The Burden of Haemophilia in 5 European Countries: Methods and Results from the Cost of Haemophilia in Europe: A Socioeconomic Survey (CHESS) 2022

Speaker(s)

Mancuso ME1, O'Mahony B2, Brighton S3, Smith C4, García-Diego DA5, Arzenton M6, Sannié T7, Curtis R8, Coffin D9, Brennan F10, Blenkiron T3, Burke T11, Ferri Grazzi E3
1Center for Thrombosis and Hemorrhagic Diseases, Milan, Italy, 2Irish Haemophilia Society, Dublin, Ireland, 3HCD Economics, Knutsford, Cheshire, UK, 4The Haemophilia Society, London, UK, 5FedHemo, Madrid, Spain, 6Italian Federation of Haemophilia Associations (FedEmo), Rome, Italy, 7Association Française des Hémophiles, Paris, France, 8Hematology Utilization Group Study (HUGS), Walnut Creek, CA, USA, 9World Federation of Hemophilia, Montreal, QC, Canada, 10European Haemophilia Consortium, Brussels, Belgium, 11University of Chester, Chester, UK

Presentation Documents

BACKGROUND: The considerable burden of haemophilia in Europe necessitates continued generation of up-to-date information. CHESS aims to assess the socio-economic burden of haemophilia in adult male patients of all severities in 5 European countries: Italy, France, Germany, Spain and the United Kingdom.

OBJECTIVES: The primary objective was to quantify haemophilia-related costs, by disease severity, encompassing direct medical costs (e.g. treatment costs, hospital visits), direct non-medical costs (e.g. health aids/devices, travel costs) and indirect costs (e.g. work productivity impact, caregiver burden). Clinical burden was also assessed.

METHODS: CHESS is a 12-month retrospective, repeated cross-sectional study recruiting patients via their haematologists for the collection of physician-reported demographic, clinical and healthcare resource use data and patient-reported productivity and humanistic burden. Total costs (per patient) were quantified by summing the costs across components, using quantities collected in CHESS 2022 wave and unit costs sourced from the literature. Results 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 754 physician-forms and 166 corresponding patient-forms. Half of the cohort had severe haemophilia, 19% mild and 31% moderate condition. In the mild cohort, mean (SD) ABR was reported as 1.1 (3.2), increasing to 1.5 (2.1) and 2.7 (4.6) for moderate and severe respectively. Joint damage was also observed, with mean (SD) problem joints reported as 0.8 (1.4), 0.8 (1.3) and 1.0 (1.2) for the mild, moderate and severe cohorts. The highest per-patient cost component was factor treatment cost, averaging at approximately €47,000 for mild, €99,000 for moderate and €380,000 for severe condition. Total costs per-patient excluding factor also increased with severity, at approximately €3,000, €9,000 and €12,000 respectively.

CONCLUSIONS: These findings demonstrate the significant clinical and financial burden of haemophilia in Europe.

Code

RWD71

Topic

Clinical Outcomes, Economic Evaluation

Topic Subcategory

Clinician Reported Outcomes

Disease

Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)