The challenge of regulating nanomedicine: Key issues

Nano-enabled products and their applications have continued to evolve in recent years. Indeed, nanotechnologies have been integrated into many academic and industrial sectors, and have spurred new directions in terms of research, patents, commercial opportunities and technology transfer. However, many nanopatents are of dubious scope and breadth yet continue to be granted by patent offices internationally. Underpinning many issues in the regulation and patenting of such technologies is the difficulty that has been encountered in agreeing a definition, and this continues to pose a significant problem to regulators, policy-makers, drug companies, ethicists and legal professionals. This chapter aims to provide an overview of the regulatory and scientific landscape for nanotechnologies applied to medicine. The first US Food and Drug Administration (FDA)-approved nanodrug, Doxil^®, is discussed as a paradigm for some of the sentient points. The chapter also provides information on the utility of physiologically based pharmacokinetic modeling for nano-enabled regulatory submissions, and a view of important gaps in knowledge relating to the mechanistic basis for modeling in the context of efficacy and safety of novel materials. There are potentially serious and inhibitory consequences if nanodrugs are overregulated, and a balanced approach is required, at least on a case-by-case basis, that addresses the needs of commercialization against mitigation of inadvertent harm to patients or the environment. Obviously, not every nanotherapeutic or nano-enabled product needs to be regulated. However, more is clearly needed from regulatory agencies like the FDA and European Medicines Agency than a stream of guidance documents that are in draft format, position papers that lack any legal implication, presentations that fail to identify key regulatory issues and policy papers that are often short on specifics. There is a very real need for regulatory guidelines that follow a science-based approach that are responsive to the associated shifts in knowledge and risks.