PharmacoEconomics Open最新文献

{"title":"Incorporating Resource Constraints in Health Economic Evaluations: Overview and Methodological Considerations.","authors":"Praveen Thokala, Henrique Duarte, Stuart Wright, Don Husereau, Isabelle Durand-Zaleski, Peter Lindgren, Roelien Postema, Gerardo Machnicki, Louis Garrison","doi":"10.1007/s41669-024-00537-z","DOIUrl":"https://doi.org/10.1007/s41669-024-00537-z","url":null,"abstract":"<p><p>It is well known that healthcare resource constraints influence the capacity to deliver care, affecting both the costs and outcomes of medical interventions. If these constraints are not adequately accounted for in economic evaluations, there may be a lack of understanding regarding the full impact of implementing health technologies, leading to decisions being made with suboptimal information. This paper offers an overview of the types of healthcare resource constraints and their potential effects, and introduces a framework grounded in operations research and health economics principles, outlining the methodological considerations for incorporating resource constraints into economic evaluations. Drawing from a literature review and advisory group feedback, three categories of resource constraints were identified: single-use resource constraints, reusable resource constraints and patient throughput constraints. The proposed framework outlines a comprehensive set of steps necessary for effectively incorporating constraints into health economic evaluations and details specific approaches and methodological considerations for each stage to ensure a more accurate and realistic assessment of health interventions. This paper also aims to raise awareness among payers and decision-makers with regards to the limitations of technology evaluations in a resource-constrained health system. Specifically, it suggests that health technology assessment agencies ought to offer guidance on incorporating constraints into the submissions they receive. Moreover, it advocates for a more comprehensive economic evaluation in economic assessments to fully capture an intervention's value.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142838640","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}

{"title":"Health-Economic Modelling of Improved Behavior in Insulin Injection Technique in Belgium.","authors":"Kristof Theys, Sofie Vermander, Lieven Annemans, Christophe De Block, Michel P Hermans, Imke Matthys, Frank Nobels, Trung Nguyen, Vanessa Preumont, Katerina Zakrzewska, Frank Vanderdonck","doi":"10.1007/s41669-024-00547-x","DOIUrl":"https://doi.org/10.1007/s41669-024-00547-x","url":null,"abstract":"<p><strong>Background: </strong>Adequate insulin injection technique (IIT) is crucial to optimize the efficacy of diabetes therapy. Widespread non-practice of injection-site rotation and frequent reuse of insulin pen needles (PN) promote high rates of lipohypertrophy (LH) among people living with diabetes (PwD). LH is associated with increased insulin requirement and suboptimal insulin absorption leading to worsened glycemic control and increased risk for hypoglycemia. Avoiding out-of-the-pocket patient costs of PN could reduce PN reuse, thereby limiting its contribution to LH occurrence.</p><p><strong>Objectives: </strong>A model was developed to compute the impact of a behavior shift in reuse on clinical and economic outcomes for type 1 and insulin-treated type 2 diabetes populations in Belgium.</p><p><strong>Methods: </strong>Patient populations were characterized by treatment-specific characteristics and grouped by their frequency of PN replacement. The intervention was modelled to cause a change in reuse frequency, with the effects propagating downstream of the model. Model and input parameters were based on literature research and expert opinions from a Delphi panel, since available data was found to be limited, incomplete or inconsistent and assumptions were needed.</p><p><strong>Results: </strong>Using the current situation as comparator, this analysis showed a reduction of healthcare expenditures following an improvement in IIT. Considering a 5-year time horizon, this study yields potential savings of 52.6 million euros (28.1-77.9 million euros) when 55% of PwD improve PN reuse behavior.</p><p><strong>Conclusion: </strong>Our model shows that even in an era of technological advances and established diabetes care, lack of adherence to correct IIT has an important impact on economic and health outcomes of PwD in Belgium.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142829627","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}

