Randomized controlled trials and real life studies. Approaches and methodologies: a clinical point of view.
Open accessAbstract
Randomized Controlled Trials (RCTs) are the "gold standard" for evaluating treatment outcomes providing information on treatments "efficacy". They are designed to test a therapeutic hypothesis under optimal setting in the absence of confounding factors. For this reason they have high internal validity. The strict and controlled conditions in which they are conducted, leads to low generalizability because they are performed in conditions very different from real life usual care. Conversely, real life studies inform on the "effectiveness" of a treatment, that is, the measure of the extent to which an intervention does what is intended to do in routine circumstances. At variance to RCTs, real life trials have high generalizability, but low internal validity. Recently the number of real life studies has been rapidly growing in different areas of respiratory medicine, particularly in asthma and COPD. The role of such studies is becoming a hot topic in respiratory medicine, attracting research interest and debate. In the first part of this review we discuss some of the advantages and disadvantages of different types of RCTs and analyze the strengths and weaknesses of real life trials, considering the recent examples of some studies conducted in COPD. We then discuss methodological approaches and options to overcome some of the limitations of real life studies. Comparing the conclusions of effectiveness and efficacy trials can provide important pieces of information. Indeed, these approaches can result complementary, and they can guide the interpretation of each other results.
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Effectiveness studies could confirm the outcomes of RCTs.
“However, effectiveness studies could confirm outcomes of RCTs (Berg et al., 2020; Saturni et al., 2014).”
From Effectiveness of web-based guided self-help cognitive behavioral therapy-enhanced for binge-eating disorder: An implementation study (2024) · cites this paper as Saturni et al., 2014
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References 139 · Cited by 245
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- Authors
- Saturni, S., Bellini, F., Braido, F., Paggiaro, P., Sanduzzi, A., Scichilone, N., Santus, P. A., Morandi, L., & Papi, A.
- Affiliations
- University of Ferrara (IT)
- Ospedale Policlinico San Martino (IT)
- Azienda Ospedaliera Universitaria Pisana (IT)
- University of Naples Federico II (IT)
- University of Palermo (IT)
- University of Milan (IT)
- Corresponding author
- Papi, A.
- Venue
- Pulmonary Pharmacology & Therapeutics
- Publisher
- Elsevier BV
- Published
- 2014-04
- Volume
- 27
- Issue
- 2
- Pages
- 129-138
- ISSN
- 1094-5539 (ISSN-L)
- OpenAlex
- W2101568459
- Cited by
- 245
- References
- 139
- Citations by year
- 2026: 12 · 2025: 17 · 2024: 17 · 2023: 24 · 2022: 25 · 2021: 30 · 2020: 29 · 2019: 25 · 2018: 28 · 2017: 20 · 2016: 7 · 2015: 7 · 2014: 3 · 2013: 1
- FWCI
- 1.56
- Type
- journal article
- Language
- en
- Open access
- green
- hdl.handle.net
- Licence
- other-oa
- Locations
- Pulmonary Pharmacology & Therapeutics (publishedVersion)
- PubMed (publishedVersion)
- CINECA IRIS Institutial research information system (University of Pisa) (submittedVersion)
- CINECA IRIS Institutial Research Information System (University of Genoa) (submittedVersion)
- Nova Science Publishers (Nova Science Publishers, Inc.) (submittedVersion, other-oa)
- Institutional Research Information System University of Ferrara (University of Ferrara) (submittedVersion)
- Topics
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Chronic Obstructive Pulmonary Disease (COPD) Research
- Physical Sciences › Mathematics › Statistics and Probability › Advanced Causal Inference Techniques
- Keywords
- randomized controlled trials, real-life study, efficacy versus effectiveness, generalizability, internal validity, chronic obstructive pulmonary disease, asthma, respiratory medicine
- MeSH terms
- Asthma, Humans, Research Design, Confounding Factors, Epidemiologic, Randomized Controlled Trials as Topic, Pulmonary Disease, Chronic Obstructive
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