BALANCING ACCESS AND EFFICACY: REALIZING THE POTENTIAL OF STEM CELL THERAPIES IN THE UNITED STATES WITH BEST PRACTICES FROM JAPAN

Abstract

We are currently on the cusp of a revolution in medicine that utilizes human cells and tissues to revitalize, regenerate and cure human conditions. Stem cell therapy is a cornerstone of regenerative medicine and decades of promising preliminary clinical findings have resulted in rising anticipation by patients, physicians, investors and other affected stakeholders for imminent clinical translation and adoption of these therapies in routine care. However, expectations have been repeatedly unmet. Simultaneously, there has been a proliferation of stem cell clinics in the United States providing unproven and sometimes unsafe SCTs to patients who pay out-of- pocket for them.

Thus, in 2017 the Food and Drug Administration (FDA) published four guidance documents that clarified their regulatory framework for stem cell therapies in the United States with the stated goals of (1) keeping patients safe from physical harm and financial exploitation by unethical stem cell clinics and (2) facilitating innovation, timely clinical translation and delivery of advanced stem cell therapies. However, three years later, hundreds of clinics continue to provide a vast array of unproven stem cell therapies direct-to-consumers and are non-compliant with FDA regulations. Additionally, no stem cell-based therapies have yet achieved clinical translation. Thus, this thesis aims to identify why these concerning trends are continuing in spite of the FDA’s revised regulatory framework and its intentions, and to determine how the FDA can better support the accomplishment of their two main goals.

This thesis first identifies how the FDA’s present regulations are leaving patients vulnerable and inhibiting clinical translation. It then explores Japan’s unique regulatory framework for regenerative medicine and identifies best practices applicable to the United States. Ultimately, I provide policy recommendations for the FDA that center around two main objectives: (1) Facilitating the production of conclusive safety and efficacy evidence as the basis for reasonable healthcare decisions by all stakeholders and (2) Coordinating federal, state and private sector regulatory stakeholders to proactively ensure patient safety in the direct-to- consumer stem cell therapy market.