OBJECTIVES: Teduglutide (orphan drug designation), an analogue of glucagon-like peptide 2, has been licensed for the treatment of short bowel syndrome (SBS) in patients following intestinal adaptation after surgery. SBS is an ultra-rare, long-term and potentially life-threatening condition requiring parenteral nutrition (PN). Patients on PN suffer severe complications like intestinal failure liver disease, catheter-related sepsis, liver and intestinal transplant or death. Several studies demonstrated that teduglutide reduces volume and days on PN which led to a reduction in severe complications in children and adults.

METHODS: We developed independent Markov models for adults and children comparing teduglutide versus SoC. Transition states were days in PN (7 to 0), catheter-related sepsis, intestinal transplant, and death. The model was informed with data from the STEP trials and observational studies published up to 2022. Utilities were obtained from published data and costs from local sources (in US dollars). Effectiveness was expressed in QALYs. Time horizon was life-long for adults and 5y for children. The willingness-to-pay threshold considered was 1 GDP per QALY (8,810 USD).

RESULTS: In adults, our model predicted that teduglutide reduced mortality (-46.9%), sepsis (-81.5%), intestinal transplant (-84.5%) and increased PN weaning (+251%). Incremental effectiveness was 4.39 QALY at 574,773 USD cost. ICER was 130,891 USD/QALY (14.9 times the threshold). In children, teduglutide reduced mortality (-67.7%), sepsis (-61.4%), intestinal transplant (-78.8%) and increased PN weaning (+637.5%). Incremental effectiveness was 0.67 QALY at 418,597 USD cost. The ICER was 624,792 USD/QALY (70.9 times the threshold).

CONCLUSIONS: In our model, teduglutide is an effective intervention with significant benefits on relevant clinical endpoints. At the current price, teduglutide is beyond the cost-effectiveness threshold considered in Argentina. The implementation of value-based contracting methodologies and multidisciplinary approaches may enhance patient access in Argentina considering the high-cost burden to treat this debilitating disease with this orphan medication.