{"title":"Comparing Different Implementation Strategies for Collaborative Dementia Care Management in Terms of Patients' Characteristics, Unmet Needs, and the Impact on Quality of Life and Costs in Germany.","authors":"Moritz Platen, Wolfgang Hoffmann, Bernhard Michalowsky","doi":"10.1007/s41669-024-00548-w","DOIUrl":"https://doi.org/10.1007/s41669-024-00548-w","url":null,"abstract":"<p><strong>Objective: </strong>To compare the implementation of collaborative dementia care management (cDCM) across two settings regarding patients' characteristics, unmet needs, and the impact on health-related quality of life (HRQoL) and costs.</p><p><strong>Methods: </strong>This analysis was based on data from the DCM:IMPact study, implementing cDCM in a physician network (n = 22 practices) and two day care centers (DCC) for community-dwelling persons with dementia over 6 months in Germany. Participants completed comprehensive assessments at baseline and 6 months after, soliciting sociodemographic and clinical characteristics, unmet needs, HRQoL and healthcare resource utilization. Patient characteristics and unmet needs were analyzed descriptively, and the impact of cDCM on costs and HRQoL was assessed using multivariable regression models.</p><p><strong>Results: </strong>At baseline, patients from the physician network (n = 46) exhibited more pronounced neuropsychiatric symptoms (NPI mean score 14 versus 10, p = 0.075), were more frequently diagnosed with dementia (76% versus 56%, p = 0.040), were less likely assigned a care level by the long-term care insurance (63% versus 91%, p ≤ 0.001) to access formal care services and had more unmet needs (12.8 versus 11.0, p ≤ 0.001), especially for social care services than DCC patients (n = 57). After 6 months, the adjusted means indicated that cDCM implemented in the physician network was more effective [EQ-5D-5L utility score; 0.061; 95% confidence interval (CI) - 0.032 to 0.153] and less costly (- 5950€; 95% CI - 8415€ to - 3485€) than cDCM implemented in DCC.</p><p><strong>Conclusions: </strong>Patients and the healthcare system may benefit more when cDCM is implemented in physician networks. However, patient characteristics indicated two samples with specific conditions and various unmet needs using different ways of accessing healthcare, demonstrating the need for cDCM in both settings, which must be considered when implementing cDCM to integrate the respective sectors efficiently.</p><p><strong>Trial registration: </strong>German Clinical Trials Register: DRKS00025074. Registered 16 April 2021-retrospectively registered.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824282","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}

{"title":"Cost-Effectiveness of Nivolumab Plus Ipilimumab versus Chemotherapy for Previously Untreated Metastatic NSCLC Using Mixture-Cure Survival Analysis Based on CheckMate 227 5-Year Data.","authors":"Alison D Griffiths, Robert O Young, Yong Yuan, Mohammad A Chaudhary, Adam Lee, Jason Gordon, Philip McEwan","doi":"10.1007/s41669-024-00545-z","DOIUrl":"https://doi.org/10.1007/s41669-024-00545-z","url":null,"abstract":"<p><strong>Objectives: </strong>This study assessed the cost-effectiveness of nivolumab plus ipilimumab (NIVO+IPI) versus platinum-doublet chemotherapy (chemo) in untreated metastatic non-small cell lung cancer (NSCLC) using mixture-cure modelling, an approach used to analyse immuno-oncology treatments due to their underlying methods depicting delayed but durable response in some patients.</p><p><strong>Methods: </strong>A mixture-cure economic model was developed from a US third-party payer perspective to assess the lifetime costs and benefits of NIVO+IPI versus chemo using data from Part 1 of the phase III CheckMate 227 trial with 5 years of follow-up. The model consisted of four health states: progression-free without long-term response (non-LTR), progression-free with long-term response (LTR), post-progression, and death. The primary outcomes were the incremental cost per life-year (LY) and quality-adjusted life-year (QALY) gained for NIVO+IPI versus chemo over a patient's lifetime time horizon. Model uncertainty was evaluated using one-way sensitivity analyses and probabilistic sensitivity analysis.</p><p><strong>Results: </strong>NIVO+IPI treatment showed a significant improvement in overall survival versus chemo; mean gain 1.69 LYs and 1.42 QALYs. The majority of the 4.04 LYs accrued by NIVO+IPI were in the LTR state (2.27 years). The incremental cost of NIVO+IPI versus chemo was US$125,321, resulting in an incremental cost/QALY gained of US$88,219.</p><p><strong>Conclusions: </strong>This study suggests NIVO+IPI may be a cost-effective first-line treatment when compared with chemo in a US setting given a threshold of US$150,000 per QALY. The cost-effectiveness analysis used a mixture-cure approach, which may offer a more appropriate modelling method in immuno-oncology given LTR, by more accurately capturing the potential treatment benefit.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142786431","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}

