For Any Cancer Diagnosis
What is "compassionate use"?A.
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.
Should you have any further questions, there are three ways you can contact the National Cancer Institute’s Cancer Information Service (CIS):
For Breast Cancer
My mother has stage 4 breast cancer and I'd like to know if she would be eligible for any clinical trials.A.
Whether or not a clinical trial would be an option for your mother will be determined by several factors. The guidelines that clinical trials follow state who will be able to join the study, based on the questions the research is trying to answer. Therefore, your mother’s type of cancer, as well as the stage of her disease, her age and whether she has received any prior treatment would be examples of some of the eligibility criteria that may come into play. I would encourage you and your mother to speak to her doctor about this important question, since only her doctor can determine whether a clinical trial would be appropriate.
To locate clinical trials that might be suitable for her, call the National Cancer Institute’s (NCI) Cancer Information Service at 1-800-422-6237, or you can search the NCI’s clinical trials database. If you need help using our clinical trials online search form, read Help Using the NCI Clinical Trials Search Form.
CancerCare’s clinical trials publications are also excellent resources.
For Lung Cancer
My husband has just been diagnosed with small cell lung cancer with small tumors in his liver. He starts chemo next week with 4 hours, day 1 and 2 hours, day 2 and 3. He repeats this every 18 days for six sessions. Is this the normal treatment for small cell lung cancer? Where would I look to find information on clinical trials?A.
Later-stage small cell lung cancer is characterized by a spread of the disease from the lungs to other organs such as the liver, and is normally treated with aggressive chemotherapy. Since the treatment is aggressive, it must be administered over a number of days for each session, and the sessions are spaced out with non treatment breaks of 18 to 21 days to make sure that the patient’s overall health is not affected. Patients whose tumors respond well to chemotherapy may be considered for a radiation treatment to the brain called Prophylactic Cranial Irradiation (PCI) in which the entire brain receives radiation with the intent of stopping the spread of the disease. For more information about the treatment of small cell lung cancer, please visit the National Cancer Institute’s website.
CancerCare collaborates with EmergingMed’s clinical trials matching service, which is designed to assist lung cancer patients in identifying and accessing clinical trials which may be appropriate to their medical situation.
How does one get compassionate care in clinical trials when there is nothing left in the way of lung cancer treatments?A.
We believe you are asking about “Compassionate Drug Use” also called “Compassionate Use,” which is the use of therapeutic drugs that have not yet been approved by the FDA to treat cancer, and no other treatments are available. These drugs are called investigational drugs and are usually only available to patients who are participating in a clinical trial. Use of these drugs outside of a clinical trials is extremely limited.
Requests for the compassionate use of an investigational drug must be made via a request from the patient’s doctor to the drug company requesting their permission for its use and their willingness to supply the drug for the patient’s use. If the drug company consents, and frequently they do not, then the doctor must ask the FDA for their permission to use the drug for his or her patient only. The request to the FDA must include detailed medical information about the patient, why the request is necessary, a proposed plan of treatment and the patient’s signed consent. The length of time for FDA approval varies, but may be expedited in emergency situations.
For more information on compassionate drug use, the American Cancer Society provides a helpful reference guide.