“Compassionate Use” Access
to Investigational Drugs
Single Patient Investigational New Drug
Application or “Compassionate Use”
“Single patient Investigational New Drug (IND) application” refers to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available. Drugs that are being scientifically tested but have not yet been approved by the United States Food and Drug Administration (FDA) are called investigational drugs. Being permitted to use one of these drugs outside of a clinical trial specifically designed to study that drug may be commonly referred to as “compassionate use.”
“Compassionate use” is more properly termed “single patient Investigational New Drug (IND) application.”
Single patient IND is very carefully regulated and the approval process to use such drugs for an alternate purpose is rigorous. The FDA has developed a patient- and family-friendly website that provides information about access to investigational drugs under several circumstances, as well as information that might help you make a decision about whether or not to seek access.
Remember that this information applies only to drugs that are considered investigational. If there is no current clinical trial and the drug is not approved for treatment of any condition, it would not fall under this approach to drug access.
Another significant aspect of requesting single patient IND access to a drug is the ethical decision-making that parents, care givers and regulators must negotiate as they decide about an unapproved use of a drug. A recent article published in the Archives of Disease in Childhood entitled, Compassionate and Innovative Treatments in Children: A Proposal for an Ethical Framework by Drs. Joe Brierley and Vic Larcher, outlines a series of questions that can be considered when deliberating about single patient IND use. Issues include potential for effectiveness, how to obtain informed consent, access to the drug by the patient population in question, and others. Please see the full article (linked above) for more details.
There is additional information on the FDA Web site called FDA Basics that may be helpful in providing some background on drug development.
Click below for information outlining the process required for a drug to travel from the lab to patients in the U.S.