Presentation Details
| The Burden of Hemophilia in the US: Methods and Results from the Cost of Hemophilia: a Socioeconomic Survey (CHESS) US (2022) Randall Curtis1, Maria Elisa Mancuso2, 3, Enrico Ferri Grazzi4, Donna Coffin5, Brian O'Mahony6, 7, Clive Smith8, Daniél Anibal García Diego9, Matteo Arzenton10, Thomas Sannié11, Fiona Brennan12, Tom Burke4, 13, Sarah Brighton4. 1Hematology Utilization Group Study (HUGS), Walnut Creek, CA, USA.2Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital, Rozzano, Italy.3Humanitas University, Pieve Emanuele, Milan, Italy.4HCD Economics, Knutsford, United Kingdom.5World Federation of Hemophilia, Montreal, QC, Canada.6Irish Haemophilia Society Ltd, Dublin, Ireland.7Trinity College, Dublin, Ireland.8The Haemophilia Society, London, United Kingdom.9Federación Española de Hemofilia (FedHemo), Madrid, Spain.10Italian Federation of Haemophilia Associations (FedEmo), Rome, Italy.11Association Française des Hémophiles (AFH), Paris, France.12Faculty of Health and Social Care, University of Chester, Chester, United Kingdom |
Abstract
Background: Hemophilia is known to result in considerable clinical and socioeconomic burden in the United States (US). As such, it is important to further the generation of up-to-date information, consistent with current standard of care for the condition. CHESS III US aimed to assess the socio-economic burden of hemophilia in adult male patients of all severities in the US. Objectives The primary objective of the study was to quantify hemophilia-related costs, by disease severity, encompassing direct medical costs (i.e. treatment costs, hospital visits, tests and procedures performed) direct non-medical costs (i.e. health aids/devices, alternative and complementary therapy, travel costs) and indirect costs (i.e. work productivity impact, caregiver burden). Clinical burden by disease severity was also assessed. Methods: CHESS III US employed a retrospective, cross-sectional methodology, recruiting a sample of hematologists to complete an electronic case report form (CRF) covering demographic, clinical and healthcare resource use data for the 12 months prior to the date of extraction. Data collection took place from Apr-Nov 2022. The study was conducted with ethical approval from the University of Chester (UK) and with guidance from an Expert Reference Group. Total costs were quantified by summing the costs across all aspects of direct medical and non-medical, as well as indirect, costs using quantities collected in CHESS III US and unit costs sourced from the literature to enable estimation. Total cost (per-patient) was extrapolated using prevalence estimates from the 2021 WFH Annual Global Survey to estimate US population cost. Reported results focus on the clinical burden of hemophilia and related direct medical costs and are presented by clinical severity (based on baseline factor level): mild (>5-40%), moderate (1-5%), and severe (<1%). Results: The final sample consisted of 467 CRFs. Patients with severe hemophilia comprised 29% of the cohort, while mild and moderate condition represented 25% and 46% respectively. In the mild cohort, mean (SD) ABR was reported as 0.4 (0.9), increasing to 1.3 (2.0) and 1.6 (3.4) for moderate and severe respectively (Table 1). Joint damage was also observed, with mean (SD) problem joints reported as 0.5 (0.6), 1.1 (1.3) and 1.1 (1.8) for the mild, moderate and severe cohorts respectively. A similar pattern was observed in target joints (Table 1). Overall, levels of chronic pain increased with condition severity, with markedly higher levels in patients with moderate or severe hemophilia. Moderate or severe chronic pain was reported in 4% of mild, 18% of moderate, and 23% of severe hemophilia cohorts respectively (Table 1). The highest per-patient cost component was factor treatment cost, averaging at approximately $78,000 for mild, $226,500 for moderate and $636,500 for severe condition, respectively (estimated $462mn, $892mn and $5,457mn for the US hemophilia population). Excluding factor, direct medical costs for the US population were estimated to be highest for severe hemophilia ($41mn) decreasing for moderate ($14mn) and mild ($12mn) condition (Table 2). Conclusions: These findings demonstrate the significant clinical and financial burden of hemophilia to patients and the healthcare system in the US, with greater burden in more severe forms of the condition.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.