The FDA/CDER recently stated that “Most compounds ultimately do not make it to market due to a combination of scientific technical and regulatory challenges” (D’Mello et al., 2017). How does one navigate the way forward in a regulatory landscape so fraught with potential pitfalls? This discussion will focus on the importance of a resilient global regulatory strategy and effective communication with regulators, specifically regarding novel technologies of interest as new regulatory paradigms to assess drug safety. With the advancement of technologies such as induced pluripotent stem cells (iPSCs) regulators are highlighting concepts such as “Clinical trials in a dish” whereby iPSCs from peripheral blood can be reprogrammed into large numbers of various cell types including neurons, hepatocytes or cardiomyocytes.
In vitro clinical trials with specific cell or tissue specimens enable the assessment of how oncology treatment may react with particular types of cancer or other diseases, importantly provide the ability to predict how individual patients will respond in advance of therapy. Other examples will be discussed including the ability to identify patients who will benefit and those that may suffer harm from particular therapies with off target cardiotoxicity will significantly contribute to the safety assessment of new therapies in early phases of drug development. The potential for iPSCs and “clinical trials in a dish” to greatly improve safety outcome in patients is an exciting aspect to a new horizon in precision medicine that is very close to implementation and underlines the necessity of an up-to-date and resilient regulatory strategy.
Chairperson: Katy King, Principal Regulatory Affairs Consultant & Managing Director, Biotech Regulatory Solutions