Rinsho yakuri/Japanese Journal of Clinical Pharmacology and Therapeutics

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World Health Organization (WHO) has recognized the recent Ebola outbreak as exceptionally uncontrollable circumstances, and their expert committee concluded unanimously that it would be ethically acceptable to use unregistered interventions that have never before been tested for safety and efficacy in humans, under certain conditions. In this article, we mainly discuss the conditions set by WHO, which would allow the use of unproven drugs. These conditions include (1) public health measures remain the priority in outbreak control, (2) guided by traditional ethical criteria, (3) based on the best possible assessment of risk and benefit, (4) demonstrated to be safe and efficacious in non-human primates, (5) shared understanding of the criteria for compassionate use (CU), (6) uncertainty of safety in humans transparently communicated to all stakeholders, (7) sufficient capacity for experimental therapy and monitoring, (8) transparent collection and sharing of scientifically useful data from use of the agents, (9) avoid interfering with properly designed clinical studies, and (10) concerns about the impact on the health systems in the affected countries. In particular, safety and efficacy in primates are considered to be the minimum criteria for compassionate use of an unauthorized experimental drug under severely emergent circumstances, such as public health crisis. In Japan, introduction of CU programs has been expected to be an option for the treatment of refractory and rare diseases. Discussion about the use of candidate drugs in pre-clinical trial phase for Ebola hemorrhagic fever provides valuable information to establish appropriate guidelines for the introduction of CU to Japan.

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