Compassionate use is a process that allows patients with a serious or life-threatening disease, such as cancer, to access an experimental drug when they do not qualify for a trial, or when there is no trial. Other terms used to refer to compassionate use include “expanded access”, “pre-approval access”, “early access“, “single patient IND“, “individual patient protocol“, or a “single patient request.”
There are currently about 900 cancer drugs in development for adult patients, but almost none are being studied in children. Often children are not eligible for clinical trials of new drugs only because of their age. Sometimes physicians have reasons to believe that a drug being tested for one type of cancer might be of some benefit to a patient with another type of cancer.
The severity of your patient’s disease or condition is a matter of clinical judgment. However, compassionate use requests are usually associated with life-threatening situations or persistent or recurrent morbidity that has a substantial impact on day-to-day life.
To apply for compassionate use, your patient must be unable to participate in a clinical trial of the drug you are applying for, and there must be no approved therapeutic alternative. The patient, or the patient’s guardian, must be willing to sign an informed consent accepting possible risks of using an investigational product.
If you are treating such a patient and know of an investigational drug that might help, we can help you in navigating the application process.