Schmidt, AF;
Klungel, OH;
Nielen, M;
De Boer, A;
Groenwold, RHH;
Hoes, AW;
(2016)
Tailoring treatments using treatment effect modification.
Pharmacoepidemiology and Drug Safety
, 25
(4)
pp. 355-362.
10.1002/pds.3965.
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Abstract
Background and objective: Applying results from clinical studies to individual patients can be a difficult process. Using the concept of treatment effect modification (also referred to as interaction), defined as a difference in treatment response between patient groups, we discuss whether and how treatment effects can be tailored to better meet patients' needs. / Results: First we argue that contrary to how most studies are designed, treatment effect modification should be expected. Second, given this expected heterogeneity, a small number of clinically relevant subgroups should be a priori selected, depending on the expected magnitude of effect modification, and prevalence of the patient type. Third, by defining generalizability as the absence of treatment effect modification we show that generalizability can be evaluated within the usual statistical framework of equivalence testing. Fourth, when equivalence cannot be confirmed, we address the need for further analyses and studies tailoring treatment towards groups of patients with similar response to treatment. Fifth, we argue that to properly frame, the entire body of evidence on effect modification should be quantified in a prior probability.
| Type: | Article |
|---|---|
| Title: | Tailoring treatments using treatment effect modification |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1002/pds.3965 |
| Publisher version: | http://dx.doi.org/10.1002/pds.3965 |
| Language: | English |
| Additional information: | This is the peer reviewed version of the following article: Schmidt, AF; Klungel, OH; Nielen, M; De Boer, A; Groenwold, RHH; Hoes, AW; (2016) Tailoring treatments using treatment effect modification. Pharmacoepidemiology and Drug Safety, 25 (4) pp. 355-362, which has been published in final form at: http://dx.doi.org/10.1002/pds.3965. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Self-Archiving (http://olabout.wiley.com/WileyCDA/Section/id-820227.html#terms). |
| Keywords: | randomized controlled trial; nonrandomized study design; observational study design; statistics; effect modification; interaction; generalizability; pharmacoepidemiology |
| 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 Cardiovascular Science UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Cardiovascular Science > Population Science and Experimental Medicine |
| URI: | https://discovery.ucl.ac.uk/id/eprint/1479397 |
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