ArticleClinicoEconomics and outcomes research : CEOR2026
Impacts and Economic Burden of Pompe Disease on Patients and Families in Thailand: A Mixed Method Study.
Article in ClinicoEconomics and outcomes research : CEOR, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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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.
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