FDA Report Outlines Approach to Personalized Medicine

Miriam E. Tucker

October 29, 2013

In This Article

SILVER SPRING, Maryland — In a detailed new report, the US Food and Drug Administration (FDA) has outlined its approach for regulating new personalized therapies and devices.

The report, Paving the Way for Personalized Medicine: FDA's Role in a New Era of Medical Product Development, outlines the fundamental ways in which the FDA has modified its traditional approaches to drug and device regulation in the new era of products that are tailored toward specific patient subtypes, rather than broad diagnostic groups.

"We're very, very excited about this report because I think it captures the broad context of what's happening in science and medicine today and the role of the FDA as we enter the era of personalized medicine, and for us, personalized medical product development," FDA commissioner Margaret A. Hamburg, MD, said in a press briefing held at the FDA headquarters Monday.

Dr. Hamburg noted that the European Union's definition of personalized medicine, "Providing the right treatment to the right patient, at the right dose at the right time," has always been medicine's goal.

"Now, with the advance of science and technology and the understanding of both the underlying mechanisms and the human response to disease, we have so many more opportunities to target therapies in exciting ways and really improve the care that we can offer and the effectiveness of treatments," she said

Starting in 1998 with the approval of trastuzumab (Herceptin, Genentech) for the treatment of HER2-positive breast cancers, the FDA has approved more than 100 drugs that contain specific information about biomarkers in the labeling. Sometimes that information is added postlicensure.

More recently, the FDA has approved 4 different cancer drugs for use in patients with tumors that have specific genetic characteristics that are identified by companion test kits, a new regulatory scenario that has prompted collaboration of multiple branches within the FDA's Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and Center for Devices and Radiological Health (CDRH), along with the National Center for Toxicological Research, which is located in Arkansas.

Two recent notable FDA approvals have also demonstrated the extent to which science and technology are advancing personalized medicine and influencing the way the agency does business. On January 31, 2012, the FDA approved ivacaftor (Kalydeco, Vertex Pharmaceuticals), the first-ever drug to address the underlying cause of cystic fibrosis among patients with a specific gene mutation. The drug was approved in just 3 months, half the normal time for FDA "priority review," because it was considered to offer such a major advance and the submission was well-prepared.

In addition, earlier this year, the FDA granted emergency use provisions to physicians at the University of Michigan in Ann Arbor and Akron Children's Hospital in Ohio to create a bioresorbable airway splint constructed with a 3-dimensional printer to treat an infant with a life-threatening condition that led to airway collapse.

"We're really trying to harness advancements in science and technology to evaluate new tools so that we can help speed innovation," Dr. Hamburg said at the briefing.


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