Q. What is "compassionate use"?
Being part of a clinical trial is the most common way that patients receive investigational drugs (i.e. drugs that have not yet been “FDA-approved”). The term “compassionate use” or “compassionate exemption” means that a patient is allowed to receive a drug even though he/she does not meet the eligibility criteria of a clinical trial in which a drug is being studied.
The decision to provide a drug in this manner is made on a case-by-case basis and there must be a reasonable expectation the drug will prolong life or improve a person’s quality of life. In addition, the sponsor of the clinical trial must agree to make the drug available and, as noted in a National Cancer Institute fact sheet, Access to Investigational Drugs, the drug being studied must also meet the following criteria:
- There must be substantial clinical evidence that the drug may benefit persons with particular type of cancer.
- The drug must be able to be given safely outside a clinical trial.
- The drug must be in sufficient supply for ongoing and planned clinical trials.
Read Understanding the Approval Process for New Cancer Treatments to learn more about the drug approval process, special needs programs such as “compassionate use” exemptions, as well as something known as “off-label” drug use, which can be a confusing issue for some patients.
Should you have any further questions, there are three ways you can contact the National Cancer Institute’s Cancer Information Service (CIS):