, PubMed and Embase databases. Inclusion criteria were quantitative studies on SCI reporting healthcare costs, social costs and/or HRQoL measured with the Euroqol EQ-5D or Short-Form 36. Risk of bias was assessed using the QualSyst tool. Descriptive analyses, random-effects direct meta-analysis and random-effects meta-regression were conducted.</p><p><strong>Results: </strong>A total of 67 studies were eligible for inclusion. SCI individuals tend to report higher HRQoL in mental than physical dimensions of the Short-Form 36. Neurological level of SCI negatively affects HRQoL. Cross-sectional studies find employment is associated with better HRQoL, but the effect is not observed in longitudinal studies. The estimated lifetime expenditure per individual with SCI ranged from US$0.7 million to US$2.5 million, with greater costs associated with earlier age at injury, neurological level, United States of America healthcare setting and the inclusion of non-healthcare items in the study.</p><p><strong>Conclusions: </strong>SCI is associated with low HRQoL on mobility and physical dimensions. Mental health scores tend to be greater than physical scores, and most dimensions of HRQoL appear to improve over time, at least over the first year. SCI is associated with high costs which vary by country.</p><p><strong>Clinical trials registration: </strong>This review was registered in PROSPERO (registration number: CRD42021235801).</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499558/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141988563","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-11-01Epub Date: 2024-08-28DOI: 10.1007/s41669-024-00520-8
Danny Gibson, Neil Branscombe, Neil Martin, Andrew Menzies-Gow, Priya Jain, Katherine Padgett, Florian Yeates
{"title":"Modelling Adverse Events in Patients Receiving Chronic Oral Corticosteroids in the UK.","authors":"Danny Gibson, Neil Branscombe, Neil Martin, Andrew Menzies-Gow, Priya Jain, Katherine Padgett, Florian Yeates","doi":"10.1007/s41669-024-00520-8","DOIUrl":"10.1007/s41669-024-00520-8","url":null,"abstract":"<p><strong>Background: </strong>Oral corticosteroids (OCS) are effective anti-inflammatory agents used across a range of conditions. However, substantial evidence associates their use with increased risks for adverse events (AEs), causing high burden on healthcare resources. Emerging biologics present as alternative agents, enabling the reduction of OCS use. However, current modelling approaches may underestimate their effects by not capturing OCS-sparing effects. In this study, we present a modelling approach designed to capture the health economic benefits of OCS-sparing regimens and agents.</p><p><strong>Methods: </strong>We developed a disease-agnostic model using a UK health technology assessment (HTA) perspective, with discounting of 3.5% for costs and outcomes, a lifetime horizon, and 4-week cycle length. The model structure included type 2 diabetes mellitus, established cardiovascular disease, and osteoporosis as key AEs and drivers of morbidity and mortality, as well as capturing transient events. Quality-adjusted life-years (QALYs), life-years, and costs were determined for OCS-only and OCS-sparing treatment arms. Outcomes were determined using baseline 50% OCS-sparing, considering several OCS average daily doses (5, 10, 15 mg).</p><p><strong>Results: </strong>A treatment regimen with 50% OCS dose-sparing led to lifetime incremental cost savings per patient of £1107 (95% confidence interval £1014-£1229) at 5 mg, £2403 (£2203-£2668) at 10 mg, and £19,501 (£748-£51,836) at 15 mg. Patients also gained 0.033 (0.030-0.036) to 0.356 (0.022-2.404) QALYs dependent on dose. The benefits of OCS sparing were long-term, plateauing after 35-40 years of treatment.</p><p><strong>Conclusions: </strong>We present a modelling approach that captures additional long-term health economic benefits from OCS sparing that would otherwise be missed from current modelling approaches. These results may help inform future decision making for emerging OCS-sparing therapeutics by comparing them against the cost of such treatments.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499505/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142081127","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Artificial-Intelligence Cloud-Based Platform to Support Shared Decision-Making in the Locoregional Treatment of Breast Cancer: Protocol for a Multidimensional Evaluation Embedded in the CINDERELLA Clinical Trial.","authors":"Ludovica Borsoi, Elisabetta Listorti, Oriana Ciani","doi":"10.1007/s41669-024-00519-1","DOIUrl":"10.1007/s41669-024-00519-1","url":null,"abstract":"<p><strong>Background: </strong>Shared decision-making (SDM) plays a crucial role in breast cancer care by empowering patients and reducing decision regret. Patient decision aids (PtDAs) are valuable tools for facilitating SDM, now available in digital and artificial intelligence (AI)-powered formats to offer increasingly personalized contents. The ongoing CINDERELLA clinical trial (ClinicalTrials.gov: NCT05196269) evaluates an innovative AI cloud-based approach using a web platform and a mobile application (CINDERELLA APProach) versus the conventional approach to support SDM in breast cancer patients undergoing locoregional treatment. This protocol outlines a trial-based multidimensional evaluation, encompassing economic, financial, implementability, and environmental considerations associated with the CINDERELLA APProach.