Paolo Bianco and colleague Douglas Sipp wrote a very
provocative and interesting piece recently published in the journalNature (http://www.nature.com/news/regulation-sell-help-not-hope-1.15409)discussing
a movement to permit stem cell medicines, among others, to be sold in the
market without the requirement to show much safety and efficacy data permitting
the market to determine safety and effectiveness of compounds. Here, patients
would basically pay to obtain products and also be research subjects. Certain
powerful groups are calling for the deregulation of clinical medicine as a
business model to bring innovative products to the marketplace. But before I
begin explaining what Bianco and Sipp discuss, we need to cover the current
system of regulatory oversight of medical products.
The current standard for market approval of drugs,
devices, biologics and other products is known as clinical translation. The
clinical translation process involves moving research from preclinical phases
to clinical phases. Preclinical research aims to provide evidence of
proof-of-principle and product safety through in vitro (e.g., cell-based assays) and in vivo (e.g., cell transplantation in disease animal model)
experiments. The idea here is that work using animals will help to ensure that
outcomes in humans will be clinically relevant and safe. However animal models
are not always the best indicators of effectiveness of interventions in humans
despite how genetically similar they are to humans. Demonstration of safety and
efficacy in preclinical research is required to persuade regulatory authorities
like the US Food and Drug Administration (FDA) to permit approval for
researchers to enter phase 1 clinical trials.
The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.