{"title":"Economic Evaluation of Total Knee Replacement Compared with Non-Surgical Management for Knee Osteoarthritis in India.","authors":"Amatullah Sana Qadeer, Ananda Meher, Jennifer Rachel, Winnie Paulson, Abhilash Patra, Naline Gandhi, Nirupama Ay, Lipika Nanda, Sarit Kumar Rout, Ambarish Dutta","doi":"10.1007/s41669-024-00541-3","DOIUrl":"https://doi.org/10.1007/s41669-024-00541-3","url":null,"abstract":"<p><strong>Objective: </strong>This study is an economic evaluation of total knee replacement (TKR) in comparison with non-surgical management in India.</p><p><strong>Methods: </strong>Cost-utility analysis and budget impact analysis (BIA) were conducted on individuals aged ≥ 50 years with osteoarthritis of the knee (OA knee) Kellgren-Lawrence grades 2 and 3 using a provider's perspective. Three scenarios were considered, varying the age at which TKR is administered while assuming a 20-year lifespan for the implant. A Markov model was used to determine incremental cost-effectiveness ratios (ICERs). Sensitivity analysis was conducted incorporating implant costs and other input parameters.</p><p><strong>Results: </strong>Net quality-adjusted life-years (QALYs) gained per OA knee treated with TKR were superior when performed at the age of 50, regardless of OA severity and across all scenarios. The lowest ICER was 36,107 Indian National Rupees (INR) (USD 482.9)/QALY gained, observed at 50 years, while the highest was INR 61,363 (USD 820.72)/QALY gained at 70 years for grade-2 severity. Sensitivity analysis revealed that the ICER was most sensitive to the cost of non-surgical management, health utility values gained in an improved state, and the cost of TKR across scenarios. For the BIA in Scenario 1, with 40% coverage for TKR, costs reach INR 5013 crores (cr) (USD 670,477,060) in 2023 and INR 8444 cr (USD 1,024,628,736) in 2028 (1% of government budgets). In Scenario 2 (full coverage), costs are INR 12,532 cr (USD 1,520,683,008) (2.7%) in 2023, declining to 2.4% in 2028. In Scenario 3, covering 40% under the National Health Mission (NHM), costs vary from 17% in 2023 to 25% in 2028.</p><p><strong>Conclusion: </strong>This study concludes that TKR is a cost-effective treatment option compared with non-surgical management for OA knee in India, irrespective of age, implant types, and severity.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771186","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}

{"title":"Acknowledgement to Referees.","authors":"","doi":"10.1007/s41669-024-00546-y","DOIUrl":"https://doi.org/10.1007/s41669-024-00546-y","url":null,"abstract":"","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142751370","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}

{"title":"The Association between COVID-19 Status and Economic Costs in the Early Stages of the COVID-19 Pandemic: Evidence from a UK Symptom Surveillance Digital Survey.","authors":"Sung Wook Kim, Caterina Alacevich, Catia Nicodemo, Raphael Wittenberg, Simon de Lusignan, Stavros Petrou","doi":"10.1007/s41669-024-00544-0","DOIUrl":"https://doi.org/10.1007/s41669-024-00544-0","url":null,"abstract":"<p><strong>Introduction: </strong>In the absence of a vaccination programme, the coronavirus disease 2019 (COVID-19) pandemic had substantial impacts on population health and wellbeing and health care services. We explored the association between COVID-19 status, sociodemographic, socioeconomic and clinical factors and economic costs during the second wave of the COVID-19 pandemic.</p><p><strong>Data: </strong>The study used patient-reported digital survey and symptom surveillance data collected between July and December 2020, in collaboration with a primary care computerised medical record system supplier, EMIS Health, in the UK. The study included 11,534 participants.</p><p><strong>Methods: </strong>Generalised linear models (GLM) and two-part regression models were used to estimate factors associated with economic costs (£sterling, 2022 prices) estimated from two perspectives: (i) a UK National Health Service (NHS) and personal social services (PSS) perspective and (ii) a societal perspective.</p><p><strong>Results: </strong>Experience of the onset of COVID-19 symptoms started more than 3 months ago was associated with significantly higher NHS and PSS costs (GLM: £319.8, two-part: £171.7) (p < 0.001) and societal costs (GLM: £776.9, two-part: £675.6) (p < 0.001) in both models. A positive test result within the previous 14 days was associated with significantly higher NHS and PSS costs (two-part: £389.1) (p < 0.05) and societal costs (GLM: £470.7, two-part: £439.2) (p < 0.01). Age between 31 and 55 years was associated with significantly higher societal costs than age between 16 and 30 years.</p><p><strong>Conclusion: </strong>This study identifies and quantifies factors associated with the economic costs incurred during the second wave of the COVID-19 pandemic in the UK. The results of our study can inform cross-country comparisons and other cost comparisons.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142751371","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}

