Sharma, M;
Nazareth, I;
Petersen, I;
(2019)
Observational studies of treatment effectiveness: worthwhile or worthless?
Clinical Epidemiology
, 11
pp. 35-42.
10.2147/CLEP.S178723.
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Abstract
Observational studies which evaluate effectiveness are often viewed with skepticism owing to the fact that patients are not randomized to treatment, meaning that results are more prone to bias. Therefore, randomized controlled trials remain the gold standard for evaluating treatment effectiveness. However, it is not always possible to conduct randomized trials. This may be due to financial constraints, for example, in identifying funding for a randomized trial for medicines that have already gained market authorization. There can also be challenges with recruitment, for example, of people with rare conditions or in hard-to-reach population subgroups. This is why observational studies are still needed. In this manuscript, we discuss how researchers can mitigate the risk of bias in the most common type of observational study design for evaluation of treatment effectiveness, the cohort study. We outline some key issues that warrant careful consideration at the outset when the question is being developed and the cohort study is being designed. We focus our discussion on the importance of deciding when to start follow-up in a study, choosing a comparator, managing confounding and measuring outcomes. We also illustrate the application of these considerations in a more detailed case study based on an examination of comparative effectiveness of two antidiabetic treatments using data collected during routine clinical practice.
| Type: | Article |
|---|---|
| Title: | Observational studies of treatment effectiveness: worthwhile or worthless? |
| Location: | New Zealand |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.2147/CLEP.S178723 |
| Publisher version: | http://doi.org/10.2147/CLEP.S178723 |
| Language: | English |
| Additional information: | Copyright © 2019 Sharma et al. This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms. |
| Keywords: | diabetes mellitus, effectiveness, epidemiology, public health, therapeutics |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health > Epidemiology and Public Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health > Primary Care and Population Health |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10065590 |
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