PharmacoEconomics Open最新文献

{"title":"Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany.","authors":"Niklaus Meier, Hendrik Fuchs, Katya Galactionova, Cedric Hermans, Mark Pletscher, Matthias Schwenkglenks","doi":"10.1007/s41669-024-00480-z","DOIUrl":"10.1007/s41669-024-00480-z","url":null,"abstract":"<p><strong>Background and objective: </strong>Haemophilia B is a rare genetic disease that is caused by a deficiency of coagulation factor IX (FIX) in the blood and leads to internal and external bleeding. Under the current standard of care, haemophilia is treated either prophylactically or on-demand via intravenous infusions of FIX. These treatment strategies impose a high burden on patients and health care systems as haemophilia B requires lifelong treatment, and FIX is costly. Etranacogene dezaparvovec (ED) is a gene therapy for haemophilia B that has been recently approved by the United States Food and Drug Administration and has received a recommendation for conditional marketing authorization by the European Medicines Agency. We aimed to examine the cost-effectiveness of ED versus extended half-life FIX (EHL-FIX) prophylaxis for moderate-to-severe haemophilia B from a German health care payer perspective.</p><p><strong>Methods: </strong>A microsimulation model was implemented in R. The model used data from the ED phase 3 clinical trial publication and further secondary data sources to simulate and compare patients receiving ED or EHL-FIX prophylaxis over a lifetime horizon, with the potential for ED patients to switch treatment to EHL-FIX prophylaxis when the effectiveness of ED waned. Primary outcomes of this analysis included discounted total costs, discounted quality-adjusted life years (QALYs), incremental cost-effectiveness, and the incremental net monetary benefit. The annual discount rate for costs and effects was 3%. Uncertainty was examined via probabilistic analysis and additional univariate sensitivity analyses.</p><p><strong>Results: </strong>Probabilistic analysis indicated that patients treated with ED instead of EHL-FIX prophylaxis gained 0.50 QALYs and experienced cost savings of EUR 1,179,829 at a price of EUR 1,500,000 per ED treatment. ED was the dominant treatment strategy. At a willingness to pay of EUR 50,000/QALY, the incremental net monetary benefit amounted to EUR 1,204,840.</p><p><strong>Discussion: </strong>Depending on the price, ED can save costs and improve health outcomes of haemophilia patients compared with EHL-FIX prophylaxis, making it a potentially cost-effective alternative. These results are uncertain due to a lack of evidence regarding the long-term effectiveness of ED.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"373-387"},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058170/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140194336","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 Analysis of Digital Breast Tomosynthesis Added to Synthetic Mammography in Breast Cancer Screening in Brazil.","authors":"Henrique Lima Couto, Ludmila Peres Gargano, Vilmar Marques de Oliveira, Bertha Andrade Coelho, Eduardo Carvalho Pessoa, Augusto Tufi Hassan, Agnaldo Lopes Silva, Linei Augusta Brolini Delle Urban, Luciano Chala Fernandes, Nisha Sharma, Ritse Mann, Stuart A McIntosh, Fernando Zanghelini","doi":"10.1007/s41669-023-00470-7","DOIUrl":"10.1007/s41669-023-00470-7","url":null,"abstract":"<p><strong>Background: </strong>Literature meta-analysis results show that digital breast tomosynthesis (DBT) combined with synthesized two-dimensional (s2D) mammograms can reduce recalls and improve breast cancer detection. Uncertainty regarding the screening of patients with breast cancer presents a health economic challenge, both in terms of healthcare resource use and quality of life impact on patients.</p><p><strong>Objective: </strong>This study aims to estimate the cost effectiveness of DBT + s2D versus digital mammography (DM) used in a biennial breast cancer screening setting of women aged 40-69 years with scattered areas of fibroglandular breast density and heterogeneous dense breasts in the Brazilian supplementary health system.</p><p><strong>Methods: </strong>A cost-effectiveness analysis was performed on the basis of clinical data obtained from a systematic review with meta-analysis performed to evaluate the analytical validity and clinical utility of DBT + s2D compared with DM. The search was conducted in the PubMed, Cochrane Library and Embase databases, with the main descriptors of the technology, a comparator, and the clinical condition in question, on 9 June 2022. The hybrid economic model (decision tree plus Markov model) simulated costs and outcomes over a lifetime for women aged 40-69 years with scattered areas of fibroglandular breast density and heterogeneous dense breasts. We analyzed incremental cost-effectiveness ratio (ICER) to measure the incremental cost difference per quality-adjusted life year (QALY) of adding DBT + s2D to breast cancer screening.