September 29, 2009

Considering Usual Medical Care In Clinical Trial Design

In this week's PLoS Medicine, Liza Dawson (National Institutes of Health) and colleagues discuss the scientific and ethical issues associated with choosing clinical trial designs when there is no consensus on what constitutes usual care. For example, in 2002 a clinical trial designed to evaluate the best way of ventilating patients with a severe lung condition called acute respiratory distress syndrome sparked a major controversy. Critics charged that management of the condition in the different arms of the study did not adequately reflect usual medical care, and alleged that it was essential for scientific and ethical reasons to have a usual care comparison arm in the study. The controversy over trial design enmeshed the National Institutes of Health (NIH), the Office for Human Research Protections (OHRP) and the critical care research community. The trial was put on hold and reviewed by two independent expert panels. Experts pointed to the need for further analysis of the scientific and ethical issues involved in choosing trial designs when there is no consensus on standard of care. Dawson and colleagues discuss these issues in their policy paper. They enumerate five factors that make consensus on these issues particularly difficult, and recommend specific criteria for assessing proposed study designs.

Funding: The NIH funded the 2005 meeting on Considering Usual Care in Clinical Trial Design: Scientific and Ethical Issues, which involved development of a background paper and case studies which are included in this paper. This paper and its conclusions do not represent an official position or policy of the US Government, the Department of Health and Human Services, or the National Institutes of Health. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

Competing Interests: DZ is employed by the National Institutes of Health as a senior scientist, and is the Director, She has no other relevant interests. LMF is now retired but was an employee of the NIH institute (the National Heart, Lung, and Blood Institute) that funded the ARDS Network, and was a senior level NHLBI employee at the time of the controversy regarding the ARDSNet study that stimulated the conference discussed in the paper.

Citation: Dawson L, Zarin DA, Emanuel EJ, Friedman LM, Chaudhari B, et al. (2009) Considering Usual Medical Care in Clinical Trial Design. PLoS Med 6(9): e1000111. doi:10.1371/journal.pmed.1000111


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