Value in Health
July–August 2017 Volume 20, Issue 7, p837-1002
ISPOR KNOWLEDGE PRODUCTS
Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report
Katy Benjamin, Margaret K. Vernon, Donald L. Patrick, Eleanor Perfetto, Sandra Nestler-Parr, Laurie Burke
p838–855 Published in issue: July-August, 2017
Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Trials
p856–857 Published in issue: July-August, 2017
Ethics of Informed Consent for Pragmatic Trials with New Interventions
Shona Kalkman, Scott Y.H. Kim, Ghislaine J.M.W. van Thiel, Diederick E. Grobbee, Johannes J.M. van Delden
p902–908 Published online: May 16, 2017
Objectives: Pragmatic trials evaluate the comparative benefits, risks, and burdens of health care interventions in real-world conditions. Such studies are now recognized as valuable to the perimarketing stage of drug development and evaluation, with early pragmatic trials (EPTs) being explored as a means to generate real-world evidence at the time of regulatory market approval. In this article, we present an analysis of the ethical issues involved in informed consent for EPTs, in light of the generally recognized concern that traditional ethical rules governing randomized clinical trials, such as lengthy informed consent procedures, could threaten the “real world” nature of such trials. Specifically, we examine to what extent modifications (waivers or alterations) to regulatory consent for EPTs would be ethical.
Methods: We first identify broadly accepted necessary conditions for modifications of informed consent (namely, the research involves no more than minimal risk of harm, the research is impracticable with regulatory consent, and the alternative to regulatory consent does not violate legitimate patient expectations) and then apply those criteria to the premarket and early postmarket contexts.
Results and Conclusions: The analysis shows that neither waivers nor alterations of regulatory consent for premarket EPTs will be ethically permissible. For postmarket EPTs with newly approved interventions, waivers of consent will be ethically problematic, but some studies might be conducted in an ethical manner with alterations to regulatory consent.