BACKGROUND: Olodaterol and indacaterol are authorised only for the treatment of adults with chronic obstructive pulmonary disease (COPD). Use of these drugs in patients with asthma, irrespective of COPD, has been associated with increased morbidity and mortality, and is considered off-label.
OBJECTIVES: To quantify the potential off-label use of olodaterol and indacaterol and characterize use in clinical practice.
METHODS: This descriptive drug utilization study used routinely collected health data in the PHARMO Database Network in the Netherlands (PHARMO overall and PHARMO-GP for the subset of patients with general practitioner [GP] data), the population registers in Denmark, and the IMS Real-World Evidence Longitudinal Patient Database (RWE LPD) (GP panel and pulmonologist panel) in France. All new users (treatment naïve) of olodaterol or indacaterol between 2014 and 2017 were included in the study. On-label use was defined as use among adults with a recorded diagnosis of COPD. Off-label use was use among adults with a recorded diagnosis of asthma without a recorded diagnosis of COPD or as use among patients ≤ 18 years. Potential off-label use was defined as no recorded diagnosis of either COPD or asthma.
RESULTS: The study included 4,158 new users of olodaterol (1,386 in PHARMO, 1,712 in Danish National Registers, and 1,060 in the RWE LDP panels) and 9,966 new users of indacaterol (1,841 in PHARMO, 6,406 in Danish population registers, and 1,719 in the RWE LDP panels). For both drugs, median patient age at treatment start ranged from 63 to 71 years, and approximately 50% were female across the data sources. Initiators of both drugs were similar regarding comorbidity burden and comedications. Prevalence of off-label use ranged from 3.5% for both drugs (PHARMO overall) to 11.9% for olodaterol and 12.4% for indacaterol (RWE LPD GP panel). Prevalence of on-label use ranged from 47.8% (PHARMO overall) to 77.7% (RWE LPD pulmonologist panel) for olodaterol and from 28.7% (Danish population registers) to 70.1% (RWE LPD pulmonologist panel) for indacaterol. The remaining new users of olodaterol and indacaterol were classified as potential off-label users, with prevalence ranging from 17.3% (RWE LPD pulmonologist panel) to 48.6% (PHARMO overall) for olodaterol, and from 20.5% (RWE LPD pulmonologist panel) to 66.6% (Danish population registers) for indacaterol.
CONCLUSIONS: The prevalence of off-label use of both drugs was lower than reported in other studies evaluating off-label use of medications across Europe, and there was no paediatric use. Lack of direct evidence on indication (potential off-label) for a high proportion of patients is a limitation for some data sources.
