OBJECTIVES: The short bowel syndrome with intestinal failure (SBS-IF) describes a rare but devastating medical condition where patients are dependent to parenteral support (PS) due to an absolute loss of bowel length resulting in a variety of medical sequalae and subsequent increased morbidity and mortality. Interdisciplinary treatment may include therapy with the effective, but expensive intestinotrophic peptide teduglutide. This study aimed for an evaluation of the cost-effectiveness of using teduglutide in adults with SBS-IF from a societal perspective in order to evaluate the socioeconomic impact.

METHODS: A time-discrete Markov-model was developed to simulate the treatment effect (lifetime-costs, quality-adjusted life-years/QALYs and life-years/LYs) of teduglutide plus best supportive care (BSC) compared with BSC alone in SBS-IF patients who are dependent on PS. The health states of the model were structured around the number of days on PS. During each model cycle, patients were either weaned by 1 or 2 days per week or remain in the same PS states. Clinical data from three data sets were used: (1) an Austrian observational study (base case) (2) pooled observational studies and (3) the randomized STEPS study. Direct costs (2022 €) were derived from published sources. A willingness-to-pay threshold set at 300,000 €/QALY gained was incorporated. One-way and probabilistic sensitivity analysis were performed.

RESULTS: Under the base case assumption teduglutide is associated with costs of 2,238,808.66 € per patient and 10.501 QALYs (13.385 LYs) over a life-time-horizon. No teduglutide accounted for 1,182,698.95 € and 2.133 QALYs (8.181 LYs). the ICUR amounts to 126,610.04 €. In case the pooled clinical data set the ICUR improves to 192,750.68 €. If clinical data based on the STEPS studies were used the ICUR increased to 245,958.52 €.

CONCLUSIONS: In adult patients with SBS-IF, the use of teduglutide plus BSC is cost-effective and improve the quality-of-life significantly.