</p><p><strong>Results: </strong>DBT + s2D incurred a cost saving of € 954.02 per patient, in the time horizon of 30 years, compared with DM, and gained 5.1989 QALYs, which would be considered a dominant intervention. These results were confirmed in sensitivity analyses.</p><p><strong>Conclusion: </strong>Switching from DM to biennial DBT + s2D was cost effective. Furthermore, reductions in false-positive recall rates should also be considered in decision making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"403-416"},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058155/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139486292","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":"An Economic Evaluation of a Streamlined Day-Case Atrial Fibrillation Ablation Protocol and Conventional Cryoballoon Ablation versus Antiarrhythmic Drugs in a UK Paroxysmal Atrial Fibrillation Population.","authors":"Joe W E Moss, Derick Todd, Lukasz Grodzicki, Beatrice Palazzolo, Richard Mattock, Stuart Mealing, Maxim Souter, Benedict Brown, Tom Bromilow, Damian Lewis, James McCready, Muzahir Tayebjee, Ewen Shepherd, Thiagarajah Sasikaran, Clare Coyle, Eleni Ismyrloglou, Nicholas A Johnson, Prapa Kanagaratnam","doi":"10.1007/s41669-023-00471-6","DOIUrl":"10.1007/s41669-023-00471-6","url":null,"abstract":"<p><strong>Background and aims: </strong>Symptom control for atrial fibrillation can be achieved by catheter ablation or drug therapy. We assessed the cost effectiveness of a novel streamlined atrial fibrillation cryoballoon ablation protocol (AVATAR) compared with optimised antiarrhythmic drug (AAD) therapy and a conventional catheter ablation protocol, from a UK National Health Service (NHS) perspective.</p><p><strong>Methods: </strong>Data from the AVATAR study were assessed to determine the cost effectiveness of the three protocols in a two-step process. In the first stage, statistical analysis of clinical efficacy outcomes was conducted considering either a three-way comparison (AVATAR vs. conventional ablation vs. optimised AAD therapies) or a two-way comparison (pooled ablation protocol data vs. optimised AAD therapies). In the second stage, models assessed the cost effectiveness of the protocols. Costs and some of the clinical inputs in the models were derived from within-trial cost analysis and published literature. The remaining inputs were derived from clinical experts.</p><p><strong>Results: </strong>No significant differences between the ablation protocols were found for any of the clinical outcomes used in the model. Results of a within-trial cost analysis show that AVATAR is cost-saving (£1279 per patient) compared with the conventional ablation protocol. When compared with optimised AAD therapies, AVATAR (pooled conventional and AVATAR ablation protocols efficacy) was found to be more costly while offering improved clinical benefits. Over a lifetime time horizon, the incremental cost-effectiveness ratio of AVATAR was estimated as £21,046 per quality-adjusted life-year gained (95% credible interval £7086-£71,718).</p><p><strong>Conclusions: </strong>The AVATAR streamlined protocol is likely to be a cost-effective option versus both conventional ablation and optimised AAD therapy in the UK NHS healthcare setting.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"417-429"},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058164/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139513292","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":"A Canadian Simulation Model for Major Depressive Disorder: Study Protocol.","authors":"Shahzad Ghanbarian, Gavin W K Wong, Mary Bunka, Louisa Edwards, Sonya Cressman, Tania Conte, Sandra Peterson, Rohit Vijh, Morgan Price, Christian Schuetz, David Erickson, Linda Riches, Ginny Landry, Kim McGrail, Jehannine Austin, Stirling Bryan","doi":"10.1007/s41669-024-00481-y","DOIUrl":"10.1007/s41669-024-00481-y","url":null,"abstract":"<p><strong>Background: </strong>Major depressive disorder (MDD) is a common, often recurrent condition and a significant driver of healthcare costs. People with MDD often receive pharmacological therapy as the first-line treatment, but the majority of people require more than one medication trial to find one that relieves symptoms without causing intolerable side effects. There is an acute need for more effective interventions to improve patients' remission and quality of life and reduce the condition's economic burden on the healthcare system. Pharmacogenomic (PGx) testing could deliver these objectives, using genomic information to guide prescribing decisions. With an already complex and multifaceted care pathway for MDD, future evaluations of new treatment options require a flexible analytic infrastructure encompassing the entire care pathway. Individual-level simulation models are ideally suited for this purpose. We sought to develop an economic simulation model to assess the effectiveness and cost effectiveness of PGx testing for individuals with major depression. Additionally, the model serves as an analytic infrastructure, simulating the entire patient pathway for those with MDD.