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Compassionate Drug Use

Compassionate Drug Use

Woman in hospital bed about to receive a drug injection.

What is compassionate drug use?

Compassionate drug use is the use of an investigational drug when you are seriously ill but not enrolled in a clinical trial. Investigational drugs are in the process of being studied but have not yet been approved as safe and effective by the FDA.

Normally, investigational drugs undergo strict testing through many stages of clinical trials before they are available to the public. They are tested for safety, side effects, how well they work and at what doses, and with what conditions or diseases they are to be used. The process may take up to many years. Under certain circumstances, these drugs can be used outside of a clinical trial. 

Compassionate drug use is legal and a fairly new practice. The FDA first approved investigational drugs to be used in this way for critically ill patients in 1987.

Who is eligible to use these drugs?

The most common way people access unapproved drugs is by enrolling in a clinical trial. This is the safest route. The FDA closely regulates clinical trials, and people taking new drugs are carefully watched. However, not everyone will qualify for a clinical trial. Because the use of new, unapproved drugs is risky, only certain people who are not enrolled in clinical trials can receive investigational drugs for compassionate use. According to guidelines from the National Cancer Institute, you are eligible if you:

  • Have used standard treatments and found them unsuccessful

  • Are ineligible for any ongoing clinical trials

  • Have no acceptable treatment options

  • Have a cancer diagnosis for which an investigational drug has shown some benefit and is currently being studied in clinical trials

  • Are likely to have benefits that outweigh the risks involved

Accessing investigational drugs

To use investigational drugs, your doctor has to apply for it through a treatment investigational new drug application (Treatment IND). The application process includes information on your condition and past treatments.

The most common way to get approval for an investigational drug outside of clinical trials is through expanded access or special exception or single-patient exception.

  • Expanded access uses drugs that have been well-tested and are nearing FDA approval. The drug must show in studies that it is effective in treating a specific cancer. The program is offered by a drug company and the drug is given out to a group of people.

  • Special exceptions are individual requests (usually by a doctor) made to drug companies or to whomever is overseeing a program or clinical trial. The decision to give out the drug is weighed on a case-by-case basis. There must be some proof that the drug would either improve the patient's quality of life or lengthen his or her life.

Risks of using investigational drugs

There are drawbacks to compassionate drug use for you and for the drug approval process. One is that they may interfere with clinical trials if more people seek this route rather than participating in trials. Although the NCI gathers some safety information on compassionate-use drugs, it is not as helpful in getting FDA approval of these drugs. You are not watched as closely as you would be if you were part of a clinical trial.

Other problems include the chances of receiving no lasting benefit from the drug, undiscovered harmful side effects, and extra costs not covered by health insurance that may be associated with using the drug.

For more information

To learn more about investigational drugs and clinical trials, talk with your doctor about your options, the risks and side effects of the drug under consideration, and what the process is to apply to use the drug. Clear communication with the doctor who treats you is important. Together, you can determine whether a clinical trial or compassionate use is right for you.  

Call NCI's Cancer Information Service at 800-4-CANCER (800-422-6237) or visit www.clinicaltrials.gov to search for clinical trials for your condition.

If you do not qualify for a clinical trial, your doctor can ask the trial's sponsor about requesting a single-patient exception. He or she may also contact the NCI or the company making the drug to find out if expanded access programs exist.

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