• Shona Kalkman, Ghislaine J M W van Thiel, Diederick E Grobbee, Anna-Katharina Meinecke, ZuidgeestMira G PMGDepartment of Epidemiology, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands., Johannes J M van Delden, and Work Package 3 of the IMI GetReal Consortium.
• Department of Medical Humanities, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, PO Box 85500, 3508, GA, Utrecht, The Netherlands. s.kalkman@umcutrecht.nl.
• Trials. 2016 Aug 22; 17 (1): 419.

BackgroundWe explored the views of key stakeholders to identify the ethical challenges of pragmatic trials investigating pharmaceutical drugs. A secondary aim was to capture stakeholders' attitudes towards the implementation of pragmatic trials in the drug development process.MethodsWe conducted semistructured, in-depth interviews among individuals from different key stakeholder groups (academia and independent research institutions, the pharmaceutical industry, regulators, Health Technology Assessment (HTA) agencies and patients' organizations) through telephone or face-to-face sessions. Interviews were structured around the question "what challenges were experienced or perceived during the design, conduct and/or review of pragmatic trials." Respondents were additionally asked about their views on implementation of pragmatic trials in the drug development process. Thematic analysis was used to identify the ethically relevant features across data sets.ResultsWe interviewed 34 stakeholders in 25 individual sessions and four group sessions. The four perceived challenges of ethical relevance were: (1) less controlled conditions creating safety concerns, (2) comparison with usual care potentially compromising clinical equipoise, (3) tailored or waivers of informed consent affecting patient autonomy, and (4) minimal interference with "real-world" practice reducing the knowledge value of trial results.ConclusionsWe identified stakeholder concerns regarding risk assessment, use of suboptimal usual care as a comparator, tailoring of informed consent procedures and ensuring the social value of pragmatic trials. These concerns increased when respondents were asked about pragmatic trials conducted before market authorization.

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