FDA Details Access to 'Investigational' Drugs

WEDNESDAY Aug. 12, 2009 -- 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.

The new rules will not interfere with the drug-approval process, officials said.

"We want to achieve a balance between the need to gather information to demonstrate whether drugs work and the fact that critically ill patients urgently want access and may be willing to take greater risks if they have no reasonable alternatives," said FDA Commissioner Dr. Margaret A. Hamburg. "The expanded access procedures will allow patients to get access without disrupting the gathering of information."

Under the new protocol, single patients as well as intermediate- and large-sized groups could gain access to experimental drugs.

The second rule published Wednesday details the circumstances under which manufacturers can actually charge patients for use of investigational drugs.

"Making these drugs available outside of a clinical trial can be costly to manufacturers," Woodcock said. "By permitting charging for expanded access we hope to stimulate more companies to provide investigational drugs."

According to Woodcock, deciding which companies will be allowed to recover part of their costs this way will be a "judgment call" on the part of the FDA. "They will have to give us financial information and they will have to convince us that the drug would not be developed absent this charging," she said. The company could also charge for administrative costs and for the cost of monitoring the patient, she added.

It's possible that some patients could get access to investigational drugs as early as late-stage Phase I trials, the earliest types of trials, Woodcock said.

"Together, the two final rules announced will make wider availability of investigational drugs possible by providing clearer guidelines and by reducing barriers that prevent access from being granted," she said. "We hope that the two together may result in more patients with serious and life-threatening illnesses receiving access to investigational drugs."

More information

For more on access to investigational drugs, visit the FDA.

Posted: August 2009


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