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Clinical Trials and Medical Care: Defining the Therapeutic Misconception

  • Gail E Henderson mail,

    To whom correspondence should be addressed. E-mail:

  • Larry R Churchill,
  • Arlene M Davis,
  • Michele M Easter,
  • Christine Grady,
  • Steven Joffe,
  • Nancy Kass,
  • Nancy M. P King,
  • Charles W Lidz,
  • Franklin G Miller,
  • Daniel K Nelson,
  • Jeffrey Peppercorn,
  • Barbra Bluestone Rothschild,
  • Pamela Sankar,
  • Benjamin S Wilfond,
  • Catherine R Zimmer
  • Published: November 27, 2007
  • DOI: 10.1371/journal.pmed.0040324

Reader Comments (7)

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Author's response to "Is the failure to recognise, outside of clinical trials, uncertainty about the effects of healthcare interventions the greatest therapeutic misconception?" (Dr Mike Clarke)

Posted by plosmedicine on 31 Mar 2009 at 00:20 GMT

Author: Jeffrey Peppercorn
Position: Assistant Professor of Medicine
Institution: Division of Hematology and Oncology, University of North Carolina at Chapel Hill
Submitted Date: February 13, 2008
Published Date: February 13, 2008
This comment was originally posted as a “Reader Response” on the publication date indicated above. All Reader Responses are now available as comments.

As Dr. Clarke points out, there is some uncertainty involved with any medical intervention, but there is a substantial difference between taking an intervention that has proven safe and effective in well designed clinical trials and subjecting a patient to the uncertainty that it will help and be tolerable for them, and the uncertainty of benefit or risk from an intervention whose safety and efficacy in any patient have not yet been defined. We agree that patients treated outside of clinic trials should understand what is and is not known about the risks and benefits of any medical intervention and should make an informed choice regarding their treatment.

The larger problem that Dr. Clarke alludes to is the availability of some unproven or experimental interventions outside of clinical trials. In this case, the nature of uncertainty of likelihood of benefit or harm is comparable to that of a clinical trial, but the regulatory and procedural protections that guide human subjects research such as monitoring for patient safety, protocol driven care (in the absence of data to guide case by case management), and a requirement for informed consent, may not occur outside of a trial. It is possible that patients treated with unproven interventions outside of a trial may confuse this with evidence based medicine, and that this could compromise their decision making or consent to therapy. However, there is still an important distinction that we must make between use of unproven therapy with the sole goal of providing benefit to an individual, and research, which by definition includes the goal of producing generalizable knowledge. In a clinical trial, regardless of the potential for benefit to the individual, we are in some sense using human beings towards an end other than themselves (or in addition to their own welfare), and they should understand and consent to this.

The term “therapeutic misconception” could be applied to other misconceptions about therapy, but as in its original formulation, in this manuscript we focused on misunderstanding of research that can arise as a result of confusing care in the research setting (which may or may not include therapeutic intent) and care in the standard medical practice setting (which may or may not involve proven therapies). As Dr. Clarke illustrates, in addition to improving understanding of clinical research among research participants, there is a need to better define the ethics of access to unproven therapies outside of clinical trials. Several of the authors of this manuscript (JP, FM, SJ, GH) are actively working in this area (See Peppercorn et al., Journal of Clinical Oncology, 2006 ASCO Annual Meeting Proceedings. Vol 24, No 18S (June 20 Supplement), 2006: 6047).

Jeffrey Peppercorn, MD, MPH

Competing interests declared: I am a co-author of this paper.