FDA Issues Final Rules to Help Patients Gain Access to Investigational Drugs

The US Food and Drug Administration (FDA) recently published two rules that seek to clarify the methods available to seriously ill patients interested in gaining access to investigational drugs and biologics when they are not eligible to participate in a clinical trial and don't have other satisfactory treatment options.

To support the effort to help these patients, the agency also launched a new website where patients and health-care professionals can learn about options for investigational drugs. In general, these options include being treated with a drug that has been approved by FDA, being given an investigational drug as part of a clinical trial, or obtaining access to an investigational drug outside of a clinical trial.

The new rule, "Expanded Access to Investigational Drugs for Treatment Use," makes investigational drugs more widely available to patients by clarifying procedures and standards. The other rule, "Charging for Investigational Drugs Under an Investigational New Drug Application," clarifies the specific circumstances and the types of costs for which a manufacturer can charge patients for an investigational drug when used as part of a clinical trial or when used outside the scope of a clinical trial.

"With these initiatives, patients will have the information they need to help them decide whether to seek investigational products," said Margaret A. Hamburg, MD, Commissioner of Food and Drugs. "For patients seeking expanded access to investigational drugs and biologics, the new rules make the process easier to understand."