title: The Social Value of Pragmatic Trials.
abstract: Pragmatic trials aim to directly inform health care decision-making through the collection of so-called 'real world data' from observations of comparative treatment effects in clinical practice. In order to ensure the applicability and feasibility of a pragmatic trial, design features may be necessary that deviate from standard research ethics requirements. Examples are traditional requirements to seek written informed consent and to perform extensive data and safety monitoring. Proposals for deviations from standard research ethics practice have resulted in controversy about their ethical acceptability. One of the justifications for altered procedures is the allegedly high social value of pragmatic trials. In order to properly operationalize the concept in the ethical assessment of pragmatic trial designs, specification is warranted. We identified three determinants from common claims about a pragmatic trial's social value: (1) the extent to which the research question has real world relevance, (2) the trial design's ability to generate a real world answer and (3) the probability of direct uptake of the results by decision-makers in practice. Subsequently, we discuss how these determinants should be applied to the practice of pragmatic trials, and to what extent they might be applicable to explanatory trials.
authors.forename: Shona
authors.initials: S
authors.lastname: Kalkman
authors.forename: Ghislaine
authors.initials: G
authors.lastname: van Thiel
authors.forename: Rieke
authors.initials: R
authors.lastname: van der Graaf
authors.forename: Mira
authors.initials: M
authors.lastname: Zuidgeest
authors.forename: Iris
authors.initials: I
authors.lastname: Goetz
authors.forename: Diederick
authors.initials: D
authors.lastname: Grobbee
authors.forename: Johannes
authors.initials: J
authors.lastname: van Delden
mesh_terms: D032984:Ethics, Research; D006801:Humans; D007258:Informed Consent; D012107:Research Design; D012945:Social Values
keywords: comparative effectiveness; pragmatic trials; real world evidence; research ethics; social value