Randomized Controlled Trials vs. Real-World Studies in COPD Pharmacotherapy: Any Preferences?

Randomized Controlled Trials vs. Real-World Studies in COPD Pharmacotherapy: Any Preferences?

A study led by Donald P. Tashkin, MD with UCLA’s David Geffen School of Medicine, and colleagues from UC Irvine and the Clinical Research Institute of Southern Oregon looked into analytic epidemiological studies running the gamut from noninterventional observational studies (population-based, case-control or cohort studies) to interventional studies (clinical trials). But in the context of chronic obstructive pulmonary disease (COPD) pharmacotherapy, what is the better approach? This is not an unimportant topic given the impact of COPD on society and some of the pros and the cons—the costs and the benefits—of the different study models.

Published in Dovepress, an important supplemental video is included titled “Comparing RCTs and Real-World Studies in COPD.” For those interested in learning about the differences between randomized controlled trials and Real-Word studies—or for that matter, the pros and cons of each approach, the video is worth listening to.

The COPD Problem

COPD is a type of obstructive lung disease characterized by long-term breathing problems and poor airflow. Symptoms can range from shortness of breath to cough with sputum production. It is a progressive disease and often worsens over time. Over time, life conditions degrade as basic everyday activities become difficult. Chronic bronchitis and emphysema are older terms used for various types of COPD. Worldwide, 65 million people suffer from this disease, and about 3 million will die per year.

The Study

The investigators studied the different research approaches or study designs and consequently discussed the advantages and disadvantages in the context of COPD pharmacotherapy. Yes, randomized controlled trials (classical clinical trials) are the “gold standard” for evaluating the safety and efficacy of an intervention, but the authors argue observational studies executed in real-world scenarios can be very useful in probing for evidence on the actual effectiveness of the intervention in clinical practice.

Both RCT & RWS are Important

Both randomized controlled trials and real-world studies are important and can be complementary tools that help investigators under both efficacy and effectiveness from the clinician’s perspective.

Consider Well Designed Pragmatic Trials

Pragmatic clinical trials that use real-world data while retaining randomization can bridge the gap between the clinical trial and those real-world studies based on noninterventional observational studies.

Understand One Size Doesn’t Fit All

As it turns out, of course, RCTs and RWDs may have strengths and weaknesses depending on the situation and context; but they can be complementary if there are ways to leverage them, not to mention consider carefully designed pragmatic trials in the context of COPD. The study authors write that findings from all sorts of studies generates progress in clinical research, as explained in real-world examples from COPD in this study.

Lead Research/Investigator

Donald P. Tashkin, MD with UCLA’s David Geffen School of Medicine