Caring for pediatric patients with short bowel syndrome (SBS) imposes significant mental and physical burden on caregivers. Teduglutide has been shown in clinical trials to reduce the need for parenteral support of pediatric patients with SBS.

This study aimed to provide a comprehensive cost-effectiveness analysis of teduglutide for pediatric patients with SBS in Japan, including considering the caregiver’s burden, an aspect that has been missing from previous teduglutide cost-effectiveness analyses in Japan.

Teduglutide was estimated to cost more than standard of care but prolonged patient quality-adjusted life years (QALYs). The estimated incremental cost-effectiveness ratio (ICER) for teduglutide versus standard of care ranged from 9.5 million JPY/QALY from the public healthcare payer’s perspective to 3.5 million JPY/QALY from a societal perspective (i.e., considering caregiver’s quality of life and productivity loss in addition to the public healthcare payer’s perspective).

In our scenario analyses, which were consistent with the base-case analysis, different ICERs were observed for teduglutide versus standard of care depending on whether caregivers’ quality of life and productivity loss were considered.

This study demonstrated that incorporating the burden of caregivers in the cost-effectiveness analysis provided a more comprehensive assessment of the value of teduglutide for patients, their families, and society. This approach enhances our understanding of the overall value of a treatment, especially for diseases with significant caregiver burden.