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Perpetual debate regarding the delicate balance between access and innovation and the protection of the public health and safety dominate discussions of the United States Food and Drug Administration ("FDA"). Established chiefly as a command and control federal administrative agency, iterative changes in legislation have shaped the FDA's activity in drug, biologic, and medical device regulation over the course of the last one hundred plus years. The most recent fundamental reframing of the agency's authority and directive presented itself in the 21 st Century Cures Act, reflecting an important role for patient perspectives in the regulatory process. This Article explores recent developments in patient-focused product development efforts at the FDA and offers modest insights on the increasing role of patients, and patient advocacy groups, in agency decision-making. The Article terms this era "21 st century citizen pharma."

In 2014, the Japanese government amended the laws concerning regenerative medicine. This reform aimed to contribute to the appropriate promotion of regenerative medicine and new drug discovery for intractable diseases using stem cells. It also helped restrict stem cell tourism, that is, provision of stem cell therapy of unclear efficacy and safety to tourists from abroad, and its relaxed regulations may even lead to the resolution of the drug lag problem. Stem cell medicine is positioned as a part of a national growth strategy that requires cooperation among the industry, government, healthcare field, and academia. It can be characterized as a "mesoscopic strategy," in that it aims to achieve high-level technological developments that would allow results from human-induced pluripotent stem cell and traditional stem cell research to contribute to regenerative medicine and drug development for intractable diseases, while attempting to strike a balance with commercialization and improved access of citizens to cutting-edge medical care.

This paper discusses exceptional circumstances under which patients outside of clinical trials are likely to receive innovative stem cell-based interventions. These circumstances involve: (1) stem cell interventions not initially amenable to a clinical trials approach; (2) expanded access to investigational stem cell products ("compassionate use"); and (3) off-label uses of FDA approved stem cell products. This paper proposes a new approach to regulating these exceptional cases.