The pilot program will include Project Facilitate, a new call center run by the agency’s oncology staff to provide a single point of contact for doctors submitting requests to the program. Although the agency has streamlined its expanded-access program in recent years, the process still may seem confusing to doctors who have little experience with it, Pazdur said.
The FDA’s expanded access program, sometimes called “compassionate use,” is designed to help patients with immediate life-threatening or serious illnesses who don’t qualify for clinical trials and have no other treatment options. Typically, a doctor first asks a drug company to provide the experimental treatment. If the firm agrees, the physician submits a protocol to an institutional review board (IRB) — which makes sure the patient is properly informed about the treatment. The FDA then decides whether to approve the request — and almost always does.
Under the new plan, FDA staffers will help the doctor from the start on paperwork and finding an IRB and appropriate contacts in drug companies. If the pharmaceutical firm rejects the doctor’s request, the FDA will ask why. If it doesn’t, the FDA will ask the doctor whether the patient was helped and whether there were any side effects.
The FDA cannot require drug manufacturers to provide unapproved drugs to patients, and many companies are reluctant to do so because of the cost or limited supplies. Because of that, said Alison Bateman-House, a medical ethicist at the New York University School of Medicine, it isn’t clear whether the FDA plan will result in “any real difference” in the number of patients who receive unapproved therapies. Still, she praised the FDA effort as a “a well-intentioned initiative.”
Last year, Congress passed a “right to try” law allowing doctors and patients to bypass the FDA and IRBs in seeking unapproved medications from pharmaceutical companies. But the law does not require drug companies to agree to provide the therapies. Bateman-House said she believes only two patients have used the law to get treatments.
The FDA’s new pilot project was announced at the annual meeting of the American Society of Clinical Oncology in Chicago.