Personalized Medicine: The Future is Now

By Margaret A. Hamburg, M.D.

Margaret Hamburg, M.D.The difference between science and science fiction is a line that seems ever harder to distinguish, thanks in part to a host of astonishing advances in medical science that are helping to create a new age of promise and possibility for patients.

Today cancer drugs are increasingly twinned with a diagnostic device that can determine whether a patient will respond to the drug based on their tumor’s genetic characteristics; medical imaging can be used to identify the best implantable device to treat a specific patient with clogged coronary arteries; and progress in regenerative medicine and stem cell therapy using a patient’s own cells could lead to the replacement or regeneration of their missing or damaged tissues. Given these trends, the future of medicine is rapidly approaching the promising level of care and cure once imagined by Hollywood in futuristic dramas like Star Trek.

But these examples are not science fiction. They are very real achievements that demonstrate the era of “personalized medicine” where advances in the science of drug development, the study of genes and their functions, the availability of increasingly powerful computers and other technologies, combined with our greater understanding of the complexity of disease, makes it possible to tailor treatments to the needs of an individual patient. We now know that patients with similar symptoms may have different diseases with different causes. Individual patients who may appear to have the same disease may respond differently (or not at all) to treatments of that disease.

FDA has been playing a critical role in the growth of this new era for a number of years. Even before I became FDA Commissioner the agency was creating the organizational infrastructure and putting in place the regulatory processes and policies needed to meet the challenges of regulating these complex products and coordinating their review and oversight. It has been my pleasure to serve at FDA during this next exciting period and to help ensure that the agency continues to prioritize this evolution by anticipating, responding to, and encouraging scientific advancements.

I am very pleased to be able to present a new report by FDA as part of our ongoing efforts in this field. Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development describes many of the exciting developments and looming advances in personalized medicine, lays out the historical progress in this field, and examines FDA’s regulatory role: from ensuring the availability of safe and effective diagnostic devices, to addressing the challenges of aligning a drug with a diagnostic device, to post-market surveillance.

Outside collaboration and information sharing is essential for this field to flourish. On Tuesday, the American Association for Cancer Research and AdvaMedDX held a fruitful daylong conversation on personalized medicine to treat cancer. I was one of the speakers, participating in a conversation with Dr. Francis Collins, the head of the National Institutes of Health. Our discussion focused in part on current status of drug and diagnostic co-development and the challenges and potential of whole genome sequencing, where data can be collected on a patient’s entire genetic makeup at a reasonable cost in a reasonable amount of time.

FDA is committed to fostering these cooperative efforts, as it will require the full force of government, private industry, academia and other concerned stakeholders to maximize our efforts and fully realize the promise of personalized medicine. Our new report outlines that commitment, and helps chart the way forward so that more people can live long and prosper.

Margaret A. Hamburg is the Commissioner of the Food and Drug Administration

FDA Voice Interviews Stephen Spielberg, MD, PhD

FDA Voice: Dr. Spielberg, thank you for agreeing to let us interview you for FDA Voice.  Can you tell us about your position at FDA and what that entails?

Dr. Spielberg: I am the Deputy Commissioner for Medical Products and Tobacco. In creating this new position, Commissioner Hamburg envisioned that it would “provide high-level coordination and leadership across the Centers for drug, biologics, medical devices, and tobacco products, and support, coordinate and advocate for the work and needs of the Centers.”  In my short time at FDA, I have been working with the Center Directors and their staff to understand shared challenges and opportunities to advance regulatory science and practice across all “human products”.  Together, we have begun to define areas of mutual interest and synergy where we can work together and with external partners in the public and private sectors to bring the best of science to bear on our public health responsibilities, to advance managerial and operations support to optimize our core tasks, and to assure in everything we do that FDA is at the cutting edge of promoting and protecting the public health.

 Stephen Spielberg, MD, PhDFDA Voice: Why did you join FDA?

Dr. Spielberg: Throughout my career, I have been involved with FDA.  In fact, my MD-PhD training at the University of Chicago, particularly in the Department of Pharmacology, was in the context of former UC faculty (Drs. E.M.K. Geiling and Francis Kelsey) who were intimately involved with creation of FDA by their work on elixir of sulfanilamide (Federal Food, Drug, and Cosmetic Act of 1938) and thalidomide (Drug Amendments of 1962). So, in retrospect, I suppose I “grew up” with knowledge and appreciation of FDA.  Over the years, I have served as a member of the Pediatric Subcommittee, the Science Board, rapporteur for ICH E-11, and helped get BPCA and PREA through Congress. At this stage of my career, it is a true honor for me to be able to serve the Agency at a critical time in biomedical science and therapeutics.

FDA Voice: What did you do before joining FDA?

Dr. Spielberg: I have had a 35 year career as a pediatrician and clinical pharmacologist, including in academic settings in the US and Canada, as well as in the pharmaceutical industry. My research career has focused on human pharmacogenetics and pharmacogenomics, mechanisms of adverse drug reactions, and pediatric/development pharmacology and pediatric clinical trials. In the years prior to coming to FDA, I was Dean of Dartmouth Medical School, and subsequently headed a new personalized medicine program at Children’s Mercy Hospital in Kansas City, MO, as well as working with the Institute for Pediatric Innovation, a non-profit organization focused on advancing therapeutics for children.

FDA Voice:  What is the favorite part of your job here at FDA?

Dr. Spielberg: This is a remarkable time to be at FDA. Biomedical science is advancing at an incredible rate. We are now beginning to see the impact of genomic and other science in defining the causes of disease, and in the discovery and development of new therapies. Medicine now is at a place that I could barely have imagined when I began my career, but we have the age old challenges of how to skillfully and wisely use the knowledge we have at any time to advance the health of individual patients and of all patients we serve. At such a time, what could be more challenging and satisfying than being here at FDA and having the opportunity to advance the promotion and protection of the public health.

FDA Voice:  If you could tell the American public one thing that you think they don’t know about what your office does to directly benefit them, what would it be?

Dr. Spielberg: Every day, I am impressed by the outstanding, dedicated, hard working people here at FDA. Their focus on our public health mission is remarkable, and it is an honor to work with them.

FDA Voice: Dr. Spielberg, thank you so much for your time!

Stephen Spielberg, MD, PhD, is Deputy Commissioner for Medical Products and Tobacco