{"title":"Burden of Disease of Breast Cancer in Italy: A Real-World Data Analysis.","authors":"F S Mennini, A Marcellusi, P Sciattella, M Scortichini, Angela Ragonese, F Cattel, R D'Antona, L Del Mastro, S Gori, G Perrone, R Migliorini, M Trabucco Aurilio","doi":"10.1007/s41669-024-00543-1","DOIUrl":"https://doi.org/10.1007/s41669-024-00543-1","url":null,"abstract":"<p><strong>Background: </strong>Breast cancer (BC) constitutes a significant public health challenge in Italy, with a considerable impact on healthcare resources and societal costs. Despite advancements in diagnostics and therapies, the economic burden of BC remains substantial, necessitating a comprehensive evaluation to inform healthcare policy and resource allocation. The aim of this study is to estimate both direct health costs and social security costs related to BC.</p><p><strong>Methods: </strong>This study utilized real-world data from the Italian Health Information System (HIS) and Local Health Unit (LHU) Umbria 2 HIS databases spanning 2010-2019. Direct health costs, encompassing hospitalizations, outpatient visits, and drug prescriptions, were assessed alongside social security costs associated with BC-related work inability. Statistical analyses and probabilistic models were employed to estimate costs and evaluate trends over time.</p><p><strong>Results: </strong>The analysis revealed an annual cost of €273 million for hospital admissions related to BC in Italy, with metastatic BC (MBC) incurring the highest average annual cost per patient (€5018). When outpatient visits and drug consumption were incorporated, the mean annual cost per patient in LHU Umbria 2 rose to €11,380 for MBC. Social security costs, predominantly comprising disability benefits (DBs), totaled €579 million annually. Overall, the study estimated the total annual economic impact of BC in Italy to exceed €1 billion, with social costs representing 50.4% of the total burden.</p><p><strong>Conclusions: </strong>This comprehensive assessment underscores the substantial economic strain imposed by BC in Italy and highlights the need for early detection and intervention strategies to mitigate costs and enhance patient outcomes. These findings offer valuable insights into the economic landscape of BC, guiding policy decisions and resource allocation efforts aimed at optimizing BC management and alleviating its societal burden.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-11-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142693334","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}

