National Cancer Institute NCI Cancer Bulletin: A Trusted Source for Cancer Research News
June 15, 2010 • Volume 7 / Number 12

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FDA Update

FDA-ASCO Educational Module Expands Access to Investigational Drugs

Screenshot of Expanded Access to Investigational Drugs Web page

When a patient has a serious or life-threatening disease and no satisfactory alternative therapies, what options does a physician have in providing care for the patient?
 
Depending on the patient’s clinical course and situation, he or she may be eligible to receive an investigational drug to treat the disease or condition. Treatment use of an investigational drug, referred to as expanded access, has occurred since the 1970s. However, this mechanism was not widely known or used by physicians. Expanded access to an investigational drug provides a patient with access to drugs that have not been approved by the FDA. One of the requirements is that the patient has a serious or life-threatening disease and no satisfactory alternative therapy. The goal of providing this type of access is to treat patients.

Because many physicians were unaware of the expanded access mechanism and the process for requesting expanded access, the FDA published a new rule on expanded access in August 2009. The new rule clarified existing regulations on providing treatment use of investigational drugs and also outlined criteria, submission requirements, and safeguards for expanded access programs.

To familiarize oncologists with expanded access and its processes, the FDA and the American Society of Clinical Oncology (ASCO) have developed an educational module that explains expanded access, the process for requesting an expanded access program for an individual-patient Investigational New Drug (IND) application, other options for patients who have no satisfactory alternative therapies, and investigator responsibilities in sponsoring an expanded access program.

The module is available on the ASCO University Web site and is available for members and nonmembers free of charge. The module also includes a glossary of regulatory terms often used when requesting expanded access, an expanded access checklist, and helpful templates often submitted with an expanded access IND application.

Dr. Richard Pazdur
Director, FDA Office of Oncology Drug Products

Dr. Tamy Kim
Acting Associate Director for Regulatory Affairs, FDA Office of Oncology Drug Products

NCI’s Efforts to Provide Patients with Access to Investigational Drugs

NCI’s Cancer Therapy Evaluation Program (CTEP) has a long-standing program designed to provide patients access to investigational anticancer agents. The Treatment Referral Center (TRC), managed by CTEP's Pharmaceutical Management Branch (PMB), is the point of contact for clinical investigators seeking investigational treatments for their patients. TRC staff can research and provide information on relevant on-going clinical trials. If a patient is not eligible to participate in a clinical trial, TRC staff have mechanisms to provide expanded access to Investigational New Drugs (IND), the most often used being a “special exception” mechanism.

For agents where CTEP holds an IND Application, and where there is company collaborator concurrence, CTEP provides single-patient access to investigational agents that have both a demonstrated safety profile and demonstrated activity in the specific disease. These agents are available to all CTEP-registered investigators. Since 1988, CTEP has treated over 19,000 patients through the special exception program.

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