New Regulatory Pathways for Stem Cell-Based Therapies: Comparison and Critique of Potential Models

Abstract

The regulation of stem cell-based therapies is challenging in many respects. Growing interest in these innovative therapies has led some to question FDA’s regulation of them, arguing that it is too restrictive and impedes both innovation by providers and access by patients. At the same time, others urge rigorous standards and enforcement, given significant risks to patients of physical harm or exploitation. This chapter examines attempts to craft specific provisions for additional flexibility in regulating cell and tissue therapies. After noting current US proposals and recent regulatory reforms in Japan, it focuses on the discussion and consultations regarding Australia’s exemption for autologous cell and tissue therapies and the hospital exemption in Europe’s regulation for advanced therapies. The Australian experience illustrates the potential for unintended negative consequences of an unduly broad exemption. One very relevant lesson from the hospital exemption is that adding flexibility to the regulatory framework involves a trade-off between limited short-term access to new treatments and broad longer-term access. Another is the importance of considering how a proposed new exemption would relate to existing forms of flexibility and whether greater gains would come from improving those existing mechanisms.

An earlier version of this chapter was previously published by von Tigerstrom B (2015) “Revising the Regulation of Stem Cell-Based Therapies: Critical Assessment of Potential Models”. Food and Drug Law Journal 70(2):315–337. Material from that article is reprinted with permission from the Food and Drug Law Institute. This work was funded by the Stem Cell Network (Public Policy Core Project Grant “From Banking to International Governance: Fostering Innovation in Stem Cell Research”) and by the Government of Canada through Genome Canada and the Ontario Genomics Institute (OGI-064). The author gratefully acknowledges research assistance by University of Saskatchewan law students Jordan Richards (J.D. 2014), Emily Harris (J.D. 2015), Kate Rattray (J.D. 2015) and Kimberley Osemlak (J.D. 2017) and helpful suggestions from anonymous reviewers.