Project 458280

Intestinal failure (IF) is a devastating condition resulting in long-term parenteral nutrition support (nutrition through an intravenous line). Pediatric IF leads to significant costs to the healthcare system and patients. With increased survival of children with IF, the economic and health-related quality of life (HRQOL) impacts of IF on patients and their caregivers have been increasingly scrutinized. Furthermore, new expensive treatments that promote intestinal adaptation such as teduglutide require economic evaluation. Economic evaluations in pediatric IF are limited by a lack of pediatric HRQOL instruments. Instruments that measure HRQOL in children can be applied to both patients and their caregivers. However, agreement between these methods is not well established. A parent-child dyad approach to pediatric HRQOL has been described, which could be a valuable alternative to assess children's HRQOL. The first objective of this study is to measure the cost-effectiveness of teduglutide in children with IF. We will perform an economic evaluation of teduglutide using a microsimulation model (model that simulates the course of a disease accounting for individual differences). The second objective is to assess the validity of alternative approaches to measuring HRQOL in children with IF. The third objective is to measure HRQOL in caregivers of children with IF and determine its correlation with the patient's HRQOL. HRQOL questionnaires will be administered during outpatient clinic visits by interview to children with IF 8 years of age or older and parents separately and then together as a dyad to assess the child's health state. The results of the health economic evaluation of teduglutide have the potential to change clinical management and inform teduglutide funding decision-making in Canada. This study will also determine the best methods for measuring HRQOL in children and spillover effect on caregivers, an area that has been neglected in economic evaluations.