</p><p><strong>Methods: </strong>A within-trial cost-consequence and cost-utility analysis from a societal perspective will be performed using patient-level data on outcomes and resource use. The latter will be valued in monetary terms using country-specific unit costs or patient valuations. A budget impact analysis will be performed over 1 and 5 years from the budget holder perspectives. The CINDERELLA APProach implementability will be assessed through an evaluation of its usability, acceptability, organizational impact, and overall feasibility. The environmental impact will be quantitatively assessed across several dimensions, such as quantity, appropriateness, and emissions, supplemented by qualitative insights. Overall, data for the evaluation will be gathered from patient questionnaires, interviews with patients and managers, focus groups with healthcare professionals, and app electronic data.</p><p><strong>Discussion: </strong>A thorough understanding of the broad consequences of the CINDERELLA APProach may foster its successful translation into real-world settings, hopefully benefiting breast cancer patients and clinical practice.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499581/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292817","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomics OpenPub Date : 2024-11-01Epub Date: 2024-10-03DOI: 10.1007/s41669-024-00528-0
Ellen Kim DeLuca, Ann Chen Wu, Kurt D Christensen, Davene R Wright, Jennifer Yeh, Hadley Stevens Smith
{"title":"Modernizing Newborn Screening in the Genomic Era: Importance of Health-Related Quality of Life.","authors":"Ellen Kim DeLuca, Ann Chen Wu, Kurt D Christensen, Davene R Wright, Jennifer Yeh, Hadley Stevens Smith","doi":"10.1007/s41669-024-00528-0","DOIUrl":"10.1007/s41669-024-00528-0","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499486/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142366102","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Cost-Effectiveness of Dupilumab and Oral Janus Kinase Inhibitors for the Treatment of Moderate-to-Severe Atopic Dermatitis in Singapore.","authors":"Clarence Ong, Jamaica Briones, Zhi Zhen Lim, Nisha Suyien Chandran, Haur Yueh Lee, Benny Kaihui Li, Yik Weng Yew, Hwee-Lin Wee","doi":"10.1007/s41669-024-00507-5","DOIUrl":"10.1007/s41669-024-00507-5","url":null,"abstract":"<p><strong>Background: </strong>Atopic dermatitis (AD) affects both adults and children, impacting their quality of life and productivity; however, traditional systemic treatments such as cyclosporine have limitations. Emerging novel systemic interventions, including monoclonal antibodies and Janus kinase (JAK) inhibitors, have been shown to improve patient outcomes.</p><p><strong>Objective: </strong>This study evaluates the cost-effectiveness of novel systemic interventions for moderate-to-severe AD in adults compared with the best supportive care (BSC) in Singapore.</p><p><strong>Methods: </strong>The economic evaluation used a hybrid model consisting of a decision tree and Markov model. Treatment responses at 16 weeks were based on a network meta-analysis that was developed specifically for this study. Long-term response, discontinuation rates, episodes of flares and treatment-emergent adverse events were obtained from key dupilumab, abrocitinib, baricitinib and upadacitinib trials. The study had a 5-year time horizon and considered the healthcare payer's perspective. Sensitivity and scenario analyses were performed as well.</p><p><strong>Results: </strong>Baricitinib 4 mg and 2 mg have the lowest incremental cost-effectiveness ratios, at Singapore dollars (S$) 60,730/quality-adjusted life-year (QALY) and S$66,842/QALY, respectively. Upadacitinib 30 mg offers the highest incremental QALY gain, while baricitinib 2 mg offers the least. The cost of the intervention drugs accounted for the highest proportion of the overall expenses (68-93%) for those in the maintenance state. Other influential factors within the model included (1) the incremental utility derived from intervention response; (2) the probability of achieving Eczema Area and Severity Index 75 (EASI-75) with BSC; and (3) the relative risk of achieving EASI-75 with the interventions. In a scenario where the cost of all drugs is matched to the lowest-priced drug, the top three cost-effectiveness interventions are dupilumab, upadacitinib 30 mg and abrocitinib 200 mg, respectively.</p><p><strong>Conclusion: </strong>The interventions are not found to be cost-effective at their existing prices when compared with BSC. Ideally, a composite score of treatment success and quality-of-life scores ought to be included, but such data were unavailable. Future research should consider conditional discontinuation data and long-term outcomes when such data become accessible.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499508/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141603925","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}