FDA expands access to investigational drugs

Internal Medicine News, Sept 15, 2009 by Damian McNamara

Two new final regulations issued by the Food and Drug Administration clarify and expand access to investigational drugs for patients with serious or life-threatening conditions.

"This topic is very important. These two rules clarify and improve FDA practices on investigational drugs, how they can be used prior to approval," Dr. Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, said during an FDA teleconference.

The first rule clarifies the agency's criteria for making investigational drugs more widely available to patients. The second rule clarifies what a manufacturer can charge patients for an investigational drug, used either inside or outside of a clinical trial. Manufacturers are not required, however, to make investigational products available for treatment use.

Historically, patients accessed promising investigational drugs once the agents had moved along in the development process, typically around the time of phase III trials. "But we know patients often want earlier access, and they may be willing to take more risk," Dr. Woodcock said.

The new regulations lay out a pathway for accessing drugs as early as the end of a phase I trial, Dr. Woodcock said. She emphasized that patients and their doctors must acknowledge the greater risks associated with an agent taken earlier in the development process.

The second regulation instructs manufacturers on the process to recoup costs associated with providing an investigational agent. Companies can charge patients for treatment and administrative costs associated with providing the drug outside a trial after demonstrating to the FDA the financial burden involved. The hope is that allowing manufacturers to charge will provide a greater incentive to expand access to investigational agents, Dr. Woodcock said.

In a clinical trial, a scenario in which a company's potential financial gain is greater, the criteria are more stringent, and companies are allowed to recoup only the drug costs.

A new FDA Web site explains how providers and patients can obtain investigational drugs: Go to www.fda.gov and search for ucm 172492.