Chicago, IL—At ASCO 2019, the FDA announced an oncology-specific pilot program for physicians and patients who are seeking access to investigational therapies. The expanded access pilot program is a concierge service, said Richard Pazdur, MD, Director, FDA’s Oncology Center of Excellence, who spoke at a press conference where the new program—Project Facilitate—was announced.
Project Facilitate is designed as a 2-pronged program administered by the FDA’s Oncology Center of Excellence and includes an expanded access navigator provided by the Reagan-Udall Foundation for the FDA.
Expanded access, also known as “compassionate use,” is designed to give patients who have exhausted all other treatment options and are not able to enroll in clinical trials access to investigational therapies. According to Dr Pazdur, Project Facilitate will streamline the process for patients and physicians to gain access to investigational agents. At the same time, it will enable the FDA to obtain information to improve the expanded access process and to gain more knowledge about investigational agents, including outcomes and adverse events in this patient population.
“At the current time, the FDA doesn’t know the number of patients requesting expanded access,” Dr Pazdur said. “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.”
Dr Pazdur said that the FDA receives approximately 1000 expanded access requests each year. Many of these requests come from patients who are in dire need of new treatment options, and for whom investigational therapies are a last resort. Under the new program, FDA approvals for expanded access can be processed over the telephone in urgent cases, although follow-up is required.
Through Project Facilitate, the Reagan-Udall Foundation will provide a searchable database that physicians and patients can use to identify clinical trials of investigational agents or new combinations. The database will also provide information about expanded access oncology programs that will expedite an information search, according to Ellen V. Sigal, PhD, Board Chair, Reagan-Udall Foundation, Washington, DC.
“For the first time, those who need quick access to drug availability and expanded access options will find it in one place, without having to visit site by site or sift through thousands of studies that don’t [serve] their needs,” Dr Sigal told listeners at the meeting.
The plan is to have physicians first visit the Reagan-Udall Foundation site and then go to Project Facilitate, which expedites the physicians’ process of applying for expanded access from drug companies. This process should be more streamlined than in the past, with the FDA focusing on broadening access and following up with physicians about outcomes.
Dr Pazdur said that the FDA’s goals are to reduce inequities that result from the lack of information about expanded access, as well as to create a process that is no longer burdensome to physicians.
“Through this pilot program, 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 acting as a facilitator for the process,” Dr Pazdur said in a statement.
The FDA plans to share outcomes and adverse events of patients who have received investigational oncology drugs through the new expanded access process with the pharmaceutical companies that provide the drug.
Expanded access depends on a drug company’s agreement to make an investigational therapy available outside of a clinical trial. The drug company may deny such requests because of limited drug supplies and other reasons that are beyond the control of the FDA, Dr Pazdur noted.