The Food and Drug Administration revealed its pilot program, designed to assist oncologists and patients requesting access to unapproved therapies in the cancer space.
The Food and Drug Administration (FDA) revealed its pilot program, designed to assist oncologists and patients requesting access to unapproved therapies in the cancer space.
The two-pronged program is called Project Facilitate, run by the FDA Oncology Center of Excellence, and Expanded Access Navigator, which is operated by the Reagan-Udall Foundation for the FDA.
“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,” acting FDA commissioner Dr. Ned Sharpless said in a press release. “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 (the) Expanded Access (program) 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.”
At the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting, Dr. Richard Pazdur, director of the Oncology Center of Excellence, discussed the expanded access or “compassionate use” programs — which make investigational drugs, biologics and medical devices available to patients who have exhausted other options for treatment and do not qualify for clinical trials that would provide these agents.
He noted that the joint program would streamline the process of gaining access to investigational agents while helping the FDA obtain information to improve the EA process and better understand investigational agents.
“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. Today’s launch of 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.”
Many Expanded Access requests come from individual patients who are in extreme need and willing to try a therapy that has not been proven or may not have been cleared for marketing by the FDA. The agency, which must authorize requests for Expanded Access, receives about 1,000 requests per year for participation in these programs. Depending on the urgency, the FDA approvals can be done over the phone, although follow-up is required.
Now, The Reagan-Udall Foundation will provide a searchable database that physicians and patients can use to establish whether any clinical trials are available for treatment with the investigational agents. The database will also provide information about Expanded Access programs, thereby offering physicians and patients a streamlined information search, according to Ellen V. Sigal, chair of the Reagan-Udall Foundation.
After visiting the foundation’s website, physicians would then turn to Project Facilitate, which is tasked with ushering physicians through the process of reaching out to drug companies for Expanded Access. FDA officials said this process would be more coordinated than in the past, as their current focus is broadening access and then following up with physicians about outcomes.
Expanded Access can only happen if a drug company agrees to make a drug available outside of a clinical trial. Requests are sometimes turned down because only limited supplies of investigational drugs are available, and the FDA cannot require a company to make its drugs available through Expanded Access.
“For the first time, those who need quick access to drug availability and expanded access options will find it in 1 place without having to visit site by site or sift through thousands of studies that don’t [serve] their needs,” Sigal said.
This article was adapted from an article that originally appeared on OncLive, as “FDA Streamlines Expanded Access for Oncology Agents.”