Socioeconomic Burden Among Children and Adults With Achondroplasia in Europe and Latin America
Author(s)
Yichen Zhang, MPH, PhD1, Anne Dee, PhD2, Khaled Maman, MSc3, Thomas Butt, PhD4, Shravan Kumar Adepu, BSc5, Joana Raquel Raposo dos Santos, PhD6, Ankit Pahwa, MS7.
1BioMarin Pharmaceutical, Inc., Novato, CA, USA, 2BioMarin Pharmaceutical, San Rafael, CA, USA, 3BioMarin, London, United Kingdom, 4Executive Director, Health Economics and Outcomes Research, BioMarin Pharmaceutical, London, United Kingdom, 5ICON plc, Hyderabad, India, 6ICON plc, Dublin 18, Ireland, 7Icon clinical research, Indore, India.
1BioMarin Pharmaceutical, Inc., Novato, CA, USA, 2BioMarin Pharmaceutical, San Rafael, CA, USA, 3BioMarin, London, United Kingdom, 4Executive Director, Health Economics and Outcomes Research, BioMarin Pharmaceutical, London, United Kingdom, 5ICON plc, Hyderabad, India, 6ICON plc, Dublin 18, Ireland, 7Icon clinical research, Indore, India.
OBJECTIVES: To estimate the socio-economic burden among children and adults with achondroplasia using observational real-world studies.
METHODS: This was a cross-sectional analysis of data pooled from LIAISE (Lifetime Impact of Achondroplasia Study in Europe) and LISA (Lifetime Impact Study in Achondroplasia in South America) studies. Descriptive analyses were used to characterize the socio-economic burden and regression analyses to explore the association of demographic and clinical characteristics on the socioeconomic factors.
RESULTS: 153 adults and 203 children were included. In the overall population, 12.1% required walking assistance (5.6% used a wheelchair and 6.5% used a walking aid). Among adults, 37.9% were married or living as couple, and 38.4% and 69.8% reported the condition had impacted education and employment status respectively. Only half of the adults (56.2%) were employed or self-employed and 66% of unemployed adults felt it was due to their achondroplasia. The need for additional support at the workplace was reported by 54.7% of adults. On average, 18.7 days of work were missed due to achondroplasia in the past 12 months. Adjusting for inflation and purchasing power parity, disability benefits received per month [median (IQR), in US dollars] for adults and children were: 915 (330, 17,392) and 566 (328, 951), and social benefits received per month were 372 (0, 628) vs. 412 (222, 842) respectively. After adjusting for clinical characteristics, a more severe height z-score was associated with higher spending on personal assistance among adults (p<0.05) and, similarly, with higher spending on travel to hospital (p<0.05). Additionally, in Latin America, a more severe height z-score was associated with higher impact of achondroplasia on a person’s employment status (p<0.05).
CONCLUSIONS: Children and adults with achondroplasia in Europe and Latin America experienced impacts on their social status, health and economic situation, some of which were associated with the severity of their condition.
METHODS: This was a cross-sectional analysis of data pooled from LIAISE (Lifetime Impact of Achondroplasia Study in Europe) and LISA (Lifetime Impact Study in Achondroplasia in South America) studies. Descriptive analyses were used to characterize the socio-economic burden and regression analyses to explore the association of demographic and clinical characteristics on the socioeconomic factors.
RESULTS: 153 adults and 203 children were included. In the overall population, 12.1% required walking assistance (5.6% used a wheelchair and 6.5% used a walking aid). Among adults, 37.9% were married or living as couple, and 38.4% and 69.8% reported the condition had impacted education and employment status respectively. Only half of the adults (56.2%) were employed or self-employed and 66% of unemployed adults felt it was due to their achondroplasia. The need for additional support at the workplace was reported by 54.7% of adults. On average, 18.7 days of work were missed due to achondroplasia in the past 12 months. Adjusting for inflation and purchasing power parity, disability benefits received per month [median (IQR), in US dollars] for adults and children were: 915 (330, 17,392) and 566 (328, 951), and social benefits received per month were 372 (0, 628) vs. 412 (222, 842) respectively. After adjusting for clinical characteristics, a more severe height z-score was associated with higher spending on personal assistance among adults (p<0.05) and, similarly, with higher spending on travel to hospital (p<0.05). Additionally, in Latin America, a more severe height z-score was associated with higher impact of achondroplasia on a person’s employment status (p<0.05).
CONCLUSIONS: Children and adults with achondroplasia in Europe and Latin America experienced impacts on their social status, health and economic situation, some of which were associated with the severity of their condition.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE667
Topic
Economic Evaluation, Patient-Centered Research
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Work & Home Productivity - Indirect Costs
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Rare & Orphan Diseases