Yale Journal of Health Policy, Law, and Ethics

Early Access to Unapproved Medicines in the United States and France

Erika Lietzan & Isabelle Moine-Dupuis

ABSTRACT. In 2018, President Trump signed a federal "right to try" law, claiming that it would give desperately ill patients earlier access to unapproved medicines, by allowing the patient, doctor, and drug company to arrange for access without federal oversight. Critics of the law argued that it would not meaningfully increase access to experimental medicines, because federal oversight was not the obstacle in the first place. And they were correct. U.S. law already permitted companies to provide terminally ill patients with early access to unapproved medicines. The problem was instead that companies did not take advantage of this option. This Article offers new insights into U.S. law on early access, as well as the new right-to-try law, by offering a comparative perspective using French law. We explore the historical, legal, and cultural differences between France and the United States that may explain differences in their early access systems and why the right-to-try law emerged in one country but not the other. The differing approaches reflect in part differing reactions to arguments grounded in personal autonomy and patients' rights, when held up against utilitarian arguments for premarket approval and traditions of medical paternalism. Using the French experience, this Article also considers the possibility that the key to increasing use of expanded access in the United States might be financial: making it worthwhile for companies, by allowing them to profit from sales, and making the medicines and associated healthcare services free for patients through insurance coverage.

AUTHOR. Erika Lietzan is an Associate Professor at the University of Missouri School of Law. She is grateful to Alec Larsen (University of Missouri School of Law Class of 2019) and Henry Adams (2020) for research assistance. Her work on this project was also supported by an Armstrong Teasdale Schlafy & Davis Faculty Research Fellowship, a John W. Cowden Faculty Research Fellowship, and a Lewis A. & Verne H. Snyder Faculty Research Fellowship. Isabelle Moine-Dupuis is an Associate Professor at the University of Burgundy and member of the Center for Research in International Trade and Investment Law (Credimi), the French Health Law Association (AFDS), and Norms, Science and Technics (NoST, CNRS). The authors are grateful to Christine Coughlin, Jeanne Fabre, Aaron Kesselheim, Paul Litton, Lyrissa Lidsky, Eveline Van Keymeulen, Tom Watson, Patricia Zettler, and three anonymous reviewers for comments and suggestions.

RECOMMENDED CITATION. Erika Lietzan & Isabelle Moine-Dupuis, Early Access to Unapproved Medicines in the United States and France, 19 Yale J. Health Pol'y L. & Ethics (2020).