Real-World Usefulness Is Missing
In the article by Lange et. al. (1), important aspects of real-world use are not mentioned. Randomized controlled trials (RCTs) are the gold standard for clinical trials in which the efficacy of an intervention under ideal conditions is examined. The primary goal of demonstrating efficacy is the answer to the question of evidence-based medicine: „Can it work?“ (1). The clarification of this question is classified as an experiment on humans and requires ideal conditions.
RCTs can no longer be required as a gold standard for clinical trials in which the effectiveness of an intervention under real-world conditions is examined. The primary goals for demonstrating effectiveness and fitness for real-world use relate to answering the questions „does it work, and is it worth it?“ (2). Examining these two properties requires real-world conditions and instruments other than proof of efficacy (3).
Real-world conditions include co-morbidities and their therapies, which are perceived by researchers as disturbing factors. However, both questions of „does it work?“ and „is it worth it?“ can only be answered considering real-world care.
Testing for effectiveness is essential. Disturbing factors influence care outcome positively or negatively. The lack of equipoise prevents patients from participating in RCTs. „Equipoise“ refers to the perceived comparability of two therapies, which—from the patients‘ point of view—should not be considered when, for example, the advantages and disadvantages of loss of a limb in surgery is weighed against radiotherapy for organ preservation.
As the correspondence format limits the number of arguments that can be presented, and the number of authors who can co-sign, we refer readers to our homepage www.clinical-economics.com for more information.
Prof. Dr. med. Franz Porzsolt
President, Institute of Clinical Economics (ICE) e.V., Ulm, Germany
Prof. Dr. med. Karl-Walter Jauch
Head of Board, Hospital of the Ludwig-Maximilians-Universität München, Germany
Conflict of interest statement
The authors declare that no conflict of interest exists.
|1.||Lange S, Sauerland S, Lauterberg J, Windeler J: The range and scientific value of randomized trials—part 24 of a series on evaluation of scientific publications. Dtsch Arztebl Int 2017; 114: 635–40 VOLLTEXT|
|2.||Haynes B: Can it work? Does it work? Is it worth it? The testing of healthcare intervention is evolving. BMJ 1999; 319: 652–3. CrossRef PubMed Central|
|3.||Porzsolt F, Rocha NG, Toledo-Arruda AC, et al.: Efficacy and effectiveness trials have different goals, use different tools, and generate different messages. Pragmat Obs Res 2015; 6: 47–54 CrossRef MEDLINE PubMed Central|