FDA Report Outlines Approach to Personalized Medicine

Miriam E. Tucker

October 29, 2013

In This Article

Will Personalized Medicine Shift Paradigms?

Rick Pazdur, MD, director of the FDA's Office of Oncology and Hematology Products, believes personalized medicine will dramatically change the way drugs are discovered, tested, and regulated. "What we're seeing now is a revolution.... We're redefining the diseases that we treat," he noted.

In oncology, where drug toxicities are tolerated to a greater extent than in other areas of medicine, the determination of efficacy has been a major issue. When toxicity is high and efficacy relatively low, large randomized trials are typically needed to see long-term effects on both survival and progression-free survival.

Now, however, targeted therapies have produced such unprecedented efficacy that some oncologists have begun questioning the need for randomized clinical trials. If, for example, a targeted therapy has demonstrated a 70% to 80% response rate, is it ethical to pit it against a conventional comparator with a response rate of just 10% to 20%?

"Is there a reason to do a randomized study? This is something we have to question. Obviously these studies have been done, but looking back, I question whether they should have been done," Dr. Pazdur said.

In another potential shift, drug trials directed at small groups of patients with specific mutations may not allow for the gathering of sufficient safety data in premarketing trials, requiring a shift to the postmarketing setting instead. "I think this is a good thing, as it represents the real world," Dr. Pazdur commented.

He also predicted an eventual end to the current practice by which each targeted drug is marketed along with a companion test kit. "As we develop more and more [targeted] drugs in a particular setting...it makes no sense from the long-term perspective for each drug to have its own individual companion diagnostic, because that's not the way medicine is practiced."

Rather, he said, each patient will have a biopsy and a pathology lab will run a standardized diagnostic on that. "I think our current system is going to need tinkering with."

Dr. Pazdur lamented that he is nearing retirement age. "I will not be able to participate in what I think is the most fascinating part of oncology in the next 20 or 30 years. When I take a look at the eyes of [young FDA reviewers], I envy them, because they are going to have the excitement of participating in this revolution."

All of the speakers are FDA employees and have disclosed no relevant financial relationships.

Paving the Way for Personalized Medicine: FDA's Role in a New Era of Medical Product Development FDA, Full text

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