For Any Cancer Diagnosis
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):
Q. My cousin has advanced cancer. Is there a clinical trial she can get into? How do we find out?
Since clinical trials have eligibility criteria, which are specific guidelines that determine who may or may not enter a clinical trial, a good place to begin would be to have a discussion with your cousin’s doctor. It is important to know certain details about her diagnosis and, if she has already received treatment, knowledge about her treatment history is also important. Her doctor may be able to assist your cousin in finding a clinical trial that is appropriate for her.
Information about cancer clinical trials is available from the National Cancer Institute’s (NCI) Cancer Information Service (CIS). Information Specialists at the CIS use Physician Data Query (PDQ), a comprehensive database, to identify and provide detailed information about specific clinical trials. PDQ includes all NCI-funded clinical trials and some studies conducted by independent investigators at hospitals and medical centers in the United States and Europe. To speak with an Information Specialist and have a tailored search performed for your cousin, call 1-800-4CANCER (1-800-422-6237).
You may also search for clinical trials through the NCI’s website. The NCI offers a useful guide, How to Find a Cancer Treatment Trial: A 10-Step Guide. This resource can help cancer patients gather the information needed to search for a cancer treatment clinical trial, identify sources that list clinical trials, learn about clinical trials that may be of potential benefit, and ask questions that will help them decide whether or not to participate in a particular trial.
The National Institute of Health’s (NIH) website, ClinicalTrials.gov, lists clinical trials sponsored by the NIH, other Federal agencies, and the pharmaceutical industry for a wide range of diseases, including cancer. You may also wish to contact the National Institutes of Health’s (NIH’s) Clinical Center, a U.S. Government facility located in Bethesda, Maryland, that treats patients in clinical research trials. To learn more about this center, read Cancer Clinical Trials at the National Institutes of Health Clinical Center: Questions and Answers.
Q. Are there clinical trials for cancer treatments other than chemotherapy?
Often times, people think clinical trials are limited to exploring ways of improving existing standard treatments such as chemotherapy. It is important to remember that there are other types of clinical trials, each designed to answer scientific questions in order to find better ways to prevent, screen for, diagnose and manage a disease.
Prevention trials mostly involve people who have not had a history of cancer, although some are conducted with people who have had cancer and want to prevent a return of their disease or reduce the chance of developing a new type of cancer. Participants in a prevention trial may take a particular medication, vitamin or other supplement in order to see if they are effective preventive agents.
Screening trials include people who do not have any symptoms of cancer. These studies aim to determine whether finding cancer before it causes symptoms will decrease one’s chance of dying from the disease. Imaging studies are another significant area of research and help determine the value of imaging procedures for detecting, diagnosing, guiding, or monitoring the treatment of disease.
Supportive care trials include areas of research that are investigating new ways to improve the comfort and quality-of-life of cancer patients and cancer survivors. These studies look at ways to help people who may be experiencing side effects such as nausea, depression, pain or fatigue caused by their disease or its treatment.
Genetics trials focus on how one’s genetic makeup affects detection, diagnosis, or response to cancer treatment. Other genetics research-referred to as “population” or “family-based” genetic research will look at tissue or blood samples, generally from families or large groups of people, to find genetic changes that are associated with cancer. The goal of these studies is to help understand the role of genes in the development of cancer. Learn more about genetics.
Clinical trials of all types are the only method for advancing the practice of medicine, for increasing patients' options, for improving patient outcomes, for better addressing symptom management, as well as finding better and more targeted methods for preventing, detecting and/or treating cancer.
For more information on clinical trials, or to have a tailored clinical trial search done, please contact the NCI’s Cancer Information Service (CIS) for personal, confidential help at 1-800-4-CANCER (1-800-422-6237).
For Breast Cancer
Q. My mother has stage 4 breast cancer and I'd like to know if she would be eligible for any clinical trials.
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.
In addition, NCI’s Clinical Trials: Questions and Answers fact sheet includes information about types of clinical trials, who sponsors them, how they are conducted, how participants are protected, and who pays for the patient care costs associated with a clinical trial. It also includes some questions people might ask their health care provider before entering a clinical trial.
CancerCare’s clinical trials publications are also excellent resources.
For Lung Cancer
Q. 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?
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.
Q. How does one get compassionate care in clinical trials when there is nothing left in the way of lung cancer treatments?
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. Additional information can also be found through the FDA’s Expanded Access Program.