Pragmatic randomized trials in drug development pose new ethical questions: A systematic review

Shona Kalkman*, Ghislaine J M W van Thiel, DE Grobbee, Johannes J M van Delden

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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.

Original languageEnglish
Pages (from-to)856–862
JournalDrug Discovery Today
Volume20
Issue number7
DOIs
Publication statusPublished - Jul 2015

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