Evaluating Ebola Therapies — The Case for RCTs

THE NEW ENGLAND JOURNAL OF MEDICINE                                                                                 Dec. 3, 2014
By Edward Cox, M.D., M.P.H., Luciana Borio, M.D., and Robert Temple, M.D.

...Studying investigational therapies for EVD presents scientific, practical, and ethical challenges. Not surprisingly, there has been substantial debate about the best and most appropriate study approaches.2,3 It is generally agreed that a trial with a concurrent control group, in which patients are randomly assigned to receive the test drug plus the best available supportive care (BASC) or to BASC alone, would be the most efficient and reliable way to evaluate the safety and effectiveness of candidate products.

 Some people in the health care community, however, have argued against such trials, urging instead use of a historical control — that is, making investigational drugs as widely available as their supply allows and then comparing mortality rates among treated patients with rates that would have been expected absent the drugs, on the basis of past experience with EVD.

The desire to allow all patients access to investigational drugs is understandable, but there are strong reasons to doubt the ability of such “historically controlled” studies to distinguish effective therapies from ineffective ones.

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http://www.nejm.org/doi/full/10.1056/NEJMp1414145