Are Asthmatics Enrolled in Randomized Trials Representative of Real-Life Outpatients?

Abstract

Background/Objective: This study was aimed at exploring to what extent populations enrolled in randomized controlled trials (RCTs) of inhalation combination treatment for mild/moderate asthma in adults are fully representative of ‘real-life' populations. The following is a retrospective analysis of the clinical records of outpatient subjects with an ascertained diagnosis of asthma. Methods: A retrospective analysis was performed. Stable conditions, such as smoking habit and chronic diseases other than asthma, were identified as exclusion criteria for RCTs. The selected criteria were then applied to asthmatic outpatients, yielding a population that was potentially eligible for RCTs. Results: Out of 1,909 subjects, 824 (43.2%) met at least one of the exclusion criteria for RCTs. Cigarette smoking (occurring in 34.3% of the entire population), lung diseases other than asthma (5.0%), anxiety and depression (3.3%), arrhythmias (2.3%), and coronary artery disease (1.2%) would have been the most frequent causes for exclusion from RCTs. The proportion of patients excluded from RCTs appears to increase with age, reaching 57.1% in patients aged >85 years. Conclusions: In a real-life setting, >40% of subjects with mild/moderate asthma are currently treated by protocols based on the results of RCTs for which they would not have been eligible. This proportion increases in elderly patients with comorbidities. These findings limit the generalizability of RCTs and advocate that complementary pragmatic studies be conducted.

Eligibility of real-life patients with COPD for inclusion in RCTs: a commentary

“…In this contest, the probability that a patient with COPD and concomitant atrial fibrillation is treated without any scientific evidence or safety data is high. Similar observations have been produced in chronic diseases other than COPD [9, 10]. Although under some conditions the eligibility rate could be as high as 58%, overall almost three out of four patients from real life settings are not eligible for RCTs and this should, in our opinion, limit the RCTs generalisability.…”

Drop-out rate among patients treated with omalizumab for severe asthma: Literature review and real-life experience

“…In fact, lack of efficacy, similarly to the published Real life studies [25–42], accounted for 26 % of drop-outs among NEONet patients, despite all patients being fully matched with the current prescription criteria. In the real-life setting, many patient-related variables, such as smoking habits, comorbidities, and multi-drug treatments, may affect treatment efficacy and effectiveness [51, 61], even though prescription criteria are verified. In this scenario patient’s education, in terms of awareness of the treatment and its implications, has an even more relevant role in preventing drop-outs and generally supporting adherence [62, 63].…”

Inhalation therapy in the next decade: Determinants of adherence to treatment in asthma and COPD

“…As a consequence, the extrapolation of the RCTs results to the everyday clinical practice could be potentially limited. In asthma, more than 40% of subjects with mild/moderate disease are currently treated by protocols based on the results of RCTs for which they would have not been eligible [233]. This proportion increases in elderly patient with comorbidities.…”