A Budget Impact Model of The Addition of Telotristat Ethyl Treatment In Patients With Uncontrolled Carcinoid Syndrome

Abstract

Carcinoid syndrome (CS) is a rare condition in patients with neuroendocrine tumors (NETs), characterized by flushing and diarrhea that severely impact patients’ quality of life. Though typically treated by somatostatin analogs (SSAs) as standard of care, many patients nevertheless experience uncontrolled CS symptoms despite therapy. Telotristat ethyl (TE) is a novel tryptophan hydroxylase inhibitor granted EMA orphan designation that has been shown in phase 3 studies to effectively reduce bowel movement frequency in CS. We developed a model to evaluate the 5-year budget impact of introducing TE in combination with SSAs (TE+SSAs) in patients with uncontrolled CS in Sweden. Results from the phase 3 TELESTAR trial (NCT01677910) informed TE efficacy in the model, based on the 12-week treatment response, after which health states were captured by a Markov model using 4-week cycles. The model also allowed for TE discontinuation based upon treatment response data from the TELESTAR open-label extension period. Systematic and targeted literature reviews informed CS and carcinoid heart disease (CHD) prevalence, incidence and mortality. These data were used to estimate the number of patients in Sweden eligible for treatment. Real-world CS and CHD-related costs (healthcare resource use, drug acquisition, SSA dosage) were obtained from a Swedish database study. Market share of TE was assumed to increase annually, from 2% to 10% uptake by Year 5. The net 5-year budget impact of TE+SSAs was 11,898€, which translated to a cost of less than 4.88€ per patient per month in each year of the analysis. As a much-needed treatment for a rare and potentially debilitating condition, the 5-year budget impact of the addition of TE to existing standard of care was projected to be 11,898€ according to our model, suggesting that TE may be an affordable treatment option for patients with this rare disease.