Evaluating Novel Therapies During the Ebola Epidemic

The Ebola hemorrhagic fever outbreak in West Africa poses acute and novel challenges for health policy and research ethics. Faced with an exceptionally virulent infectious agent, limited resources, and danger to health workers, local and international authorities struggle to deploy proven public health techniques that can limit the spread of the disease.1 In the midst of the crisis, experimental interventions that have never been evaluated in human trials have captured professional and public attention. The prospect of first-in-human use of these interventions during an uncontrolled epidemic raises at least 4 pressing ethical questions. First, is there a role for “compassionate use” of agents in the absence of human safety, efficacy, or dosing data? Second, given the critical scarcity of these agents, which patients should receive priority access? Third, what trial designs should be used to study these agents? Fourth, how should efforts to evaluate experimental agents coexist with established clinical and public health interventions to treat patients and to minimize spread? Two fundamental principles should guide responses to these questions. Decisions must aim to prevent the maximum number of deaths during the current outbreak. Equally important, policy makers must seek to optimize knowledge gained for use in confronting future Ebola epidemics.