Is Patients’ “Right to Try” Guaranteed by the Law?

The Overview of the Preapproval Access Pathway to the Investigational Drugs in the U.S.A.

Abstract

　The federal Right to Try（ RTT） law was enacted in 2018 in the United States where the use of investigational drugs has been actively examined. The law enables patients to have access to investigational drugs from pharmaceutical manufacturers without the need for approval by the Food and Drug Administration. Although the law can benefit patients by providing them with additional treatment options, the law may be insufficient for protecting the lives and well-being of patients and for protecting the“ right to try” as the cost coverage for investigational drugs is not regulated. Medical crowdfunding has gained significant attention from both patients and their families as a way to cover the cost of medical treatment, while it has some ethical or social issues. Based on the systems developed in the United States and Europe, the Expanded Trial and Patient-requested Therapy System were established in 2016 in Japan. While these systems provide patients with the“ right to ask to participate in a clinical trial”, they do not seem to provide individual patients with the“ right to try” a particular treatment. Future investigations are needed to evaluate the balance between providing treatment options for individual patients and collecting additional evidence.