Adv Ther. 2024 Dec;41(12):4463-4475. doi: 10.1007/s12325-024-02995-7. Epub 2024 Oct 16.

Hisato Deguchi ¹, Masafumi Kato ²

Author information

¹Market Access, Public Affairs and Patient Experience, Japan Pharma Business Unit, Takeda Pharmaceutical Company Limited, 1-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo, 103-8668, Japan. hisato.deguchi@takeda.com.

²Market Access, Public Affairs and Patient Experience, Japan Pharma Business Unit, Takeda Pharmaceutical Company Limited, 1-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo, 103-8668, Japan.

Abstract

Introduction: Short bowel syndrome (SBS) is associated with a significant mental and physical burden for patients and caregivers. Standard of care (SOC) for SBS includes parenteral support (PS) to optimize intestinal function. Teduglutide, a recombinant human glucagon-like peptide 2 analogue, reduces the need for PS in patients with SBS. In this study, we assessed the cost-effectiveness of teduglutide in pediatric patients with SBS from multiple perspectives, considering the caregiver's burden.

Methods: A Markov model was used to evaluate cost (Japanese yen, JPY) and effectiveness (quality-adjusted life years, QALYs) of teduglutide compared with SOC for pediatric patients with SBS in Japan. We conducted a base-case analysis and selected sensitivity and scenario analyses from three perspectives: (1) the public healthcare payer, (2) the public healthcare and long-term care payer, and (3) society.

Results: In the base-case analysis, the incremental cost-effectiveness ratio (ICER) was 9,533,412 JPY per QALY from the public healthcare payer perspective, 6,335,980 JPY per QALY from the public healthcare and long-term care payer perspective, and 3,510,371 JPY per QALY from the societal perspective. The probability that cost-effectiveness of teduglutide is favorable from a societal perspective was 59.3%. In all scenario analyses, consistent with the base-case analysis, ICERs for teduglutide compared with SOC were different depending on whether caregiver utility and productivity loss were considered.

Conclusions: Incorporating the caregiver's burden in the cost-effectiveness analysis of teduglutide for pediatric patients with SBS 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.