1. DiGiulio, Sarah

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SAN FRANCISCO-"We've seen kind of an explosion of requests-and I don't think anybody really expected that," Suparna B. Wedam, MD, Medical Officer at the U.S. Food and Drug Administration, said about the FDA's four expedited programs, which she discussed here at the Breast Cancer Symposium in her talk "FDA Drug Approvals: Getting Drugs to Patients Sooner." Those four programs include three designations (breakthrough therapy, priority review, and fast track designations) and one approval pathway (accelerated approval).

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In an interview after the talk, she summed up these key points and elaborated on what these approval programs mean for the future of oncology drugs.


1. You spent a lot of time during the talk on breakthrough therapy designation, which is the newest program of the four-what should oncologists know about the designation?

"With breakthrough therapy designation, you have to have clinical evidence that this [drug] is substantially better than a significant endpoint, and for fast track designation, non-clinical data can be used. Having said that, we do not accept non-clinical data usually for oncology-typically we require at least some clinical data. Especially in oncology, we feel that [non-clinical] data that comes from cell lines or animals does not always translate well into humans.


"And the other part is the level of senior level involvement. Once a drug has the breakthrough therapy designation, it really is an all-hands-on-board situation from the top down, where the senior manager is helping with the whole process-so, it's the intensity of involvement that differentiates it."


2. You mentioned a New York Times article in your talk with the headline "Speedy Drug Approvals Have Become the Rule, Not the Exception"-is that true for oncology drugs? Should these programs be the norm for oncology drugs?

"I think it is true. All of the molecular entities in the last two years that we've approved in hematology and oncology, every one of those drugs had taken advantage of at least one of the programs. And many have taken advantage of more than one of those four programs.


"Everything we deal with in oncology is a serious disease [so all of the drugs fit that requirement]. But still, we are approving drugs when we think they're safe-when we feel the benefit justifies the risk."


3. What are the risks of using these programs?

"There's caution that needs to be said. We don't have the same number of patients treated, and we don't always know that the complete picture that's not known when we approve a drug. There's a lot more that we learn post-marketing. And in some of the accelerated approvals we have even a smaller safety base. We need to be very vigilant and follow those safety signals after [the approval] to make sure that this is indeed a safe drug and if there are any safety signals, we need to deal with those.


"And we need to be engaging patients in this process to make sure they understand when a drug was approved through an expedited program. It's important to explain that the drug was approved in this way and the safety data might not be as complete-and then it's up to the patient and the physician to make that decision. We [FDA] always feel that it is safe enough to approve the drug-and that's why it's out there-but that discussion still needs to happen in the office between the patient and the physician."


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