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Review
. 2020 Jun 2:15:1225-1243.
doi: 10.2147/COPD.S244942. eCollection 2020.

Comparing Randomized Controlled Trials and Real-World Studies in Chronic Obstructive Pulmonary Disease Pharmacotherapy

Donald P Tashkin  1 , Alpesh N Amin  2 , Edward M Kerwin  3
Affiliations
Review

Comparing Randomized Controlled Trials and Real-World Studies in Chronic Obstructive Pulmonary Disease Pharmacotherapy

Donald P Tashkin et al. Int J Chron Obstruct Pulmon Dis. .

Abstract

Analytic epidemiological studies cover a large spectrum of study methodologies, ranging from noninterventional observational studies (population-based, case-control, or cohort studies) to interventional studies (clinical trials). Herein, we review the different research methodologies or study designs and discuss their advantages and disadvantages in the context of chronic obstructive pulmonary disease (COPD) pharmacotherapy. Although randomized controlled trials (RCTs) are considered the "gold standard" for evaluating the efficacy and safety of an intervention, observational studies conducted in a real-world scenario are useful in providing evidence on the effectiveness of the intervention in clinical practice; understanding both efficacy and effectiveness is important from the clinician's perspective. Pragmatic clinical trials that use real-world data while retaining randomization bridge the gap between explanatory RCTs and noninterventional observational studies. Overall, different study designs have their associated advantages and disadvantages; together, findings from all types of studies bring about progress in clinical research as elucidated through examples from COPD research in this paper.

Keywords: COPD; clinical trials; pharmacotherapy; study designs.

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Conflict of interest statement

Dr. Tashkin has participated as an advisory board member and speaker for Boehringer Ingelheim, AstraZeneca, and Sunovion. He has also worked as a consultant for Mylan and Innoviva/Theravance. He also reports personal fees from AstraZeneca, outside the submitted work. Dr. Amin has worked as a consultant and/or speaker for Boehringer Ingelheim, Sunovion, BMS, Pfizer, Portola, and AstraZeneca. He also reports personal fees from Boehringer Ingelheim, Sunovion, BMS, Pfizer, Portola, and Astra Zeneca, outside the submitted work. Dr. Kerwin has participated in consulting, advisory boards, and speaker panels for, or has received travel reimbursement from, Amphastar, AstraZeneca, Boehringer Ingelheim, Cipla, GlaxoSmithKline, Mylan, Novartis, Oriel, Pearl, Sunovion, Teva, and Theravance. He has conducted multicenter clinical research trials for approximately 40 pharmaceutical companies. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
Overview of study designs.
Figure 2
Figure 2
Research process for new drug development: possible sequence of research designs used. Abbreviations: RCT, randomized controlled trial.
See this image and copyright information in PMC

References

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MeSH terms