Conditional Approval Pathways: The “Special” Case of Global Regenerative Medicine Regulation

B iomedical innovation and the societal benefits it catalyses is essential and challenging. Yet from reductionist viewpoint, the term innovation has been consistently misapplied to describe the process of translating scientific advancements from the laboratory to patient bedside. It is easy to forget that etymologically, innovation stems from Latin roots meaning to change or develop and introduce. Historically however, and perhaps detrimentally, a disproportionate quantum of resource has been directed toward research into fundamental laboratory science (changes or developments)—neglecting the regulatory frameworks guiding therapeutic development and clinical adoption (introduction). This has also lead to a discontinuity in efforts to better evaluate and rationalize regulatory frameworks, that are often dismissed as ‘‘someone else’s problem.’’ (Or as Douglas Adams would coin, ‘‘an SEP field.’’) The central thesis discussed herein is that the regulatory incentives presently afforded to developers of regenerative medicines—spanning the full ambit of cell therapy, gene therapy, and tissue engineering—may not be instantaneously proportional or rational. That is to say, when such accelerated pathways were designed and instituted, this was based on different levels of understanding—in terms of regenerative medicine technology platforms and accelerated regulatory approaches—than we have today, based on empirical experience. And as such, may not be optimal to support the long-term sustainability of the regenerative medicine industry nor maximize the transferability of regulatory advances in regenerative medicine to support other therapeutic platforms. We support regenerative medicine innovation and believe that regulators and basic scientists deserve substantial credit and gratitude for the advances made, accelerating novel therapeutics and devices to patients with unmet medical needs. The focus of our appraisal is simply: 1. What have we learnt from accelerated regulatory pathways in regenerative medicine? 2. As the regenerative medicine industry and underpinning technologies mature, how regulatory pathways be continuously developed to ensure their rationality and proportionality? 3. Is it feasible to apply the accelerated regulatory pathways ‘‘piloted’’ in regenerative medicine to other emerging technological platforms?