BACKGROUND: Adults with intestinal failure often undergo rehabilitation with the goal of enteral autonomy. However, at 5 years, intestinal failure has a 25% mortality with nearly half of the survivors remaining on parenteral nutrition (PN). Teduglutide is a GLP-2 analogue that promotes intestinal adaptation and can decrease PN dependence, resulting in enteral autonomy in some and decreased PN in many more. Unfortunately, teduglutide, an orphan drug, is expensive with an estimated cost of over $400,000 per year. The current study evaluated the cost-effectiveness of using teduglutide in adult patients with intestinal failure.
METHODS: A Markov model was used to evaluate the costs (in US dollars) and effectiveness (in quality-adjusted life years or QALYs) of treatment with a presumed starting age of 40 years. Parameters were obtained from published data or estimation. The primary effect modeled was the increased likelihood of reduced PN days/week when using teduglutide, leading to greater quality of life and lower daily PN costs. Future costs and utilities were discounted at 3% per year. Sensitivity analyses were performed on all model parameters.
RESULTS: In the base scenario, teduglutide cost $949,910/QALY gained. On one-way sensitivity analysis, only reducing teduglutide cost decreased the cost/QALY gained to below the typical threshold of $100,000/QALY gained. Specifically, teduglutide cost would need to be reduced by over 65% for it to reach the threshold value (Figure 1). Probabilistic sensitivity analysis favored no teduglutide in 80% of iterations at a $100,000/QALY threshold (Figure 2). However, 13% of iterations actually found teduglutide therapy to be cost-saving.
Figure 1. One-way sensitivity analysis of the effect of cost of teduglutide on incremental cost-effectiveness ratio.
Figure 2. Cost-effectiveness acceptability curve from probabilistic sensitivity analysis.
CONCLUSIONS: In summary, teduglutide does not meet a traditional cost-effectiveness threshold as treatment for PN reduction in adult patients with intestinal failure compared to standard intestinal rehabilitation. Subpopulations that demonstrate maximum benefit could be cost-saving. Addressing cost alone would require a significant reduction in teduglutide price to improve cost-utility.
1. Messing, B., et al., Long-term survival and parenteral nutrition dependence in adult patients with the short bowel syndrome. Gastroenterology, 1999. 117(5): p. 1043–50.