Director of the FDA’s Oncology Center of Excellence Discusses Expanded Access, Accelerated Drug Approvals

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Dr. Clifford A. Hudis (CH): Welcome to this ASCO in Action podcast. This is ASCO's monthly podcast, series where we explore policy and practice issues that can impact oncologists, the entire cancer care delivery team, and the individuals we care for, people with cancer. My name is Clifford Hudis, and I'm the CEO of ASCO, as well as the host of the ASCO in Action podcast series. For today's podcast, I am delighted to have as my guest, Dr. Richard Pazdur, the Director of the Food and Drug Administration's Oncology Center of Excellence.

The OCE was established to expedite the review of novel cancer therapies and products by bringing together expertise from across the FDA. And we'll touch on this a little bit during our conversation. Dr. Pazdur, welcome and thank you for joining me today.

Dr. Richard Pazdur (RP): It's a pleasure Dr. Hudis.

CH: Thanks. So I want to kick off our discussion by diving right into a hot button issue, expanded access. Can you provide our listeners with some background on this, and explain what the FDA's expanded access program is, and why an oncologist might want to pursue expanded access for an individual patient?

RP: Of course. The FDA's expanded access program provides a way that patients with serious or life-threatening diseases or conditions such as cancer can try investigational medical products for treatment when no satisfactory therapies are available, and when there is no opportunity for the patient to enroll in a clinical trial. The process-- to make a request, the patient's physicians will approach the pharmaceutical company to ask for its agreement that the company will provide the medical product.

The company has the right to approve or disapprove the physician's request. Then the physician needs to send the request to the FDA. This process can be complex to navigate, particularly for oncologists or physicians who don't have experience working with the clinical trials or these types of requests.

FDA allows the vast majority of these requests to proceed. And the FDA has been working to improve the expanded access programs for a number of years, including the development of a more streamlined application process, a more streamlined form. But for many key health care professionals, especially those not familiar with the expanded access program, this process may appear confusing or somewhat burdensome.

CH: And so is this a segue to Project Facilitate, which you announced at our annual meeting a few weeks ago? Can you talk a little bit about that and, its practical implications?

RP: Yes. The Project Facilitate call center is a pilot program only for oncology that will serve a single point of contact. We have FDA oncology staff there, oncology nurses, oncology pharmacists who will assist the physician and their health care team throughout the process to submit and expanded access request for an individual cancer patient.

This is a concierge service to support the patient's medical team throughout the process. It ranges from the initiation of the FDA form 3926. The process will also provide information about IRBs, particularly central IRBs, and really will also follow up on the status of a given patient to determine if that patient has received any benefit from the therapy and if there were any adverse events that need to be reported to the FDA.

CH: So imagine that Project Facilitate works as hoped for. What's the thumbnail before and after experience? That is, how will things appear to be different to the physicians and to the patients?

RP: It should make the process easier for physicians to get information that they need to submit an expanded access request. As I said before, it's often somewhat complicated, especially for physicians don't have experience with either the drug or with the process. And it's obviously easier to talk to somebody over the phone to ask specific questions rather than just being directed to a website.

We're also working in conjunction with Reagan-Udall Foundation for the FDA, which started the expanded access navigator website to educate patients and health care professionals about the expanded access process. This navigator approach offers information provided by companies about their expanded access policy, and now inc...