Richard PazdurDirector, Oncology Center of Excellence, FDA
Acting director, Office of Hematology and Oncology Products
The FDA Oncology Center of Excellence announced a pilot program to help physicians get access to unapproved therapies for patients with cancer.
The program, called Project Facilitate, will include a call center that will serve as a point of contact with the agency. Through the call center, FDA oncology staff members will guide physicians treating patients with cancer through submission of Expanded Access requests for their patients.
Project Facilitate will also seek to provide follow-up on patient outcomes.
“This is a concierge service to support the patient’s medical team throughout the process, from providing information that will help the oncologist complete the Form FDA 3926 to following up on the status of the patient,” said Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products. “It doesn’t change FDA’s existing Expanded Access process in any way, but it should make it easier for oncologists to gather the information they need to submit an Expanded Access request. It’s often faster to speak to a knowledgeable person on the phone rather than searching through a lot of information on websites.”
Pazdur spoke with Paul Goldberg, editor and publisher of The Cancer Letter.
How is Project Facilitate different from FDA’s other Expanded Access ventures?
The Project Facilitate call center serves as a single point of contact where FDA oncology staff will assist oncologists and the healthcare team through the steps to submit an Expanded Access request for an individual cancer patient.
This is a concierge service to support the patient’s medical team throughout the process, from providing information that will help the oncologist complete the Form FDA 3926 to following up on the status of the patient. It doesn’t change FDA’s existing Expanded Access process in any way, but it should make it easier for oncologists to gather the information they need to submit an Expanded Access request. It’s often faster to speak to a knowledgeable person on the phone rather than searching through a lot of information on websites.
That said, because we want to make sure patients and their oncologists continue to have robust sources of information, Project Facilitate is working in conjunction with the 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. Patients and physicians can look for treatment options, clinical trials, and company contact information on the Navigator. The Navigator offers information provided by companies about their Expanded Access policies and includes any Expanded Access programs listed on ClinicalTrials.gov.
Companies now are required by the 21st Century Cures Act to publicly list their Expanded Access policies, if they have a drug intended to treat a serious or life-threatening disease or condition. The Navigator website helps them comply with that requirement.
Once the oncologist and the patient have identified the investigational therapy they want to try, the oncologist, nurse, pharmacist, or other member of the patient’s healthcare team can contact Project Facilitate for assistance in completing the Expanded Access request.
Why is Project Facilitate needed?
Before Project Facilitate, the Expanded Access requests for cancer patients arrived at multiple places within the FDA and were forwarded separately to FDA oncology or hematology divisions. Sometimes these requests were delayed while being sent from one place to another. I have long felt that the Expanded Access process would be so much easier for everyone if oncologists could simply call one of our oncology nurses or pharmacists for assistance.
In addition, we were seeing that most of the oncology Expanded Access requests were coming from patients and oncologists at the larger academic centers. But the patients who don’t live near these centers and who may not be able to travel to take part in a clinical trial could also benefit from access to investigational therapies that may be available through Expanded Access.
For oncologists who don’t have experience working with Expanded Access, the process can appear complex and burdensome. However, the FDA allows the vast majority of Expanded Access requests to proceed, and the agency streamlined the application form a few years ago.
So, we would encourage community oncologists to consider Expanded Access for cancer patients who have exhausted all other available therapies.
Is it only for oncology?
Project Facilitate is only for oncology Expanded Access requests at this time. Right now, Project Facilitate is a pilot program and is staffed by a team leader and a rotation of project managers who cover shifts on a volunteer basis within the Oncology Center of Excellence, but as we get a better idea of the volume of calls, we will establish some permanent positions.
Do you expect there to be an increase in the number of requests due to Project Facilitate?
We expect there will be an increase, at least initially, although possibly longer term as well. As I mentioned earlier, there could be cancer patients who would benefit from Expanded Access but were unaware of it or their oncologists thought it would be too difficult a process. While we want more patients to take part in clinical trials, and we want clinical trials that don’t exclude patients unnecessarily, Expanded Access does provide a way that patients can get access to an investigational drug if they don’t qualify for or can’t get to a clinical trial.
Using the Project Facilitate call center, how long would it take for a physician to get a formal response from the FDA?
Given the urgent need in most Expanded Access situations, the FDA has an excellent track record of responding quickly to these requests: emergency requests for individual patients are usually granted immediately by phone or within hours; non-emergency requests are generally processed within a few days. We expect the call center to be a valuable resource to help navigate healthcare providers through the process more efficiently.
How does all of this work in the Right-to-Try world?
Right-to-Try and Expanded Access are mutually exclusive programs. The main differences between these programs are, first, that under Right to Try, the drug has to have completed a phase I trial. That’s not the case with Expanded Access. Second, under Right to Try, the patient doesn’t need permission from the FDA and the FDA doesn’t review these requests.
Do you have buy-in from industry?
We held a workshop with the Reagan-Udall Foundation on May 16 at the FDA. Patient advocates and representatives from several companies attended and took part in the discussion of Project Facilitate. Generally, the comments we received were positive and constructive regarding FDA’s efforts to streamline Expanded Access. However, apparently some pharmaceutical companies require the healthcare facility to sign a liability contract in order to supply the drug, and negotiating this contract can delay Expanded Access requests. This is not an FDA requirement.
Does FDA need to estimate the amount of drug that exists to make it available?
No, the FDA doesn’t supply the drugs to the patients. The patient’s physician approaches the pharmaceutical company to ask for its agreement that it will provide the drug being sought. The company has the right to approve or disapprove the physician’s request. If the company agrees to the physician’s request, the physician can then apply to the FDA for authorization to proceed.
How would Project Facilitate collect information on denials of access by companies?
Project Facilitate can follow up on individual requests and gather information, such as how many patients received the investigational medical product and if not, the reason it was denied.
How would Project Facilitate counteract social media campaign and political pressure that are often brought to bear in such cases?
Our hope is that Project Facilitate will make it easier for oncology professionals to get accurate information they need about Expanded Access so that they can assist their patients—not just those with large Twitter or Facebook followings, but any patient, anywhere in the U.S., who has a serious or life-threatening cancer and who does not have other treatment options and is unable to access products through a clinical trial. With information about available products and the FDA Expanded Access process, patients can make an informed decision with their physician regarding their treatment options.
As healthcare professionals ourselves at the Oncology Center of Excellence, we would rather help seriously ill patients get the best care they can get without them having to launch social media campaigns.
Is there anything we’ve missed?
RP: The Project Facilitate phone number is 240-402-0004 and the email address is OncProjectFacilitate@fda.hhs.gov. We have been getting inquiries from other countries since Project Facilitate launched on June 3, so I should add that this program is only available for U.S. oncologists and their patients.