Real-world disease burden and health care resource utilization for patients with Barth syndrome
Health economic evaluations for rare diseases are scarce. With a noteworthy absence of pharmacoeconomic evidence, utilization of combined clinical case report data from the medical literature, along with registry and claims datasets, demonstrate that BTHS is a costly disease associated with high disease burden and excessive HCRU. Forthcoming treatments (e.g. elamipretide) have the potential to reduce the high disease burden/HCRU.
Authors
- Bruce H. Cohen (ORCID: https://orcid.org/0000-0003-2253-8955)
- Mary Kay Koenig (ORCID: https://orcid.org/0000-0001-5126-8515)
- Yonglin Huang (ORCID: https://orcid.org/0000-0003-4186-7973)
- Lindsay Marjoram (ORCID: https://orcid.org/0000-0002-4422-6963)
- Eric Anderson (ORCID: https://orcid.org/0009-0005-7456-943X)
Institutions
- Barth Syndrome Foundation (US)
- Akron Children's Hospital (US)
- Rome Foundation (US)
- Capital Health Medical Center Hopewell (US)
- John Randolph Foundation (US)
- The University of Texas Health Science Center (US)
- The University of Texas Health Science Center at Houston (US)
Publication Details
- Journal
- Journal of Medical Economics
- Published
- 2025-11-21
- DOI
- https://doi.org/10.1080/13696998.2025.2588729
- Primary Topic
- Mitochondrial Function and Pathology
- Type
- article
- Field-Weighted Citation Impact
- 0.00