Project Facilitate helps physicians seeking access to unapproved therapies for cancer patients
The Food and Drug Administration Oncology Center of Excellence has announced a new pilot program to assist oncology healthcare professionals in requesting access to unapproved therapies for patients with cancer. FDA says the new call center, Project Facilitate, will be a single point of contact where FDA oncology staff will help physicians treating patients with cancer through the process to submit an Expanded Access request for an individual patient, including follow-up of patient outcomes.
“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 healthcare professionals to understand the process and how to access investigational therapies,” said Acting FDA Commissioner Ned Sharpless, in the statement. Sharpless added that the agency had made some improvements to the Expanded Access framework, including development of an updated and more streamlined application form, but says the process may still be confusing or burdensome. This latest program was designed to simplify the process even more.
The FDA says it recently issued guidances encouraging companies to broaden their eligibility criteria to allow more patients with cancer to participate in clinical trials. But in those cases where patients do not fit the trial requirements or live too far from a trial site, healthcare 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 (drug, biologic or medical device) for treatment outside of clinical trials when there are no comparable or satisfactory alternative therapy options available. Because investigational medical products have not yet been approved or cleared by the FDA and have therefore not been found safe and effective for their specific use, part of the FDA’s role in granting Expanded Access requests is helping weigh whether the potential benefit of the investigational treatment justifies the potential risks.
To make a request, the patient’s physician will contact the pharmaceutical company to ask for its agreement that it will provide the medical product. The company has the right to approve or disapprove the physician’s request. The FDA authorizes most of these requests. This process can be perceived as complex to navigate, particularly for oncologists who don’t 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, 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.
Prior to the pilot program launch, FDA said Expanded Access requests for patients with cancer arrived at multiple places within the FDA and were forwarded separately to FDA oncology or hematology divisions. The 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 the investigational medical products and if not, why the requests were denied. The FDA can use this data to determine how the process is benefiting patients and healthcare professionals. In addition, the data could assist in encouraging sponsors to open clinical trials to study drugs for additional indications.
