Expanded Access

When a patient has a serious or immediately life-threatening condition that is not addressed by currently approved therapies, a physician may wish to offer an investigational drug (i.e., one that has not been approved by FDA) to treat the patient. While investigational drug use is usually limited to clinical trials where an investigational new drug (IND) application allows the product to be administered to human subjects, there are circumstances where a physician may be granted permission to use an investigational drug under expanded access.

Expanded access is the use of an investigational drug outside of a clinical trial to diagnose, monitor, or treat patients with serious or immediately life-threatening diseases or conditions for which there are no acceptable alternative therapies. Physicians that have identified a patient who may benefit from an investigational drug must first seek approval from the drug company that manufacturers the drug. If the company agrees to provide the drug, authorization must then be requested from the FDA through submission of an individual patient IND. These requests must also be authorized by an Institutional Review Board (IRB) and be conducted in compliance with FDA regulations on informed consent.

To support Duke physicians interested in obtaining expanded access to investigational drugs for individual patients, Duke University Hospital (DUH) and Duke University School of Medicine (SOM) have teamed up to provide a resource that assists physicians throughout the entire process, including support in each of the following areas:

• Pharmacy and Therapeutics Committee Approval: Duke University Hospital Pharmacy and Therapeutics (P&T) Committee will review all requests for individual patient expanded access and grant approval for utilization of non-FDA approved medications per institutional policy.
• Drug Company Approval: The Office of Regulatory Affairs and Quality (ORAQ) will assist with obtaining approval from the drug company for the expanded access use. ORAQ will also work with the Duke Office of Research Contracts (ORC) to ensure proper contractual and confidential disclosure agreements are in place for the expanded access use.
• FDA Authorization: ORAQ will prepare and submit the individual patient IND application to the FDA and will assist with maintaining that application once the investigational use has been authorized.
• IRB Authorization: A core of regulatory coordinators will assist with drafting an informed consent document and will process the IRB application through iRIS to receive IRB Chair Concurrence for the expanded access use.
• Investigational Drug Service: The investigational drug product will be received, stored and prepared for administration (as applicable) by Duke’s Investigational Pharmacy.
• MaestroCare Order: An analyst will build an order in MaestroCare so the drug can be ordered and documented in the patient medical record.

As a means to organize and expedite the workflow, the requesting physician will be informed of progress via email notifications at each step of the process. All individual patient expanded access requests will be supported by this resource, including both emergency and non-emergency use situations.

Interested in utilizing this resource? Please complete the survey of required information, and key team members and staff will be notified of your request. For questions or additional information, please contact ORAQ@duke.edu.

Individual Patient IND Request Survey

When a small group of patients may benefit from the same investigational drug, a physician may wish to open or participate in an intermediate-size expanded access program. In addition to the existing regulatory and institutional review requirements for an investigational drug, all intermediate-size expanded access programs must be reviewed and approved by the Duke University Hospital Pharmacy and Therapeutics (P&T) Committee. For information on how to submit a request for intermediate-size expanded access, please reference Duke University Hospital Policy on Utilization of Non-FDA Approved Medications. After reviewing the policy, physicians should complete the request form for utilization of a non-FDA approved drug and submit to the Center for Medication Policy. Intermediate-size expanded access programs are not currently supported through the individual patient IND service.

TEAMSS (Transforming Expanded Access to Maximize Support and Study) seeks to advance clinical care and translational research by improving patient access to experimental therapies. This federated, national consortium for Expanded Access interventions is a partnership led by the University of Michigan with Duke University, University of Rochester and University of Texas Southwestern.  Expanded Access provides an opportunity for patients who either lack therapeutic options or who are ineligible for clinical trials to potentially benefit from the clinical use of experimental drugs, biologics, and medical devices. By developing this national consortium, we will build a positive impact by, for the first time, creating a foundation for an integrated, nationwide approach to Expanded Access that can improve care for the most vulnerable patients.

TEAMSS Website