Science
13 February 2015 vol 347, issue 6223, pages 689-800
http://www.sciencemag.org/current.dtl
In Depth
Infectious Diseases
Ebola drug trials lurch ahead
Kai Kupferschmidt, Jon Cohen
News leaked last week that the drug favipiravir worked in some Ebola patients, but even researchers running the study in Guinea said questions remain about its efficacy. Interpreting the data is difficult because there is no control arm that treated patients can be compared with. This is just one of several confusing twists in the search for a treatment that can stave off death and disease from Ebola virus. Chimerix, the maker of an antiviral called brincidofovir, surprised investigators in Liberia when it suddenly ended a study of its drug after discussions with the U.S. Food and Drug Administration. Chimerix noted that the study was having trouble enrolling patients because Liberia has seen a steep drop in cases, but the researchers running it said they had hoped to expand the trial to Sierra Leone, where most new infections are happening. Liberia is also beginning another trial with ZMapp, a cocktail of Ebola antibodies, and study leaders are having difficulty convincing Sierra Leone and Guinea to join because the study uses a placebo control. Finally, a trial of convalescent serum taken from recovered patients is getting under way in Guinea, but there are now questions about whether it should be compared with favipiravir as a control.
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Policy Forum
Health Care Policy
Randomize evaluations to improve health care delivery
Amy Finkelstein1,2,3,*, Sarah Taubman2
Author Affiliations
1Department of Economics, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
2J-PAL North America, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
3National Bureau of Economic Research, Cambridge, MA 02138, USA.
The medical profession has long recognized the importance of randomized evaluations; such designs are commonly used to evaluate the safety and efficacy of medical innovations such as drugs and devices. Unfortunately, innovations in how health care is delivered (e.g., health insurance structures, interventions to encourage the use of appropriate care, and care coordination approaches) are rarely evaluated using randomization. We consider barriers to conducting randomized trials in this setting and suggest ways for overcoming them. Randomized evaluations of fundamental issues in health care policy and delivery should be—and can be—closer to the norm than the exception.