Patients and advocacy groups told a Food and Drug Administration (FDA) advisory panel on Thursday that there is not enough access to potentially life-saving cancer therapies that are still considered experimental.
The FDA asked its Oncologic Drugs Advisory Committee to consider whether changes are needed to the agency’s policy on giving severely ill patients special access to cancer therapies that are not yet approved.
The panel will not make suggestions until its next meeting in March, said committee chairperson Stacy Nerenstone of Hartford Hospital’s Helen & Harry Gray Cancer Center.
Currently, a patient who is not eligible for a clinical trial of an experimental cancer prescription drugs can seek treatment with the same therapy through an individual investigational new drug (IND) application. The application must be approved by the drug company, and the FDA has to agree with the company that there is adequate evidence of safety and efficacy.
Grant Williams, medical team leader at the FDA’s Division of Oncology Drug Products, said several hundred patients a year get individual INDs. Many also get access to products in development through expanded access programs, in which pharmaceutical companies make their treatment available to hundreds or thousands of people at once while they complete clinical trials.
But drug makers often balk at making their products available through “compassionate use” programs, especially the individual IND. Several patients said they were stonewalled when they asked for individual access, with no clear policy explained.
“Companies must be clear and honest with the public about their policies,” said Robert Erwin, director of the Marti Nelson Cancer Research Foundation. He said there are many valid reasons why drug makers might deny access, but that they should not blame the FDA or do so without explaining the reasons to the patient.
Robert Spiegel, chief medical officer of Schering-Plough, said his company has made five drugs available through these programs. But, he said, Schering and other companies worry that opening up access will hurt their ability to get patients into clinical trials of the experimental product. They are also concerned that such programs will stress their capacity to provide the drug, and may compromise their ability to ensure safety.
With increasing media coverage of new therapies, some companies are being inundated with requests. Gerard Kennealey, vice president of oncology clinical research at AstraZeneca Pharmaceuticals, said the company has received 7,000 phone calls since May, when there was a spurt of upbeat stories about its non-small cell lung cancer drug Iressa.
“We stopped counting when we got calls from 12 Senators’ offices,” said Kennealey, noting that celebrities and politicians were among the many people pleading for the drug.
AstraZeneca set up an expanded access program, and now has 200 patients enrolled. But, said Kennealey, the company has been careful to limit distribution only to non-small cell patients, and only to those who had failed other treatments, and weren’t eligible for other trials.
The National Breast Cancer Coalition said expanded access programs were the only legitimate, fair, way to make experimental therapies available, but that they should not be the norm. Jan Platner, director of administration and programs for the Coalition, said individual INDs in particular tend to undermine research, and that there is no fair way to grant them. They also contribute nothing to scientific knowledge, she said.