Clin Nutr. 2023 Dec;42(12):2363-2371. doi: 10.1016/j.clnu.2023.10.001. Epub 2023 Oct 5.

Daniela Gattini ¹, Christina Belza ², Raphael Kraus ³, Yaron Avitzur ⁴, Wendy J Ungar ⁵, Paul W Wales ⁶

Author information

¹Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; Division of Gastroenterology, Hepatology & Nutrition, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada; Group for Improvement of Intestinal Function and Treatment (GIFT), Transplant and Regenerative Medicine Centre, Toronto, Ontario, Canada.

²Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; Group for Improvement of Intestinal Function and Treatment (GIFT), Transplant and Regenerative Medicine Centre, Toronto, Ontario, Canada.

³Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; Pediatric Rheumatologist, Centre Hospitalier Sainte-Justine (CHUSJ), Montreal, Quebec, Canada.

⁴Division of Gastroenterology, Hepatology & Nutrition, Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada; Group for Improvement of Intestinal Function and Treatment (GIFT), Transplant and Regenerative Medicine Centre, Toronto, Ontario, Canada.

⁵Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; Child Health Evaluative Sciences Program, Hospital for Sick Children Research Institute, Toronto, Ontario, Canada.

⁶Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada; Division of General and Thoracic Surgery, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, United States; Cincinnati Center of Excellence for Intestinal Rehabilitation (CinCEIR), Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States. Electronic address: paul.wales@cchmc.org.

Abstract

Background & aims: A growing proportion of children with short bowel syndrome (SBS) remain dependent on long-term parenteral nutrition (PN). Teduglutide offers the potential for more children to decrease PN support and achieve enteral autonomy (EA), but at a significant expense. This study aims to assess the incremental costs of teduglutide plus standard of care compared to standard of care alone in weaning PN support per quality-adjusted life year (QALY) gained in children with SBS.

Methods: This is a cost-utility analysis comparing teduglutide with standard of care alone in children with SBS. A microsimulation model of children with SBS on PN aged 1-17 years was constructed over a time horizon of six years, with a cycle length of one month. The study adopted the healthcare system and societal payer perspectives in Ontario, Canada. The health outcome measure was QALYs, with results expressed in terms of incremental costs and QALYs. Scenario analyses were performed to examine the effects of different time horizons, timing of teduglutide initiation, and modeling cost of teduglutide based on pediatric weight-dosing.

Results: Incremental healthcare system costs for teduglutide compared to standard of care were CAD$441,314 (95% CI, 414,006 to 441,314) and incremental QALYs were 1.80 (95% CI, 1.70 to 1.89) resulting in an incremental cost-effectiveness ratio (ICER) of CAD$285,334 (95% CI, 178,209 to 392,459) per QALY gained. Incremental societal costs were CAD$418,504 (95% CI, 409,487 to 427,522) and incremental societal QALYs were 1.91 (95% CI, 1.85 to 1.98) resulting in an ICER of CAD$261,880 (95% CI, 136,887 to 386,874) per QALY gained. Scenario analysis showed that teduglutide was cost-effective when it was started two years after intestinal resection (ICER CAD$48,741, 95% CI, 17,317 to 80,165) and when its monthly cost was adjusted using weight-based dosing, avoiding wastage of the remaining 5 mg dose vial (Teduglutide dominated over SOC as the less costly and most effective strategy).

Conclusions: Although teduglutide was not cost-effective in weaning PN support in children with SBS, starting teduglutide once natural intestinal adaptation is reduced and adjusting its monthly cost to reflect cost by volume as dictated by weight-based dosing rendered the intervention cost-effective relative to standard of care. These results indicate the potential for clinicians to re-assess optimal time for initiation of teduglutide after intestinal resection, drug manufacturers to consider the use of multi-dose or paediatric-dose vials, and the opportunity for decision-makers to re-evaluate teduglutide funding.