Real-World Data (RWD) Based Cost-Effectiveness Model: Using Atherosclerotic Cardiovascular Disease (ASCVD) As Example

OBJECTIVES: Many statin-treated ASCVD patients still have high LDL-C levels, indicating statin intolerance and uncontrolled hypercholesterolemia. PCSK9 inhibitors reduce cardiovascular events by lowering LDL-C and delaying atherosclerosis. Previously published cost-effectiveness studies of PCSK9-I have yielded mixed results and often used inputs from clinical trial. Innovative cost-effectiveness models based on real-world data are needed to reflect clinical practice outcomes better. The study developed a novel real-world data based cost-effectiveness model to compare PSCK9 inhibitor treatment for ASCVD patients with LDL-C level ≥100 mg/dL.

METHODS: The lifetime Markov model was utilized. The baseline age, gender, diabetes history, LDL-C level, and CV event rate from the National Health Insurance Research Database were used to build a simulated cohort. High-intensity statin efficacy was evaluated using NHIRD patient-level data as the first arm in the mode. The relative LDL-C reduction was via network meta-analysis. Pooled clinical studies provided utility. From NHIRD, event-based and follow-up direct medical costs of CV events were computed. Prices were 2024, and discounts were 3% for cost and effectiveness.

RESULTS: Compared to high-intensity statin treatment for ASCVD patients with LDL-C levels ≥100 mg/dL, high-intensity statin/ezetimibe (hS/Eze) was dominant. The incremental cost-effectiveness ratio (ICER) per QALY gained was €31,918.75 for hS/PCSK9-I and €115,597.76 for hS/Eze/PCSK9-I. Compared to hS/Eze, the ICER per QALY gained was €44,399 for hS/PCSK9-I and €229,880 for hS/Eze/PCSK9-I. Using €28,798 (1 GDP per capita in 2023 in Taiwan) as the willingness-to-pay threshold, the probability of being cost-effective was 38.3% for hS/Eze, 24.9% for hS/PCSK9-I, and 36.8% for hS/Eze/PCSK9-I. All results from the RWD-based model reported higher ICER values than those from the RCT-based model.

CONCLUSIONS: Combining ezetimibe, PSCK9-I, or triple treatment for ASCVD patients with LDL-C ≥100 mg/DL is cost-effective compared to high-intensity. RWD could be used while developing a CE model and demonstrate the value of combination therapy.