As part of a pilot program intended to aid oncology health care professionals in requesting Expanded Access, which makes investigational treatments available to certain patients with cancer, the FDA has announced the creation of Project Facilitate, a call center to aid physicians in the process of submitting Expanded Access requests for individual patients.
"Through this pilot program, experienced FDA oncology staff will be available to support physicians and other health care professionals with their questions, assist in filling out the appropriate paperwork, and [act] as [facilitators] for the process," commented Richard Pazdur, MD, Director of the FDA's Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.
Expanded Access enables a physician to treat a patient who has a life-threatening or serious condition with an investigational medical product such as a drug, biologic, or medical device in a case where the patient does not fit requirements for a clinical trial or lives too far away to participate in it. For a patient to be granted expanded access to an investigational product, the FDA must assess whether the potential benefit of the product—which, as it has not yet been FDA approved, has not yet officially been deemed safe and effective—outweighs the potential risks. In order to make the request, the patient's physician needs to ask the pharmaceutical company whether it will provide the product, and the pharmaceutical company can either approve or deny the request.
Although the majority of Expanded Access requests are approved by the FDA, the process is often seen as complex. Prior to the new pilot program, Expanded Access requests involving patients with cancer were sent to one of multiple locations within the FDA and were then forwarded to either the oncology division or the hematology division. The pilot program's new central office for oncology Expanded Access requests will enable the FDA not only to follow up on individual requests but also to gather data regarding how many patients receive particular investigational products and why certain requests are denied. This data could be used to assess the effectiveness of the Expanded Access program and to encourage pharmaceutical companies and other sponsors to initiate clinical trials involving treatments for additional indications.
"The first option for patients who have exhausted available treatments is to enroll in a clinical trial, but when that is not an option, we support Expanded Access and are exploring ways to make it easier for patients, their families, and health care professionals to understand the process and how to access investigational therapies," commented Ned Sharpless, MD, Acting Commissioner of the FDA. "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 health care professionals, especially those not familiar with the Expanded Access program, the process may appear confusing or burdensome… we hope that this pilot program will simplify the process for oncologists and ultimately benefit patients."
For More Information
The Reagan-Udall Foundation for the FDA has also recently updated the Expanded Access Navigator, an internet resource for physicians and patients that posts information provided by medical product companies about their Expanded Access policies. The Expanded Access Navigator also now includes Expanded Access programs listed in clinicaltrials.gov.
Image credit: United States Food and Drug Administration