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Using Investigational Medicines

What are investigational medicines?

Investigational medicines are used when you are seriously ill but not enrolled in a clinical trial. These medicines are being studied but have not yet been approved as safe and effective by the FDA. It is sometimes called compassionate medicine use.

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

Investigational medicine use is legal. The FDA has been approving investigational medicines to be used in this way for critically ill people since 1987.

Healthcare provider with electronic tablet talking to woman.

Who is eligible to use these medicines?

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

  • Have used standard treatments and they didn't work

  • Are ineligible for any ongoing clinical trials

  • Have no acceptable treatment options

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

  • Are likely to have benefits that outweigh the risks involved

Getting investigational medicines

To use investigational medicines, your healthcare provider has to apply for them 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 medicine outside of clinical trials is through expanded access or special exception or single-patient exception.

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

  • Special exceptions are individual requests (often by a healthcare provider) made to medicine companies or to whomever is overseeing a program or clinical trial. The decision to give out the medicine is weighed on a case-by-case basis. There must be some proof that the medicine would either improve a person's quality of life or lengthen their life.

Risks of using investigational medicines

There are drawbacks to compassionate medicine use for you and for the medicine 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 the use of investigational medicines, it is not as helpful in getting FDA approval of these medicines. You are not monitored as closely as you would be if you were part of a clinical trial.

Other problems include:

  • Chances of getting no lasting benefit from the medicine

  • Undiscovered harmful side effects

  • Extra costs not covered by health insurance that may be linked to using the medicine

For more information

To learn more about investigational medicines and clinical trials, talk with your healthcare provider about your options, the risks and side effects of the medicine under consideration, and what the process is to apply to use the medicine. Clear communication with the healthcare provider 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 don't qualify for a clinical trial, your healthcare provider can ask the trial's sponsor about requesting a single-patient exception. Your provider may also contact the NCI or the company making the medicine to find out if expanded access programs exist.

Online Medical Reviewer: L Renee Watson MSN RN
Online Medical Reviewer: Rita Sather RN
Online Medical Reviewer: Todd Gersten MD
Date Last Reviewed: 2/1/2021
© 2021 The StayWell Company, LLC. All rights reserved. This information is not intended as a substitute for professional medical care. Always follow your healthcare provider's instructions.
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