Economic evaluation reveals high health care resource utilization and disease burden in Barth syndrome, suggesting new treatments like elamipretide could help.
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.
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Marjoram et al. (2025) studied this question.
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