</p><p><strong>Methods and analysis: </strong>Key stakeholders, including patient partners, clinical experts, researchers, and modelers, designed and developed a discrete-time microsimulation model of the clinical pathways of adults with MDD in British Columbia (BC), including all publicly-funded treatment options and multiple treatment steps. The Simulation Model of Major Depression (SiMMDep) was coded with a modular approach to enhance flexibility. The model was populated using multiple original data analyses conducted with BC administrative data, a systematic review, and an expert panel. The model accommodates newly diagnosed and prevalent adult patients with MDD in BC, with and without PGx-guided treatment. SiMMDep comprises over 1500 parameters in eight modules: entry cohort, demographics, disease progression, treatment, adverse events, hospitalization, costs and quality-adjusted life-years (payoff), and mortality. The model predicts health outcomes and estimates costs from a health system perspective. In addition, the model can incorporate interactive decision nodes to address different implementation strategies for PGx testing (or other interventions) along the clinical pathway. We conducted various forms of model validation (face, internal, and cross-validity) to ensure the correct functioning and expected results of SiMMDep.</p><p><strong>Conclusion: </strong>SiMMDep is Canada's first medication-specific, discrete-time microsimulation model for the treatment of MDD. With patient partner collaboration guiding its development, it incorporates realistic care journeys. SiMMDep synthesizes existing information and incorporates provincially-specific data to predict the benefits and costs associated with PGx testing. These predictions estimate the effectiveness, ","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"493-505"},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058136/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140288710","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-Related Quality of Life and Economic Burden Among Hospitalized Children with Hand, Foot, and Mouth Disease: A Multiregional Study in China.","authors":"Ting Zhou, Hongfei Hu, Junyang Gao, Hongjie Yu, Mark Jit, Pei Wang","doi":"10.1007/s41669-023-00468-1","DOIUrl":"10.1007/s41669-023-00468-1","url":null,"abstract":"<p><strong>Background: </strong>Hand, foot, and mouth disease (HFMD) is an infectious disease with high morbidity and mortality rates among children under 5 years old. This study aimed to explore the health-related quality of life (HRQOL), economic burden, and related influencing factors among Chinese HFMD patients.</p><p><strong>Methods: </strong>From January to October 2019, a longitudinal cohort study of 296 hospitalized patients (≤ 5 years old) with HFMD and their guardians was conducted using the proxy version of the 5-level EQ-5D-Y (EQ-5D-Y-5L, Y-5L) in face-to-face interviews in Shanghai, Zhengzhou, and Kunming, representing three regions with different economic development levels. Multiple linear regression was used to explore the factors associated with HRQOL and costs.</p><p><strong>Results: </strong>The mean Y-5L health utility score (HUS) (standard deviation, SD), and visual analogue scale (VAS) score (SD) were 0.730 (0.140) and 60.33 (16.52) at admission and increased to 0.920 (0.120) and 89.95 (11.88) at discharge, respectively. The children from Shanghai had the lowest HUSs at admission and had the best health improvement. The mean hospitalization cost and total cost were 4037 CNY and 5157 CNY, respectively. The children from Shanghai had the highest hospitalization cost (4559 CNY) and total cost (5491 CNY). Multiple regression analysis suggested that medical insurance status, type of employment, residence type, and religious status were significantly associated with the baseline HUS and improvement in the HUS after treatment. Region, loss of work time, and length of stay had a significant impact on the hospitalization cost and total cost.</p><p><strong>Conclusion: </strong>Our findings demonstrate that HFMD could lead to poor HRQOL and the economic burden varies in different regions in China. Many pediatric patients still have physical or mental health problems shortly after treatment.