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Economic Burden and Health-Related Quality of Life Among Children With Achondroplasia in China: A Cross-Sectional
Chengaxin Duan1, Yang Liu2, Xuefan Gu3
1People's Livelihood and Health Policy Research Center, School of Public Policy & Management, Tsinghua University, Beijing, China; Beijing Health Economics Association, Beijing, China.
Insights
Achondroplasia significantly impacts families in China, causing high economic costs and reduced quality of life. Addressing nonmedical expenses and complications is crucial for support.
Area of Science:
- Medical Economics
- Public Health
- Genetics
Background:
- Achondroplasia is a common genetic disorder affecting bone growth.
- Understanding its economic and quality-of-life impact is vital for healthcare policy in China.
Purpose of the Study:
- To quantify the economic burden and assess health-related quality of life (HRQoL) in Chinese children with achondroplasia.
Main Methods:
- A cross-sectional survey of 259 children with achondroplasia in China.
- Collected data on demographics, treatment, costs (2020), and HRQoL using validated questionnaires.
Main Results:
- Mean total cost per patient was RMB 57,870, with nonmedical costs exceeding 70% of direct costs.
- Complications and specific treatments increased economic burden.
- HRQoL scores were low, associated with complications, BMI, and lower income.
Conclusions:
- Achondroplasia presents a significant economic and HRQoL burden in China.
- Overlooked direct nonmedical and indirect costs are substantial.
- Managing complications and providing policy support can mitigate the impact.
Objectives:
This study aimed to measure the economic burden and health-related quality of life (HRQoL) of children with achondroplasia in China.
Methods:
A cross-sectional survey was conducted at the 2021 Chinese Achondroplasia Patients Conference using a standardized questionnaire. Data collected included demographic characteristics, treatment history, direct and indirect costs during 2020, and HRQoL outcomes. HRQoL was measured using the Pediatric Quality-of-Life Inventory 4.0 and the EuroQoL 5 Dimensions Youth version.
Results:
A total of 259 patients were included. The mean direct, indirect, and total costs per patient were RMB 39 928 (SD = 27 571), RMB 17 941 (SD = 18 252), and RMB 57 870 (SD = 35 375), respectively. Nonmedical direct costs accounted for more than 70% of direct costs. Use of Calcitriol and presence of complications significantly increased the economic burden. Multivariate analysis identified treatment (β = 19083) and craniocervical junction malformation (β = 23817) as predictors of increased total cost. Regarding HRQoL, mean Pediatric Quality-of-Life Inventory 4.0 scores were below 50 in both self- and proxy reports. EuroQoL-Visual Analog Scale scores averaged 57.25 (SD = 21.34). Regression analysis showed that complications, body mass index differences, and lower income were associated with poorer HRQoL outcomes.
Conclusions:
Achondroplasia imposes a substantial economic and quality-of-life burden on affected families in China. Direct nonmedical and indirect costs, often overlooked, represent significant components of this burden. Effective management of complications and policy support for caregiving and nonmedical expenditures may alleviate this impact.
