The Impact of Burosumab on the Clinical and Economic Burden of X-Linked Hypophosphatemia in Adult Patients in France
Author(s)
Julia HERROU, MD1, Martin BIOSSE DUPLAN, PhD2, Lucile FIGUERES, MD3, Marie-Hélène LAFAGE-PROUST, PhD, MD4, Quentin Berkovitch, MSc5, Loïc BIRON, PharmD5, Henri Leleu, PhD, MD5, Karine BRIOT, PhD, MD1.
1Hôpital Cochin, Paris, France, 2Hôpital Bretonneau, Paris, France, 3CHU de Nantes, Nantes, France, 4CHU de Saint-Etienne, Saint-Etienne, France, 5Public Health Expertise - Cencora, Paris, France.
1Hôpital Cochin, Paris, France, 2Hôpital Bretonneau, Paris, France, 3CHU de Nantes, Nantes, France, 4CHU de Saint-Etienne, Saint-Etienne, France, 5Public Health Expertise - Cencora, Paris, France.
OBJECTIVES: X-linked hypophosphataemia (XLH) is a rare metabolic bone disorder characterized by increased synthesis of fibroblast growth factor 23 (FGF-23), which results in renal phosphate wasting with numerous manifestations affecting significantly quality of life (QoL) and physical capabilities. The management of XLH has evolved in the past years owing to the availability of burosumab a humanized anti-FGF-23 monoclonal antibody. This study aimed to evaluate the socio-economic impacts and cost-effectiveness of treating XLH adult patients with burosumab in France.
METHODS: A cost-utility model was developed to estimate the clinical, including quality-adjusted life years (QALY), and cost burden of XLH over patients’ lifetime. The model incorporated data from clinical trials, published studies, and real-world evidence to compare burosumab with oral phosphate supplements and active vitamin D treatment (OMT), using cost inputs from the French National Health Insurance. Clinical outcomes included renal, dental, and bone complications, and their associated consequences. Costs covered both direct healthcare expenditures and indirect costs, such as work productivity loss.
RESULTS: For the adult XLH population in France (1,900 patients), the annual economic burden would be estimated at €24.3 million, driven by 8 renal, 42 bone, and 446 dental complications. Among burosumab-eligible patients (950 individuals), burosumab could reduce XLH-related complications by 49%, preventing 146 acute complications annually and generating €1.2 million in cost savings from reduced complications. Over a patient’s lifetime, burosumab may be associated with an incremental QALY gain of 1.78 and an incremental cost of €2.87 million, resulting in an incremental cost-effectiveness ratio (ICER) of €1,611,933 per QALY gained when compared to OMT.
CONCLUSIONS: Burosumab could reduce XLH-related complications, improve QoL, and prevent acute events among eligible patients, demonstrating clinical and socio-economic benefits for adult XLH patients in France. It would appear to be a cost-effective option, aligning with the latest published ICER benchmarks for rare diseases in France.
METHODS: A cost-utility model was developed to estimate the clinical, including quality-adjusted life years (QALY), and cost burden of XLH over patients’ lifetime. The model incorporated data from clinical trials, published studies, and real-world evidence to compare burosumab with oral phosphate supplements and active vitamin D treatment (OMT), using cost inputs from the French National Health Insurance. Clinical outcomes included renal, dental, and bone complications, and their associated consequences. Costs covered both direct healthcare expenditures and indirect costs, such as work productivity loss.
RESULTS: For the adult XLH population in France (1,900 patients), the annual economic burden would be estimated at €24.3 million, driven by 8 renal, 42 bone, and 446 dental complications. Among burosumab-eligible patients (950 individuals), burosumab could reduce XLH-related complications by 49%, preventing 146 acute complications annually and generating €1.2 million in cost savings from reduced complications. Over a patient’s lifetime, burosumab may be associated with an incremental QALY gain of 1.78 and an incremental cost of €2.87 million, resulting in an incremental cost-effectiveness ratio (ICER) of €1,611,933 per QALY gained when compared to OMT.
CONCLUSIONS: Burosumab could reduce XLH-related complications, improve QoL, and prevent acute events among eligible patients, demonstrating clinical and socio-economic benefits for adult XLH patients in France. It would appear to be a cost-effective option, aligning with the latest published ICER benchmarks for rare diseases in France.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE712
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Rare & Orphan Diseases