{"title":"Health Economic Evaluation of Antimicrobial Stewardship, Procalcitonin Testing, and Rapid Blood Culture Identification in Sepsis Care: A 90-Day Model-Based, Cost-Utility Analysis.","authors":"Wendy I Sligl, Charles Yan, Jeff Round, Xiaoming Wang, Justin Z Chen, Cheyanne Boehm, Karen Fong, Katelynn Crick, Míriam Garrido Clua, Cassidy Codan, Tanis C Dingle, Connie Prosser, Guanmin Chen, Alena Tse-Chang, Daniel Garros, David Zygun, Dawn Opgenorth, John M Conly, Christopher J Doig, Vincent I Lau, Sean M Bagshaw","doi":"10.1007/s41669-024-00538-y","DOIUrl":"10.1007/s41669-024-00538-y","url":null,"abstract":"<p><strong>Objective: </strong>We evaluated the cost-effectiveness of a bundled intervention including an antimicrobial stewardship program (ASP), procalcitonin (PCT) testing, and rapid blood culture identification (BCID), compared with pre-implementation standard care in critically ill adult patients with sepsis.</p><p><strong>Methods: </strong>We conducted a decision tree model-based cost-effectiveness analysis alongside a previously published pre- and post-implementation quality improvement study. We adopted a public Canadian healthcare payer's perspective. Two intensive care units in Alberta with 727 adult critically ill patients were included. Our bundled intervention was compared with pre-implementation standard care. We collected healthcare resource use and estimated unit costs in 2022 Canadian dollars (CAD) over a time horizon from study entry to hospital discharge or death. We calculated the incremental net monetary benefit (iNMB) of the intervention group compared with the pre-intervention group. The primary outcome was cost per sepsis case. Secondary outcomes included readmission rates, Clostridioides difficile infections, mortality, and lengths of stay. Uncertainty was investigated using cost-effectiveness acceptability curves, cost-effectiveness plane scatterplots, and sensitivity analyses.</p><p><strong>Results: </strong>Mean (standard deviation [SD]) cost per index hospital admission was CAD $83,251 ($107,926) for patients in the intervention group and CAD $87,044 ($104,406) for the pre-intervention group, though the difference ($3,793 [$7,897]) was not statistically significant. Costs were higher in the pre-intervention group for antibiotics, readmissions, and C. difficile infections. The intervention group had a lower mean expected cost; $110,580 ($108,917) compared with pre-intervention ($125,745 [$113,210]), with a difference of $15,165 ($8278). There were no statistically significant differences in quality adjusted life years (QALYs) between groups. The iNMB of the intervention group compared with pre-intervention was greater than $15,000 for willingness-to-pay (WTP) per QALY values of between $0 and $100,000. In our sensitivity analysis, the intervention was most likely to be cost-effective in roughly 56% of simulations at all WTP thresholds.</p><p><strong>Conclusions: </strong>Our bundled intervention of ASP, PCT, and BCID among adult critically ill patients with sepsis was potentially cost-effective, but with substantial decision uncertainty.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142668597","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}

{"title":"Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.","authors":"Amr A El-Sayed, Nancy S Bolous","doi":"10.1007/s41669-024-00540-4","DOIUrl":"https://doi.org/10.1007/s41669-024-00540-4","url":null,"abstract":"<p><strong>Introduction: </strong>Haemophilia is a rare genetic bleeding disorder that leads to musculoskeletal complications. The high cost of haemophilia treatment necessitates a thorough evaluation of its economic burden. However, due to the difficulty of estimating direct non-medical, indirect, and intangible costs, studies often underestimate the actual economic burden of haemophilia. This scoping review aims to summarise economic studies in haemophilia conducted from a societal perspective.</p><p><strong>Methods: </strong>A systematic search across eight scholarly databases, grey literature, and reference lists until the 5^th of July 2023 was conducted to identify relevant studies. The inclusion criteria encompassed full-text, English-language publications of economic analyses in congenital haemophilia from a societal perspective. Model-based studies and those adopting a payer perspective were excluded. Costs were adjusted to international dollars (I$) and US dollars (US$) in 2022 for comparability.</p><p><strong>Results: </strong>Out of 2993 potential sources identified, 25 studies met the inclusion criteria, covering 7226 persons with haemophilia across 22 countries. All studies reported direct medical costs, with four excluding the cost of haemostatic therapy. Fifteen studies reported direct formal non-medical costs, while eight reported direct informal non-medical costs. All but one study reported the indirect costs. The average annual costs of haemophilia varied widely based on treatment modality, disease severity, geographical location, and included cost categories. When including the cost of clotting factor replacement therapy (CFRT), the total cost for severe haemophilia without inhibitors ranged from 1566 I$ to 700,070 I$ per person per year (lowest value reported in India and highest in the United States). CFRT represented up to 99.9% of the total cost for those receiving prophylaxis and up to 95.1% for episodic treatment. Haemostatic therapies accounted for 82% of the total cost in patients with inhibitors.</p><p><strong>Conclusion: </strong>There is a significant heterogeneity in defining cost categories required for a comprehensive economic analysis from a societal perspective. While haemostatic therapies constitute a substantial portion of the overall cost, direct non-medical and indirect costs are crucial as they are often paid out-of-pocket and may impede access to treatment. It is essential for haematologists and economists to establish a standardised costing framework for future studies, particularly in the era of novel therapies.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638728","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}