FDA Issues Rules for Early Access to Experimental Drugs

Patients who suffer from serious and fatal illnesses may be granted early access to drugs that are still in the experimental stage. By clarifying the rules for granting patients access to the so-called investigational drugs, U.S. health officials said this week that they hope to be able to expand the number of people who might benefit from these medications that are not yet approved.

Dr. Janet Woodcock, director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration, said, “The fact that patients can get the drugs hasn’t changed, but there is now a clearer pathway. 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 these medications would potentially be available only to those individuals who are gravely ill or have life-threatening conditions and for whom there is no other reasonable treatment alternatives available. However, she could not say exactly how many more patients might be able to benefit from this program under the new protocol.

Lynda Dee, who is a patient representative for the FDA’s Antiviral Drugs Advisory Committee and who is a founding member of the AIDS Treatment Activist Coalition in Baltimore, stated that anything that the Food and Drug Administration does to help promote transparency is good for us, because this will go very far in helping people who need to receive these new treatments. Both Dee and Woodcock spoke at a news conference that was held on Wednesday to announce the publication of the two final rules related to these access questions, and to announce a new web site that is geared to patients and health-care professionals.