Expanded Access (also sometimes referred to as "Compassionate Use") is FDA's program allowing for the use of an investigational new drug (IND) or biologic to treat a patient with a serious or life-threatening disease or condition who does not meet the enrollment criteria for a clinical trial in progress or have alternative therapies available. Use of an investigational product by a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. However, when enrolling in a clinical trial is not possible (i.e., a patient is not eligible for any ongoing clinical trials) and no other alternative therapies are available; patients who have a serious disease or condition that may benefit from treatment with the drug may be able to receive the product through Expanded Access.
Confirm that your patient meets the requirements for Expanded Access use of an investigational therapy (listed below), then contact the AIM team using the AIM request support form. We will gather information and will facilitate all steps involved in pursuing Expanded Access to an investigational therapy for your patient.
1. The patient and his or her physician are both willing to participate;
2. The patient’s physician determines that there is no comparable or satisfactory alternative therapy available;
3. The patient’s physician determines the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition;
4. FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance;
5. FDA determines that providing the investigational product will not interfere with the ongoing clinical trial;
6. The patient’s physician volunteers to serve as the sponsor-investigator, or the entity responsible for the treatment plan development and implementation;
7. The patient is unable to participate in the clinical trial.
FDA reports allowing over 99% of single patient Expanded Access requests to proceed. FDA may contact the physician to request more information or clarification in order to avoid placing the IND on hold.
For emergency Expanded Access requests, call the Vanderbilt Human Research Protection Program at (615) 322-2918 and select option #3.
Right-to-try (RtT) refers to legislation passed at the state and, more recently, at the federal levels that purports to grant terminally ill patients access to investigational drugs without FDA oversight. Right-to-try is completely separate from the FDA Expanded Access Program and is not regulated or managed by FDA. It is currently unclear precisely how the Right-to-Try pathway will work however, FDA communications indicate that the choice to pursue the Right-to-Try path or the traditional Expanded Access path is up to the discretion of the drug company who will be providing the investigational therapy.
Both programs allow patients to apply for access to investigational therapies outside of clinical trials, however, there are some major differences between FDA's Expanded Access Program and the recently passed federal Right-to-Try law. For example, Right-to-Try only applies to investigational drugs, while Expanded Access includes all types of medical products. In Expanded Access, the treating physician, drug company, FDA and IRB work together to ensure that the proposed treatment plan offers a favorable risk/benefit profile for the patient, that informed consent is ethically sound, and that costs are appropriate. However, Right-to-Try is not regulated or managed by FDA and only the treating physician and drug company provide oversight. Unlike Expanded Access, Right-to-Try does not require rapid reporting of adverse events to FDA. Right-to-Try provides liability protection to companies involved in the therapeutic attempt, while Expanded Access does not. Importantly, neither program requires the drug company to provide the investigational medicine being requested.