FDA issues final rules to help patients access investigational drugs

The FDA has published two new final rules explaining how critically ill patients can get access to investigational therapies when they are ineligible for clinical trials and do not have other treatment options.

The first rule, “Expanded access to investigational drugs for treatment use,” clarifies procedures and standards with the aim of making investigational drugs more widely available. It clarifies existing regulations and adds new types of expanded access for treatment use. Under the final rule, expanded access to investigational drugs for treatment use will be available to:

Individual patients, including in emergencies.
Intermediate-size patient populations.
Larger populations under a treatment protocol or treatment investigational new drug application (IND).

The second rule, “Charging for investigational drugs under an investigational new drug application,” spells out the specific circumstances and costs for which a manufacturer can charge patients for an investigational drug. The rule revises the charging regulation to:

Clarify the circumstances under which charging for an investigational drug in a clinical trial is appropriate.
Set forth criteria for charging for an investigational drug for the different types of expanded access for treatment use described in FDA’s final rule on expanded access for treatment use of investigational drugs.
Clarify what costs can be recovered.

The agency has also launched a website for patients and physicians seeking more information about their options with regard to these investigational drugs. It includes information on treatment with an FDA-approved drug, treatment with an investigational drug as part of a clinical trial or obtaining access to an investigational drug outside of a clinical trial.

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