Under FDA’s statute, a sponsor or a physician may submit an expanded access request for widespread access to an investigational product if certain criteria are met:
- The investigational product is intended for use in the diagnosis, monitoring, or treatment of a serious or immediately life-threatening disease or condition;
- There is no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat that stage of disease or condition in the relevant patient population;
- The investigational product is being studied in a controlled clinical trial for the same use, under an IND or IDE or all clinical trials necessary for approval of that use have been completed;
- The sponsor of the controlled clinical trials is actively pursuing, with due diligence, marketing approval, or clearance for the same use;
- If the investigational product is being studied in a controlled clinical trial, under an IND or IDE, providing the investigational product under a treatment IND or IDE will not interfere with the enrollment in the ongoing clinical investigation(s);
- In the case of serious diseases, there is sufficient evidence of safety and effectiveness to support the use for the indication under the treatment IND or IDE;
- In the case of immediately life-threatening diseases, the available scientific evidence, taken as a whole, provides a reasonable basis to conclude that the investigational product may be effective for its intended use and would not expose patients to an unreasonable and significant risk of illness or injury.