The Oncology Center of Excellence of the U.S. Food and Drug Administration (FDA) recently announced a new pilot program to assist oncology health-care professionals in requesting access to unapproved therapies for patients with cancer. A new call center designated Project Facilitate will be a single point of contact where FDA oncology staff will help physicians through the process of submitting an Expanded Access request for an individual patient, including follow-up of patient outcomes.
Norman Sharpless, MD
“For decades, the FDA has been deeply committed to helping facilitate access to investigational medical products for patients with serious or immediately life-threatening diseases, while also protecting patients and helping them to be able to make informed decisions with their physicians. 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,” said Acting FDA Commissioner Norman Sharpless, MD.
More About Expanded Access
The FDA recently issued guidances encouraging companies to broaden their eligibility criteria to allow more patients with cancer to participate in clinical trials. However, in cases where patients do not fit the trial requirements or live too far from a trial site, health-care professionals can request permission from the FDA to treat a patient with an investigational medical product through Expanded Access.
Expanded Access is a pathway for a patient with an immediately life-threatening or serious disease or condition to gain access to an investigational medical product for treatment outside of clinical trials when there are no comparable or satisfactory alternative therapies available. Because investigational medical products have not yet been approved or cleared by the FDA—and have therefore not been found to be safe and effective for their specific use—part of the FDA’s role in granting Expanded Access requests is helping to weigh whether the potential benefit of the investigational treatment justifies the potential risks.
To make a request, a patient’s physician contacts the pharmaceutical company to ask for its agreement that it will provide the medical product. The company has the right to approve or reject the physician’s request. The FDA authorizes the vast majority of these requests. This process can be perceived as complex to navigate, particularly for oncologists who do not have experience working with clinical trials or these types of requests.
“Ultimately, a patient cannot submit an application for an investigational medical product; only a qualified physician is able to officially make the request. 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,” said
Richard Pazdur, MD
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. “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 acting as a facilitator for the process.”
The new pilot call center will enhance the FDA’s efforts to gather data on the Expanded Access program to help improve the process for physicians and patients. The 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 the 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 health-care professionals. In addition, the data could assist in encouraging sponsors to open clinical trials to study drugs for additional indications.
For more information, contact OncProjectFacilitate@fda.hhs.gov. ■