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"459-469"},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058149/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139403930","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":"Economic Evaluation of a Personalized Nutrition Plan Based on Omic Sciences Versus a General Nutrition Plan in Adults with Overweight and Obesity: A Modeling Study Based on Trial Data in Denmark.","authors":"Milanne Maria Johanna Galekop, Carin Uyl-de Groot, William Ken Redekop","doi":"10.1007/s41669-023-00461-8","DOIUrl":"10.1007/s41669-023-00461-8","url":null,"abstract":"<p><strong>Background: </strong>Since there is no diet that is perfect for everyone, personalized nutrition approaches are gaining popularity to achieve goals such as the prevention of obesity-related diseases. However, appropriate choices about funding and encouraging personalized nutrition approaches should be based on sufficient evidence of their effectiveness and cost-effectiveness. In this study, we assessed whether a newly developed personalized plan (PP) could be cost-effective relative to a non-personalized plan in Denmark.</p><p><strong>Methods: </strong>Results of a 10-week randomized controlled trial were combined with a validated obesity economic model to estimate lifetime cost-effectiveness. In the trial, the intervention group (PP) received personalized home-delivered meals based on metabolic biomarkers and personalized behavioral change messages. In the control group these meals and messages were not personalized. Effects were measured in body mass index (BMI) and quality of life (EQ-5D-5L). Costs [euros (€), 2020] were considered from a societal perspective. Lifetime cost-effectiveness was assessed using a multi-state Markov model. Univariate, probabilistic sensitivity, and scenario analyses were performed.</p><p><strong>Results: </strong>In the trial, no significant differences were found in the effectiveness of PP compared with control, but wide confidence intervals (CIs) were seen [e.g., BMI (-0.07, 95% CI -0.51, 0.38)]. Lifetime estimates showed that PP increased costs (€520,102 versus €518,366, difference: €1736) and quality-adjusted life years (QALYs) (15.117 versus 15.106, difference: 0.011); the incremental cost-utility ratio (ICUR) was therefore high (€158,798 to gain one QALY). However, a 20% decrease in intervention costs would reduce the ICUR (€23,668 per QALY gained) below an unofficial gross domestic product (GDP)-based willingness-to-pay threshold (€47,817 per QALY gained).</p><p><strong>Conclusion: </strong>On the basis of the willingness-to-pay threshold and the non-significant differences in short-term effectiveness, PP may not be cost-effective. However, scaling up the intervention would reduce the intervention costs. Future studies should be larger and/or longer to reduce uncertainty about short-term effectiveness.</p><p><strong>Trial registration number: </strong>ClinicalTrials.gov registry (NCT04590989).</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"313-331"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10883904/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138808413","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-Utility Analysis of Total Ankle Replacement Compared with Ankle Arthrodesis for Patients Aged 50-85 Years with End-Stage Ankle Osteoarthritis: The TARVA Study.","authors":"Andrew J Goldberg, Ekaterina Bordea, Kashfia Chowdhury, Iva Hauptmannova, James Blackstone, Deirdre Brooking, Elizabeth L Deane, Stephen Bendall, Andrew Bing, Chris Blundell, Sunil Dhar, Andrew Molloy, Steve Milner, Mike Karski, Steve Hepple, Malik Siddique, David T Loveday, Viren Mishra, Paul Cooke, Paul Halliwell, David Townshend, Simon S Skene, Caroline J Doré","doi":"10.1007/s41669-023-00449-4","DOIUrl":"10.1007/s41669-023-00449-4","url":null,"abstract":"<p><strong>Background: </strong>Patients with end-stage ankle osteoarthritis suffer from reduced mobility and quality of life and the main surgical treatments are total ankle replacement (TAR) and ankle fusion (AF).</p><p><strong>Objectives: </strong>Our aim was to calculate the mean incremental cost per quality-adjusted life-year (QALY) of TAR compared with AF in patients with end-stage ankle osteoarthritis, over 52 weeks and over the patients' lifetime.</p><p><strong>Method: </strong>We conducted a cost-utility analysis of 282 participants from 17 UK centres recruited to a randomised controlled trial (TARVA). QALYs were calculated using index values from EQ-5D-5L. Resource use information was collected from case report forms and self-completed questionnaires. Primary analysis was within-trial analysis from the National Health Service (NHS) and Personal Social Services (PSS) perspective, while secondary analyses were within-trial analysis from wider perspective and long-term economic modelling. Adjustments were made for baseline resource use and index values.