The FDA has unveiled what it describes as a pilot “concierge service” for oncologists and patients seeking information and assistance with expanded access (EA) to investigational therapies. The two-pronged program is called Project Facilitate, run by the FDA Oncology Center of Excellence, and EA Navigator, which is operated by the Reagan-Udall Foundation for the FDA.
Expanded access or “compassionate use” programs 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. Richard Pazdur, MD, director of the Oncology Center of Excellence, said 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.
“At the current time, the FDA doesn’t know the number of patients requesting expanded access,” Pazdur said in a presentation at the 2019 American Society of Clinical Oncology Annual Meeting.1 “Many times, the physician will go to the [drug] sponsor and the FDA is not aware if the sponsor denies these requests. We also do not know the reasons for drug sponsors’ denying these requests.”
This pilot program is exclusively for improving EA for oncologics, Pazdur said. EA has been around, at least informally, since the 1960s, and was instrumental in efforts to broadly expand access for experimental treatments during the HIV/AIDs epidemic. Many EA requests, however, 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 EA, receives about 1000 requests per year for participation in these programs.2 Depending on the urgency, FDA approvals can be done over the phone, although follow-up is required.
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 EA programs, thereby offering physicians and patients a streamlined information search, according to Ellen V. Sigal, PhD, chair of the Reagan-Udall Foundation.
“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,” she said.
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 EA. 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.
“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 [facilitate] the process,” according to a statement from the agency.3 Pazdur said the agency hopes the initiative can resolve inequities in access due to the lack of information about EA. He said the FDA is working to change a perception among physicians that the approvals process is burdensome.
Pazdur said information about outcomes gathered during the process may be shared with trial sponsors. “Frequently we are unaware of patient outcomes such as benefits from the proposed therapy and adverse events associated with that therapy,” he said. Physicians are required by law to provide that information but often they don’t, he added.
Reasons why drug sponsors turn down EA requests likely also will be shared, Pazdur said. “I’m sure we’ll publish it and make it publicly available in some fashion. We haven’t really worked that aspect out.”
EA 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 EA. Drug companies sometimes are concerned that EA may compromise their clinical trials. For example, there is a concern that adverse events (AEs) among unenrolled patients may lead to holds being placed on clinical trials.
However, a review of EA requests over a 10-year period demonstrated that only 2 drug development programs were ever placed on clinical hold due to AEs in patients receiving EA, and these were temporary.
Under the 21st Century Cures Act, companies were required to make information about their EA programs public. The Reagan-Udall Foundation component of this EA pilot is partly an attempt to broadly disseminate that information, Sigal said. “So, this is a necessary tool and urgent for patients and physicians.”