Current Status of Patients' Access to Receiving Investigational Treatments in the USA: A History of The Federal Right to Try Act

Abstract

The United States of America (USA) is one of the leading countries that have been developing a program for patients with terminal illness to receive investigational treatments. In May 2018, President Trump signed into law the federal Right to Try Act (the federal RTT act). This law amends the Federal Food, Drug, and Cosmetic Act to allow more direct access to investigational drugs for patients with terminal illnesses who have exhausted available treatment options and who do not qualify for a clinical trial. There has been a huge controversy regarding the federal and state RTT acts in the USA because of their contents, such as the definition of eligible patients and drugs, ethical review, informed consent, safety monitoring, and incentives for pharmaceutical companies. The proponents of the RTT acts have stated that the existing expanded access program by the FDA has saved a limited number of patients due to the huge burden of documentation by physicians and the time-consuming review by the FDA. The opponents to the RTT acts have pointed out that the federal RTT act has done little to change pharmaceutical companies' practices compared to those under the expanded access program by the FDA. This paper aims to provide a history of the development of RTT acts in the USA and to understand the controversial points of the RTT pathway to receive investigational treatments.