</p><p><strong>Results: </strong>Total cost at 52 weeks was higher in the TAR group compared with the AF group, from the NHS and PSS perspective (mean adjusted difference £2539, 95% confidence interval [CI] £1142, £3897). The difference became very small from the wider perspective (£155, 95% CI -  £1947, £2331). There was no significant difference between TAR and AF in terms of QALYs (mean adjusted difference 0.02, 95% CI -  0.015, 0.05) at 52 weeks post-operation. The incremental cost-effectiveness ratio (ICER) was £131,999 per QALY gained 52 weeks post-operation. Long-term economic modelling resulted in an ICER of £4200 per QALY gained, and there is a 69% probability of TAR being cost effective at a cost-effectiveness threshold of £20,000 per QALY gained.</p><p><strong>Conclusion: </strong>TAR does not appear to be cost effective over AF 52 weeks post-operation. A decision model suggests that TAR can be cost effective over the patients' lifetime but there is a need for longer-term prospectively collected data. Clinical trial registration ISRCTN60672307 and ClinicalTrials.gov NCT02128555.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"235-249"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884388/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139378108","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 Analysis of Abrocitinib Compared with Other Systemic Treatments for Severe Atopic Dermatitis in Spain.","authors":"Rosa María Romero Jiménez, Pedro Herranz Pinto, Minia Campos Domínguez, Susana Aceituno Mata, Alba Bellmunt, Miriam Prades, Daniel Arumi, Irene Hernández-Martín, Valeria Herrera-Lasso, Noelia Llevat, Alfonso De Lossada Juste, Francisco José Rebollo Laserna","doi":"10.1007/s41669-023-00459-2","DOIUrl":"10.1007/s41669-023-00459-2","url":null,"abstract":"<p><strong>Introduction: </strong>Atopic dermatitis (AD) is a chronic, inflammatory skin disease characterized by itchy, painful, and dry skin. Despite the great number of available therapies, economic evaluations are still needed to provide evidence on their cost efficiency. This research aimed to evaluate the cost effectiveness of the Janus kinase (JAK) inhibitor abrocitinib (200 mg) compared with dupilumab (300 mg), tralokinumab (300 mg), baricitinib (2 and 4 mg), and upadacitinib (15 and 30 mg) for the treatment of patients with severe AD from the Spanish National Health System (NHS) perspective.</p><p><strong>Methods: </strong>A hybrid model consisting of a decision tree linked to a Markov model was developed to estimate costs, quality-adjusted life-years (QALYs), total years in response and incremental cost-per-QALY gained (willingness-to-pay [WTP] threshold: €25,000/QALY). Adults with severe AD entered the decision tree and response (75% reduction in baseline Eczema Area and Severity Index score, EASI-75) was considered at 16 and 52 weeks. After this time, patients entered the Markov model (remainder of the 10-year time horizon), which consisted of three health states: maintenance with active therapy, subsequent treatment, or death. All costs were presented in 2022 euros (€). Additionally, cost per number-needed-to-treat (NNT) was calculated for abrocitinib and dupilumab based on a head-to-head post-hoc analysis.</p><p><strong>Results: </strong>Abrocitinib 200 mg was dominant (i.e., lower incremental costs and higher incremental benefit) compared with all studied alternatives (dupilumab 300 mg, tralokinumab 300 mg, baricitinib 2 and 4 mg, upadacitinib 15 and 30 mg) with a QALYs gain of 0.49, 0.60, 0.64, 0.43, 0.45, and 0.08, respectively, and per-person costs savings of €22,097, €24,140, €14,825, €7,116, €12,805, and €45,189, respectively. Considering the WTP threshold, abrocitinib was dominant or cost effective compared with all alternatives for most simulations. Additionally, abrocitinib was dominant compared with all alternatives when evaluating the cost effectiveness over a 5-year time horizon. NNT showed that abrocitinib was dominant versus dupilumab.</p><p><strong>Conclusions: </strong>The results of the study show that abrocitinib is a cost-effective therapy compared with other JAK inhibitors and biological therapies from the Spanish NHS perspective.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"291-302"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884369/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139486289","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 State Utilities Associated with False-Positive Cancer Screening Results.","authors":"Louis S Matza, Timothy A Howell, Eric T Fung, Sam M Janes, Michael Seiden, Allan Hackshaw, Lincoln Nadauld, Hayley Karn, Karen C Chung","doi":"10.1007/s41669-023-00443-w","DOIUrl":"10.1007/s41669-023-00443-w","url":null,"abstract":"<p><strong>Introduction: </strong>Early cancer detection can significantly improve patient outcomes and reduce mortality rates. Novel cancer screening approaches, including multi-cancer early detection tests, have been developed. Cost-utility analyses will be needed to examine their value, and these models require health state utilities. The purpose of this study was to estimate the disutility (i.e., decrease in health state utility) associated with false-positive cancer screening results.