VII. Expanded Access to Investigational Drugs for Treatment Use [21CFR 312.300]

Expanded Access to Investigational Drugs for Treatment Use [21CFR 312.300]

Expanded access, also called compassionate use, is the use of an investigational drug outside of a clinical trial to treat a patient. It provides access to promising therapeutic agents without compromising the protections of human subjects or the thoroughness and scientific integrity of product development and marketing approval.

Three criteria must be met for the FDA to approve this use.

  1. The patient must have a serious or life-threatening condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition
  2. The potential benefit to the patient justifies the potential risks of the treatment use and the risks are not unreasonable in the context of the disease or condition to be treated
  3. Providing the investigational drug for this use must 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 this use.

An expanded access IND is available to individual patients, intermediate size patient populations and larger populations. The FDA requires an IND submission for each of these three types of expanded access. The submission may be a new IND or a protocol amendment to an existing IND. Submission requirements are outlined at 21 CFR 312.305(b). Sponsors and investigators of expanded access use must comply with the responsibilities of all IND sponsors and investigators to the extent that the responsibilities apply to expanded access use 21 CFR 312.305(c).