What is Expanded Access?
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 the Expanded Access Program.
How do I pursue Expanded Access to an investigational therapy for my patient?
Confirm that your patient meets the requirements for Expanded Access use of an investigational therapy (listed below), then contact the AIM team by using the AIM request support form (or by email or phone). We will gather information and will work with you to facilitate all steps involved in pursuing Expanded Access to an investigational therapy for your patient.
What requirements must be met in order for a patient to qualify for Expanded Access use of an investigational therapy?
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.
How many Expanded Access requests are typically approved by FDA?
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.
What is the difference between an emergency request and a non-emergency request?
- In a non-emergency situation, a written request for individual patient use of an investigational drug must be submitted to the FDA. The investigational drug may be shipped and treatment of the patient may begin 30 days after the application is received by FDA or earlier if notified by the FDA that treatment may proceed. These non-emergency requests are known as individual patient INDs, or single patient expanded access requests. A guide to initiate and maintain non-emergency requests is available for physicians online.
- Emergency use of an investigational medicine may be warranted in a life-threatening situation in which no standard acceptable treatment is available, and in which there is not sufficient time to obtain IRB approval. In an emergency situation, the request to use an unapproved investigational drug may be made via telephone or other rapid means of communication, and authorization to ship and use the drug may be given by the FDA official over the telephone. In these situations, known as emergency IND (eIND) requests, shipment of and treatment with the drug may begin prior to FDA’s receipt of the written IND submission that is to follow the initial request. An emergency IND timeline is available online to guide you through the process.
How do I pursue an emergency request for Expanded Access for my patient?
For emergency Expanded Access requests, please call the Vanderbilt Human Research Protection Program at (615) 322-2918 and select option #3.
What is “Right-to-Try”?
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.
What are some differences between”Expanded Access” and “Right-to-Try”?
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.