FDA Details Access to 'Investigational' Drugs
Aug. 13 -- WEDNESDAY, Aug. 12 (HealthDay News) -- By clarifying rules governing patient access to so-called investigational drugs, U.S. health officials said Wednesday that they hope to expand the number of people who might benefit from these not-yet-approved medications.
"The fact that patients can get the drugs hasn't changed, but there is now a clearer pathway," said Dr. Janet Woodcock, director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration. "We have always been afraid that only those in the know about this can get these drugs. Better dissemination is another piece of transparency that's being provided."
Woodcock stressed that the drugs would potentially be available only to individuals who are gravely ill or have life-threatening conditions and for whom no other reasonable treatment alternatives exist. She could not say how many more patients might be able to benefit from the program under the new protocol.
"Anything FDA does to promote transparency is good for us," said Lynda Dee, a patient representative for the FDA's Antiviral Drugs Advisory Committee and a founding member of the AIDS Treatment Activist Coalition in Baltimore. "This will go far in helping people who need these new treatments."
Both Woodcock and Dee spoke at a Wednesday news conference to announce the publication of two final rules related to these access questions, and to announce a new Web site geared to health-care professionals and patients.
Since 1987, the FDA has had a formal process in place to help seriously ill patients get access to investigational drugs, although, in reality, an informal practice dates back to the 1970s.
"Tens of thousands of patients have been treated over those decades," Woodcock said. This includes patients with HIV/AIDS, cancer and other critical conditions.
Investigational drugs are ones that are in the process of being approved by the FDA for use but are still being tested for safety and effectiveness. "We don't know as much about these drugs as we do approved drugs," Woodcock said.
