A retrospective cohort study of the economic burden of Pompe disease in patients treated with enzyme replacement therapy in the United States
Pompe disease incurs a substantial economic burden in patients despite treatment with ERT. For both types of Pompe disease, the primary drivers of costs were outpatient- and ERT-related; however, patients with IOPD had higher inpatient and supportive care utilization and costs, whereas patients with LOPD had higher costs for outpatient services.
Authors
- Nishitha R. Pillai (ORCID: https://orcid.org/0000-0002-2943-2136)
- Tmirah Haselkorn
- Nigel Rozario (ORCID: https://orcid.org/0000-0003-1510-1675)
- Mark Walzer
- Robert D. Steiner (ORCID: https://orcid.org/0000-0003-4177-4590)
- Bin Xie
- Christopher Young (ORCID: https://orcid.org/0000-0002-1677-7178)
- Benedikt Schoser
- F Solomon
Institutions
- University of Minnesota (US)
- Center for Global Development (US)
- University of Wisconsin–Madison (US)
- Astellas Pharma (United Kingdom) (GB)
- Friedrich Baur Stiftung (DE)
- Astellas Pharma (United States) (US)
- Ludwig-Maximilians-Universität München (DE)
Publication Details
- Journal
- Journal of Medical Economics
- Published
- 2025-09-04
- DOI
- https://doi.org/10.1080/13696998.2025.2556629
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16