Expanded access or compassionate use is an FDA program that under certain conditions allows 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. Patients and physicians need to establish that there are no other alternative therapies available, and that the treatment benefit justifies the potential risks of treatment. They must also demonstrate that providing the investigational drug will not interfere with investigational trials that could support the product’s development or marketing approval for the treatment indication being developed in the ongoing clinical trials. In meeting these criteria, patients may gain access to an unapproved drug in an ongoing clinical regulated trial through the expanded access program1.
To apply for expanded access, patients will need to consult with their physician to make sure they have exhausted all other treatment options and that the investigational product holds the promise of a potential treatment. Their physician must agree to oversee the patient’s treatment and be willing to work with a drug developer who is willing to provide the investigational medical product. The patient’s physician will file all the paperwork with FDA and Institutional Review Board who review the expanded access protocol and consent forms to make sure the patient understands the nature of the treatment and who is responsible for all patient care and reporting. The final step is the FDA review of the expanded access protocol to decide if the treatment can proceed2. There are both emergency access pathways where FDA responds within 24 hours and a pathway that takes about 30 days for FDA review.
For those who have been reading our latest blogs on Right to Try, the expanded access program will seem very similar when it comes to patient criteria for access to investigational drugs. There are many resemblances, but the differentiating factor for Right to Try is that FDA permission for use is not required so there are far fewer regulatory hurdles. This said, the expanded access program has been in place since 1987 and many patients have received investigational drugs and biologics through the program, at an FDA allowance rate of more than 90%3.