Incorporating Caregiver Burden in Japans Cost-Effectiveness Evaluation: A Case Study of Teduglutide for Pediatric Short Bowel Syndrome

OBJECTIVES: In Japan, cost-effectiveness analysis has assumed an increasingly significant role in drug price adjustments and, in a limited case, price setting. However, standard analyses often focus solely on patient outcomes, overlooking caregiver burden in diseases requiring long-term care, potentially underestimating treatment value. Pediatric short bowel syndrome (SBS) is a representative example, as prolonged dependence on parenteral support (PS) significantly affects caregivers' quality of life (QOL) and productivity. This study aimed to integrate caregiver burden into cost-effectiveness evaluations within Japan’s healthcare system, using teduglutide, a recombinant human glucagon-like peptide 2 analogue that reduces PS dependence, as a case study.
METHODS: A Markov model was used to assess the costs and effectiveness of teduglutide compared to standard of care for pediatric SBS patients in Japan, with a particular focus on caregiver utility relative to the usual public healthcare payer perspective. Caregiver’s QALY was incorporated using EQ-5D-derived parameters based on patient PS states and accumulated over the analysis period aligned with patient QALYs. Additionally, caregiver productivity loss was accounted for by assigning wage-based costs to patient PS states.
RESULTS: Incorporating caregiver utility added 5.12 QALYs to the patient’s incremental QALYs, reducing the ICER from the public healthcare and long-term care payer perspective to 5,628,147 JPY per QALY. In addition, accounting for caregiver productivity losses reduced costs 43.16 million JPY, lowering the ICER from a societal perspective to 2,802,537 JPY per QALY. These findings demonstrate that the quantitative inclusion of caregiver burden significantly influences ICER values and suggests the application of this approach in diseases with substantial caregiver involvement to provide a more comprehensive treatment value assessment.
CONCLUSIONS: This study illustrates the practical steps for incorporating caregiver burden into Japan’s cost-effectiveness analyses, using pediatric SBS as an example. This expanded analysis captures a broader value, potentially guiding more informed resource allocation in Japan’s healthcare context.