FDA Pilot Program Aims to Simplify Access to Unapproved Cancer Drugs

Megan Brooks

June 03, 2019

The US Food and Drug Administration (FDA) has launched a pilot program to make it easier for oncologists to request access to unapproved therapies for their cancer patients who have exhausted all other options.

Dubbed Project Facilitate, the program features a single point of contact through a dedicated call center where FDA oncology staff will help oncologists through the process of submitting an expanded access request for an individual patient.

"The FDA has been working diligently to improve the expanded access framework, including development of an updated and more streamlined application form, but despite recent improvements, we understand that for many patients or healthcare professionals, especially those not familiar with the expanded access program, the process may appear confusing or burdensome," acting FDA Commissioner Norman "Ned" Sharpless, MD, said in a news release.

"Project Facilitate is part of our continued commitment to expanded access and we hope that this pilot program will simplify the process for oncologists, and ultimately benefit patients," he added.

The FDA has encouraged companies to broaden their eligibility criteria to allow more cancer patients to participate in clinical trials. However, in cases where patients do not meet trial requirements or live too far from a trial site, physicians can ask the FDA for permission to treat a patient with an investigational medical product through expanded access.

But the process of getting expanded access approval has been perceived as complex to navigate, particularly for oncologists who don't have experience working with clinical trials or these types of requests, the FDA acknowledges.

"The new Project Facilitate call center aims to help in making these requests as streamlined and efficient as possible for physicians who would like to request access to investigational therapies for their patients with cancer," Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products at the FDA's Center for Drug Evaluation and Research, said in the statement.

"Through this pilot program, experienced FDA oncology staff will be available to support physicians and other healthcare professionals with their questions, assist in filling out the appropriate paperwork, and act as a facilitator for the process."

Until now, expanded access requests for cancer patients were funneled through multiple places within the FDA and forwarded separately to FDA oncology or hematology divisions.

The new pilot program includes a central office for oncology requests so that the FDA can follow-up on individual requests and gather data, such as how many patients received investigational medical products and, if not, why the requests were denied. The FDA can use these data to determine how the process is benefiting patients and healthcare professionals. The data could also encourage sponsors to open clinical trials to study drugs for additional indications, the agency said.

The Project Facilitate phone number is 240-402-0004 and the email address is OncProjectFacilitate@fda.hhs. Healthcare professionals may call during regular business hours, 9 am to 5 pm, Eastern Standard Time, Monday through Friday.

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