Impacts and Economic Burden of Pompe Disease on Patients and Families in Thailand: A Mixed Method Study
1
Issued Date
2026-01-01
Resource Type
eISSN
11786981
Scopus ID
2-s2.0-105027097210
Journal Title
Clinicoeconomics and Outcomes Research
Volume
18
Start Page
1
End Page
11
Rights Holder(s)
SCOPUS
Bibliographic Citation
Clinicoeconomics and Outcomes Research Vol.18 (2026) , 1-11
Suggested Citation
Youngkong S., Thavorncharoensap M., Chaikledkaew U., Luangsinsiri C., Tim-Aroon T., Kuptanon C., Sathienkijkanchai A., Rojnueangnit K., Wichajarn K., Boonyawat B., Suphapeetiporn K., Wattanasirichaigoon D. Impacts and Economic Burden of Pompe Disease on Patients and Families in Thailand: A Mixed Method Study. Clinicoeconomics and Outcomes Research Vol.18 (2026) , 1-11. 11. doi:10.2147/CEOR.S542203 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/114744
Title
Impacts and Economic Burden of Pompe Disease on Patients and Families in Thailand: A Mixed Method Study
Author's Affiliation
Mahidol University
Siriraj Hospital
Faculty of Medicine, Khon Kaen University
Srinakharinwirot University
Faculty of Medicine Ramathibodi Hospital, Mahidol University
Faculty of Medicine, Chulalongkorn University
Rangsit University
Faculty of Medicine, Thammasat University
Phramongkutklao College of Medicine
Queen Sirikit National Institute of Child Health
Siriraj Hospital
Faculty of Medicine, Khon Kaen University
Srinakharinwirot University
Faculty of Medicine Ramathibodi Hospital, Mahidol University
Faculty of Medicine, Chulalongkorn University
Rangsit University
Faculty of Medicine, Thammasat University
Phramongkutklao College of Medicine
Queen Sirikit National Institute of Child Health
Corresponding Author(s)
Other Contributor(s)
Abstract
Purpose: This study aimed to explore the impacts and economic burden experienced by patients and families affected by Pompe disease in Thailand. Patients and Methods: A mixed-methods analysis was employed. To estimate the direct medical cost, databases and/or medical records of the seven rare disease (RD) centers in Thailand from 2010 to 2021 were reviewed. Structured interviews were also conducted to examine direct non-medical and indirect costs. The economic burden was presented in 2024 US dollars (USD) value (1 USD = 35.29 THB). In-depth interviews were conducted with four patients and two caregivers to explore the impacts of this disease. Results: Frequently reported symptoms included fatigue, muscle weakness, and difficulty breathing during sleep. These symptoms had significant and profound impacts on patients’ functioning, family and social roles, self-esteem, emotion, and financial status. Frequent visits to the RD centers, along with the referral process, were reported as a substantial burden to the patients and families. The mean annual direct medical cost per patient, excluding the cost of enzyme replacement therapy, ranged between 2,505 and 14,042 USD from the provider’s perspective and 1,484 USD from the patient’s perspective. Direct non-medical costs were highly significant, with the annual cost of informal care of 1,878–1,992 USD per patient. Around 43% of the patients reported requiring informal care. On average, each patient required 2.6 ± 3.5 hours of informal care per day. Conclusion: Our findings revealed substantial impacts of Pompe disease on individuals’ physical, social, emotional, and functional capacities as a result of its symptoms. Coordinated care, where patients can be treated by clinicians at local hospitals who operate in close liaison with specialists from the RD centers, is warranted. Psychosocial, welfare, and transportation supports are clearly justified to alleviate the burden and improve the quality of life of the patients.