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Published on: September 20, 2019
Pragmatic randomized trials in drug development pose new ethical questions: a systematic review
Shona Kalkman1, Ghislaine J M W van Thiel1, Diederick E Grobbee2
1Department of Medical Humanities, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Heidelberglaan 100, PO Box 85500, 3508 GA Utrecht, The Netherlands.
Abstract:
Implementation of pragmatic design elements in drug development could bridge the evidence gap that currently exists between the knowledge we have regarding the efficacy of a drug versus its true, comparative effectiveness in real life. We performed a review of the literature to identify the ethical challenges thus far related to pragmatic trials. The three central ethical questions identified for pragmatic trials are: (i) what level of oversight should pragmatic trials require; (ii) do randomized patients face additional risks; and (iii) is a waiver of informed consent ethically defensible? Despite the fact all reviewed publications dealt with post-launch pragmatic trials, these results could serve as an important starting point for conceptualizing which challenges could potentially arise in the pre-launch setting.
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