ASSESSING COST-EFFECTIVENESS AND KEY VALUE DRIVERS OF ADJUVANT TRIGLYCERIDE LOWERING THERAPY IN MANAGEMENT OF PATIENTS WITH A RARE GENETIC SYNDROME

OBJECTIVES: This research focused on a rare genetic disease in which the body does not break down fat (lipids) correctly. The current standard of care (SoC) is diet, however, patients receive off-label adjuvant triglyceride (TG) lowering treatments. There is limited literature on the long-term clinical and cost-effectiveness of adding TG lowering therapy in the management of these patients. The objective of this work was to develop a modelling framework that would adequately capture the range of benefits and costs associated with adjuvant TG lowering therapy in the management of patients with this disease.

METHODS: A decision analytic, Markov model was developed to evaluate the cost-utility of current SoC (diet) compared to TG lowering therapies (including fibrates and statins) for patient management. The model included five health states: high risk TG level, low risk TG level, acute metabolic morbidity (event – tunnel state), post-high risk TG level, and post-low risk TG level. A cycle length of 3 months, and the perspective of the UK healthcare system were used. Clinical outcomes, costs, and utilities were obtained from publicly available sources and through discussions with clinical experts.

RESULTS: The model structure adequately captured the treatment pathway for patients with the disease and the unique range of benefits, including potential quality of life improvements, as well as the differences in costs that accrue with different treatment options. The major value drivers for the TG lowering therapy included quality of life, cost of therapy, and relative treatment efficacy.

CONCLUSIONS: Based on these findings, it is feasible to model the lifelong benefits that accumulate with adjuvant TG lowering therapy and compare overall benefits and costs to current SoC.