New FDA rule eases access to investigational drugs for gravely ill

ROCKVILLE, Md. The Food and Drug Administration published two rules Wednesday designed to expand patient access to investigational drugs.

The agency said the rules are designed to clarify methods that seriously ill patients can use to gain access to investigational drugs when they’re ineligible to participate in clinical trials and don’t have other satisfactory treatment options. It also launched a Web site where patients and their doctors can learn about options for receiving investigational drugs.

One rule, “Expanded Access to Investigational Drugs for Treatment Use,” expands availability of investigational drugs by clarifying procedures and standards. The other, “Charging for Investigational Drugs Under an Investigational New Drug Application,” clarifies circumstances and costs for which a manufacturer can charge patients for a drug when it’s used inside or outside of a clinical trial.

“With these initiatives, patients will have the information they need to help them decide whether to seek investigational products,” FDA commissioner Margaret Hamburg said. “For patients seeking expanded access to investigational drugs and biologics, the new rules make the process easier to understand.”