Jan 30 2014

FDA’s Comprehensive Status Report on Personalized Medicine

By Sheila R. Plant, Ph.D., R.A.C., Regulatory Scientist at Cato Research

FDA published a report, prepared by the Office of the Commissioner in the Office of Medical Products and Tobacco, in October 2013 entitled “Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development” with an enthusiastic forward by FDA Commissioner Margaret Hamburg, MD:   http://www.fda.gov/downloads/scienceresearch/specialtopics/personalizedmedicine/ucm372421.pdf

The report describes “the unique and special role and responsibility that FDA has in helping to usher in the medical products that are central to this larger effort (personalized medicine). The report describes the ways in which FDA has evolved its regulatory processes in response to – and in anticipation of – scientific developments that are critical for the development of personalized therapeutics and diagnostics. It describes in particular the ways in which FDA has worked to bridge developments in genomics and other relevant sciences to clinical practice by advancing the tools necessary for evaluating targeted therapeutics and bringing them to market more efficiently, collaborating in key research, defining and streamlining regulatory pathways and policies, and applying new knowledge in product reviews.”

The report describes how, over a period from 2002 to 2013, FDA has reorganized their infrastructure and built up the organization to support personalized medicine.  This includes a restructuring of the Commissioner’s Office into four directorates and a high level coordinating position, Deputy Commissioner for Medical Products and Tobacco, and office which is designed to manage submissions that do not fit into a single category (drug, device or biologic) and thus poises FDA to efficiently overcome regulatory challenges for products like drugs or biologics with companion in vitro diagnostic devices.  Other recent notable enhancements include CDER’s Genomics and Targeted Therapy Group, the Personalized Medicine Staff within CDRH’s Office of In Vitro Diagnostics and Radiological Health (OIR), and CBER’s Genomics Evaluation Team for Safety.  The report also contains a table which summarizes over 20 FDA guidances that relate to personalized medicine, a fantastic reference.  The means by which FDA is working to ensure availability of safe and effective diagnostics is addressed as are the issues surrounding product interdependency, product labeling, and post-market surveillance which are perceived to be even more important  in this era of personalized medicine.  Finally, the report describes the regulatory standards, research methods, and tools that FDA is actively working on to advance new clinical products and provide a modernized evaluation and approval process.  Very early examples of personalized medicine as well as more recent  drug/biologic/companion diagnostic co-development provide a sense of the successes and the platform for building more successes in personalized medicine in the near future.