Comparative effectiveness of drugs for chronic obstructive pulmonary disease

Abstract

Current guidelines for the management of chronic obstructive pulmonary disease (COPD) recommend the use of inhaled long-acting bronchodilators, inhaled corticosteroids (ICSs) and their combinations for maintenance treatment of moderate to severe COPD. However, doctors still wonder if in patients with mild/moderate stable COPD it is best to start with a β-adrenoceptor agonist or an antimuscarinic agent. They also wonder if once- or twice-daily dosing is preferable, and if it is enough to develop a novel therapy that is dosed once daily rather than twice daily if the agents are both equally safe and effective. It also remains unclear whether and when a second bronchodilator with a different mechanism of action should be used in patients with stable COPD and when, in its place, an ICS must be added, and also whether long-acting antimuscarinic agent (LAMA)/long-acting β-adrenoceptor agonist (LABA) combination therapy is preferred over LAMA plus LABA/ICS. Moreover, there is no solid evidence of the best way to administer a triple combination product: should drugs be delivered concomitantly or sequentially? In any case, the growing evidence that COPD is a heterogeneous disease with characteristics that occur with different phenotypes suggests that a specific therapy may not be ultimately identified for every phenotype. Therefore, there is a clear need to move toward personalized treatment in COPD because phenotypic heterogeneity may affect treatment response and the clinical course of the disease. Unfortunately, however, there is not enough money or time to examine the impact of each treatment step or combination of treatments in each specific phenotype using randomized controlled trials. Consequently, doctors wonder if there is a role for comparative effectiveness research (CER), which can be considered a subset of patient-oriented research that examines available therapeutic options in particular patients to determine relevant health outcomes. There is a strong agreement that carefully designed CER studies, which explicitly address treatment and population heterogeneity, will provide exciting opportunities to develop an evidence base for guiding decisions about how best to tailor care for individual patients with COPD.