Comparative Assessment of Non-trial Access to Investigational Medical Products in the U.S. and Japan.

The U.S. and Japan have preapproval access systems for patients who have exhausted approved treatment options. We compare the systems of both countries and discuss some ethical concerns surrounding non-trial access to investigational drugs. Notable points of comparison of the two countries' systems include cost of non-trial access to investigational drugs and purpose of the non-trial access system. All stakeholders and the public must understand the ethical issues associated with non-trial preapproval access systems. The current non-trial access programs in both countries depends on pharmaceutical companies' voluntary decisions about whether patients get access to investigational products. Moreover, the potential for inequity of access raises ethical concerns. Non-trial preapproval access is an exceptional way to practice medicine with various ethical and safety concerns, so we suggest that the scope and eligibility for using these pathways should thus be limited.