</p><p><strong>Methods: </strong>In composite time trade-off interviews using a 1-year time horizon, UK general population participants valued 10 health state vignettes describing cancer screening with true-negative or false-positive results. Each false-positive vignette described a common diagnostic pathway following a false-positive result suggesting lung, colorectal, breast, or pancreatic cancer. Every pathway ended with a negative result (no cancer detected). The disutility of each false positive was calculated as the difference between the true-negative and each false-positive health state, and because of the 1-year time horizon, each disutility can be interpreted as a quality-adjusted life-year decrement associated with each type of false-positive experience.</p><p><strong>Results: </strong>A total of 203 participants completed interviews (49.8% male; mean age = 42.0 years). The mean (SD) utility for the health state describing a true-negative result was 0.958 (0.065). Utilities for false-positive health states ranged from 0.847 (0.145) to 0.932 (0.059). Disutilities for false positives ranged from - 0.031 to - 0.111 (- 0.041 to - 0.111 for lung cancer; - 0.079 for colorectal cancer; - 0.031 to - 0.067 for breast cancer; - 0.048 to - 0.088 for pancreatic cancer).</p><p><strong>Conclusion: </strong>All false-positive results were associated with a disutility. Greater disutility was associated with more invasive follow-up diagnostic procedures, longer duration of uncertainty regarding the eventual diagnosis, and perceived severity of the suspected cancer type. Utility values estimated in this study would be useful for economic modeling examining the value of cancer screening procedures.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"263-276"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884390/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139378109","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 Analysis of Adjuvant Olaparib Versus Watch and Wait in the Treatment of Germline BRCA1/2-Mutated, High-Risk, HER2-Negative Early Breast Cancer in Sweden.","authors":"Maria Polyzoi, Mattias Ekman, Anja Reithmeier, Johanna Jacob, Emma Karlsson, Evelina Bertranou, Barbro Linderholm, Robert Hettle","doi":"10.1007/s41669-023-00457-4","DOIUrl":"10.1007/s41669-023-00457-4","url":null,"abstract":"<p><strong>Introduction: </strong>This study evaluated the cost effectiveness of adjuvant olaparib versus watch and wait (WaW) in patients with germline breast cancer susceptibility gene 1/2 (gBRCA1/2)-mutated, high-risk, human epidermal growth factor receptor 2 (HER2)-negative early breast cancer (eBC), previously treated with neoadjuvant or adjuvant chemotherapy, from a Swedish healthcare perspective.</p><p><strong>Methods: </strong>A five-state (invasive disease-free survival [IDFS], non-metastatic breast cancer [non-mBC], early-onset mBC, late-onset mBC, death) semi-Markov state transition model with a lifetime horizon was developed. Transition probabilities were informed by data from the Phase III OlympiA trial, supplemented with data from additional studies in BRCA-mutated, HER2-negative mBC. Health state utilities were derived via mapping of OlympiA data and supplemented by literature estimates. Treatment, adverse events and other medical costs were extracted from publicly available Swedish sources. Incremental cost per life-year (LY) and quality-adjusted life-year (QALY) gained were estimated. Costs and outcomes were discounted at 3% annually. One-way deterministic and probabilistic sensitivity analyses (PSA) were conducted.</p><p><strong>Results: </strong>Over a lifetime horizon, adjuvant olaparib was associated with an additional 1.50 LYs and 1.22 QALYs, and incremental cost of 471,156 Swedish krona (SEK) versus WaW (discounted). The resulting ICER was 385,183SEK per QALY gained for olaparib versus WaW. ICERs remained below 1,000,000SEK across a range of scenarios, and were consistent across subgroups (hormone receptor [HR]-positive/HER2-negative and triple-negative breast cancer [TNBC]). In PSA, the probability of olaparib being cost effective at 1,000,000SEK per QALY was 99.8%.</p><p><strong>Conclusions: </strong>At list price, adjuvant olaparib is a cost-effective alternative to WaW in patients with gBRCA1/2-mutated, high-risk, HER2-negative eBC in Sweden.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"277-289"},"PeriodicalIF":2.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10884392/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138808411","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}