Looking Forward, Looking Back

At the Food and Drug Administration (FDA), I had a wonderful opportunity to spend nearly 20 years working in various capacities: in drug development science, regulatory oversight, and clinical pharmacology. Earlier this summer, I had the privilege to meet with the Personalized Medicine Coalition’s public policy committee to discuss the state of personalized medicine just after my departure from the FDA and before I began my new position leading the pharmacometrics and systems pharmacology initiative in the interdisciplinary Institute of Therapeutic Innovation at the University of Florida’s Research and Academic Center in Lake Nona (Orlando).  Personalized medicine will continue to be a passion for me in my new position.

The timing of my talk was serendipitous:  Dr. Steven Spielberg was just named as the new Deputy Commissioner for Medical Products and Tobacco at FDA. His perspective is strongly rooted in understanding disease at the molecular level, having served as director of the Center for Personalized Medicine and Therapeutic Innovation at Children’s Mercy Hospital in Kansas City, Missouri. And the FDA had just announced the draft guidance document for personalized medicine and companion diagnostics. This guidance should be seen as part of a mosaic of guidance documents from the FDA that will together guide the regulation of personalized medicine products. The Personalized Medicine Coalition has begun putting the mosaic together by listing the relevant guidance documents as a reference tool on its website.

I had a chance to reflect on the new direction FDA has taken over the past several years to embrace what we’re seeing in research and scientific discovery. Like the industrial revolution, the genomic revolution could take 60 years to fully unfold, but in the last ten years, the field and the FDA have made great progress. Many of these changes are leading researchers to re-evaluate how we focus research efforts, assess new therapeutics, and apply our shared learning through the R&D continuum.  As we’ve heard from the industry, researchers are stratifying medicines earlier in the R&D process and about 50% of new drugs have a biomarker that can potentially lead to a personalized approach. While many challenges remain, I am encouraged by the four-fold increase in Investigational New Drugs (INDs) and New Drug Applications (NDAs) that include genomic data since 2008.

To address this growth in genomic submissions, the FDA has new employees with expertise in genomics dedicated to review of these products and efforts to coordinate personalized medicine reviews will continue. I see a new era of personalized medicine not just in oncology but in cardiovascular disease, infectious disease, and neurodegenerative diseases, among others and the FDA is working to be ready.

I called on all members of the Personalized Medicine Coalition to find new ways to enable collaboration and to export the knowledge they are fostering at their companies and organizations to benefits for public health. By doing so, our combined efforts can lead to dramatic advances in improving therapeutics and healthcare. We are only just beginning to understand how new networks and powerful tools for sharing best practices can help us solve some of our most complex disease and drug response questions.