Clinical Expert Opinion on the Impact of Vosoritide on Health and Function in Children With Achondroplasia
Author(s)
Ravi Savarirayan, MD1, Carlos Bacino, MD2, Valérie Cormier-Daire, MD3, Christina Due, BSc, MSc, PhD4, Lynda Polgreen, MD5, Carlos Prada, MD6, William Wilcox, MD7, Melita Irving, MD8, Thomas Butt, PhD9.
1Murdoch Children's Research Institute, Melbourne, Australia, 2Baylor College of Medicine, Houston, TX, USA, 3University of Paris Cité, Paris, France, 4BioMarin, LONDON, United Kingdom, 5Harbor-UCLA Medical Center, Los Angeles, CA, USA, 6Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA, 7Emory University, Atlanta, GA, USA, 8Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom, 9BioMarin, London, United Kingdom.
1Murdoch Children's Research Institute, Melbourne, Australia, 2Baylor College of Medicine, Houston, TX, USA, 3University of Paris Cité, Paris, France, 4BioMarin, LONDON, United Kingdom, 5Harbor-UCLA Medical Center, Los Angeles, CA, USA, 6Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA, 7Emory University, Atlanta, GA, USA, 8Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom, 9BioMarin, London, United Kingdom.
OBJECTIVES: Vosoritide is the first and only targeted treatment currently licensed for use in achondroplasia from infancy until growth plate closure. Clinical trials have shown its impact on growth and quality of life in children with achondroplasia across different ages. This study explored the impact of vosoritide on health and functional outcomes based on clinical expert experiences.
METHODS: Clinical experts involved in the vosoritide Phase 2 trials participated in a modified Delphi study, involving a structured explorative survey and anonymous voting of statements, to identify prevalent problems and concerns that vosoritide may impact. Clinician statements were grouped into five categories: functional outcomes, mobility limitations, emotional and social limitations, complications of achondroplasia, and caregiver impact.
RESULTS: In the modified Delphi study, the clinical experts (N=9) reached ≥75% agreement on 19 final statements, with ten achieving 100% agreement. They agreed that vosoritide can positively impact functional outcomes (e.g., accessibility to toilets/sinks, reaching door handles/lights), mobility (e.g., improved stair climbing, running), emotional/social limitations (e.g., self-confidence), leg bowing, and caregiver burden (e.g., worry about complications, future challenges, and societal acceptance). Age at treatment initiation and treatment duration are considered important determinants of treatment impact.
CONCLUSIONS: Clinical expert experiences offer new insights into vosoritide's impact on achondroplasia, including functional, mobility, emotional, social outcomes, and caregiver burden. These insights can help clinicians discuss treatment with patients and inform future studies.
METHODS: Clinical experts involved in the vosoritide Phase 2 trials participated in a modified Delphi study, involving a structured explorative survey and anonymous voting of statements, to identify prevalent problems and concerns that vosoritide may impact. Clinician statements were grouped into five categories: functional outcomes, mobility limitations, emotional and social limitations, complications of achondroplasia, and caregiver impact.
RESULTS: In the modified Delphi study, the clinical experts (N=9) reached ≥75% agreement on 19 final statements, with ten achieving 100% agreement. They agreed that vosoritide can positively impact functional outcomes (e.g., accessibility to toilets/sinks, reaching door handles/lights), mobility (e.g., improved stair climbing, running), emotional/social limitations (e.g., self-confidence), leg bowing, and caregiver burden (e.g., worry about complications, future challenges, and societal acceptance). Age at treatment initiation and treatment duration are considered important determinants of treatment impact.
CONCLUSIONS: Clinical expert experiences offer new insights into vosoritide's impact on achondroplasia, including functional, mobility, emotional, social outcomes, and caregiver burden. These insights can help clinicians discuss treatment with patients and inform future studies.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO47
Topic
Clinical Outcomes, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment, Clinician Reported Outcomes
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Rare & Orphan Diseases