COST-EFFECTIVENESS OF OMALIZUMAB IN SEVERE UNCONTROLLED ALLERGIC ASTHMA USING RCT AND REAL-WORLD EVIDENCE IN THE DUTCH SETTING

OBJECTIVES: The objective of this analysis was to compare results of two cost-effectiveness analyses for omalizumab added to standard therapy in severe allergic asthma patients using an RCT (INNOVATE) compared to a real-world, prospective observational study (EXPERIENCE). METHODS: A Markov model was developed to examine the cost-effectiveness of add-on omalizumab versus standard care from the perspective of the Dutch health care system over a patient’s lifetime.  Efficacy data for clinically significant (CS) exacerbations and resource use (hospital admissions, unscheduled physician visits and emergency visits) were derived from INNOVATE or Dutch patients enrolled in EXPERIENCE. Data from each were projected to lifetime with discounted future costs (4%) and outcomes (1.5%). RESULTS: For the EXPERIENCE study, the modelled direct medical costs for patients on standard therapy were €77,615, of which 75% was for exacerbation control versus €133,475 for standard therapy + omalizumab, of which 38% was for exacerbation control.  Patients on omalizumab had more QALYs than those on standard therapy alone, 12.05 versus 10.47.  The resulting ICER was €35,257/QALY for the EXPERIENCE study.  The INNOVATE costs were lower in both treatment arms: €22,499 for standard therapy and €58,666 for standard therapy + omalizumab.  Costs were lower due to lower rate of CS exacerbations in the RCT where patients had been under best possible control at trial entry.  QALYs were similar to the EXPERIENCE study 12.05 and 10.91, respectively; resulting in €31,802/QALY. CONCLUSIONS:  Decision-makers are often presented with cost-effectiveness evidence from RCTs although they prefer to base decisions on real-world data are preferred. This study is one the first to include both in a re-evaluation dossier. It showed differences in patient characteristics (exacerbation rates and resource use) between the RCT and observational study. However it confirmed the value of omalizumab with similar ICERs, indicating that omalizumab is cost effective in both settings.