Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions.
Such investigational drugs/devices have not yet been approved by the FDA and they have not been proven to be safe and effective.
They may be effective in the treatment of a condition, or they may not. It is important to remember that the drug/biologic/medical device may have unexpected serious side effects and that patients need to consider all the possible risks when seeking access to an investigational medical product.
Under FDA’s current regulations for investigational drugs (including biologics), FDA has three categories that expanded access can be approved under. They are:
- Expanded access for individual patients, including for emergency use;
- Expanded access for intermediate-size patient populations; and
- Expanded access for widespread treatment use.
What does USFDA review while processing expanded access request?
1. Whether disease is serious / condition
2. Whether it is immediately life threatening
The FDA must also determine that:
There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
The patient cannot obtain the drug under another IND or protocol.
The potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated.
Providing the investigational drug will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access use.