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Formative research in clinical trial development: attitudes of patients with arthritis in enhancing prevention trials
  1. Holly A Taylor1,
  2. Jeremy Sugarman1,
  3. David S Pisetsky2,
  4. Joan Bathon3
  1. 1Berman Institute of Bioethics, Department of Health Policy and Management, Johns Hopkins University, Baltimore, Maryland, USA
  2. 2Division of Rheumatology and Immunology, Duke University Medical Center, Durham VA Medical Center, Durham, North Carolina, USA
  3. 3Division of Rheumatology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA
  1. Correspondence to:
    Dr H A Taylor
    Berman Institute of Bioethics, Department of Health Policy and Management, Johns Hopkins University, Hampton House, 353 624 N Broadway, Baltimore, MD 21205, USA; htaylor{at}


In preparation for randomised controlled trials (RCTs) of disease-modifying antirheumatic drugs in patients with early inflammatory arthritis (EIA), formative research was conducted to enhance the design of such trials. The objectives of this research were to (1) determine patients’ educational needs as they relate to the necessary elements of informed consent; and (2) assess patients’ interest in enrolling in a hypothetical prevention trial. In-depth interviews were conducted with nine patients. Seven patients were women and all but one white. The mean age was 48 years. During the 4-month enrolment period, only three patients with EIA were identified; six patients with longer duration of symptoms were also interviewed. Most patients were able to express the primary aim of a hypothetical prevention trial presented. Factors cited by patients favouring enrolment were potential for direct medical benefit and knowledge that they would be withdrawn from the trial if they developed symptoms. Factors cited by patients against enrolment were the inclusion of a placebo and general uncertainty regarding treatment required by the RCT design. Pending larger-scale empirical projects to explore patients’ attitudes about prevention trials, small-scale formative research in advance of such trials ought to be conducted.

  • DMARD, disease-modifying antirheumatic drug
  • EIA, early inflammatory arthritis
  • RCT, randomised controlled trial

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  • Competing interests: HAT, JS and DP have no competing interests to report. JB receives grant funding from Amgen, Bristol Myers Squibb, IDEC/Genentech and Roche.

  • The sponsor was not involved with study design, data collection, analysis or preparation of the manuscript.

    Contributions: HAT, JS and DSP conceived the study design. HAT took the primary responsibility for drafting interview materials, conducting the interviews, analyses and initial drafting of the manuscript. JB facilitated recruitment of participants. JS, JB and DSP helped to refine interview materials and versions of the manuscript. All authors read and approved the final manuscript.

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