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© 2008 The American Thoracic Society
doi: 10.1513/pats.200802-019SK
The Ethics of Randomized Clinical Trials in Pulmonary Arterial Hypertension
1 Division of Pulmonary, Allergy, and Critical Care Medicine, 2 Center for Bioethics, 3 Center for Clinical Epidemiology and Biostatistics, and 4 Leonard Davis Institute of Health Economics, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania; 5 Wall Center for Pulmonary Vascular Disease, Division of Pulmonary and Critical Care Medicine, Stanford University, Palo Alto, California; 6 Gilead Sciences, Inc., Foster City, California; and 7 Department of Medicine, College of Physicians and Surgeons, and 8 Department of Epidemiology, Joseph L. Mailman School of Public Health, Columbia University, New York, New York
Correspondence and requests for reprints should be addressed to Scott D. Halpern, M.D., Ph.D., M.Bioethics, Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania School of Medicine, 711 Blockley Hall, 423 Guardian Drive, Philadelphia, PA 19104-6021. E-mail: scott.halpern{at}uphs.upenn.edu
ABSTRACT
Randomized clinical trials (RCTs) conducted during the past decade have shown that several therapies produce improvements in surrogate endpoints for patients with pulmonary arterial hypertension (PAH). Whether these therapies also influence clinical outcomes remains uncertain. These changes in the PAH landscape have raised several complex ethical issues regarding the conduct of RCTs in PAH, but to date, these issues have not been fully explored. In this article, we consider patients' potential motives for enrolling in PAH RCTs and identify those that are ethically acceptable. Second, we consider the efficiency of PAH RCTs—the ratio of the value of the information gained from RCTs to the risks and costs of obtaining it—and how this efficiency quotient contributes to a trial's ethics by enabling the fulfillment of patients' motives for participating. Third, we discuss the ethics of PAH RCTs using placebo control subjects versus those using active-treatment control subjects. Finally, we consider the ethical issues surrounding the roles of physician-investigators in PAH RCTs. We conclude with several recommendations, including (1) that investigators seek to ensure that patients enrolling in RCTs do so primarily to fulfill altruistic motives, (2) that research be conducted to determine the long-term risks associated with brief periods of withholding PAH therapies before further placebo-controlled trials without background therapies are conducted in PAH, and (3) that incentives for investigators to enroll more patients in PAH RCTs, such as enrollment-based authorship, be eliminated.
Key Words: ethics • pulmonary hypertension • randomized trials • placebo-controlled trials • conflict of interest
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Proceedings of the American Thoracic Society 2008 5: 610-616.[Abstract] [Full Text] - Surrogate and Combined End Points in Pulmonary Arterial Hypertension
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This article has been cited by other articles:
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  M. Gomberg-Maitland Traditional and Alternative Designs for Pulmonary Arterial Hypertension Trials Proceedings of the ATS, July 15, 2008; 5(5): 610 - 616. [Abstract] [Full Text] [PDF]
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