Access to Experimental Cancer Drugs

Credit: iStock

An experimental drug is one that has been tested in the lab and with animals and approved for testing in people by the U.S. Food and Drug Administration (FDA). But, it can’t yet be advertised, sold, or prescribed. Experimental drugs may also be called “investigational.”

Ways to Access an Experimental Drug

Experimental drugs may be available through clinical trials or expanded access programs.

Clinical Trials

The most common and preferred way to access an experimental drug is through a clinical trial. Clinical trials are research studies that involve people. Trials are carefully designed to answer scientific questions about the new drug. Patients who take part are fully informed and carefully monitored. The data that are collected during a clinical trial helps researchers learn how the drug behaves in people and if it is safe and effective.

See Clinical Trials Information for Patients and Caregivers to learn more about clinical trials and how to find one for which you may be eligible.

Expanded Access (Compassionate Use)

Expanded access, which is also called compassionate use, is the use of an experimental drug outside of clinical trials to treat serious or life-threatening disease. It is a process that requires agreement between the patient and his or her doctor and is regulated by the FDA. If you meet certain criteria, you might be able to obtain an experimental drug through expanded access. To qualify, you must:

  • Have an illness that is life-threatening
  • Have no standard treatments available
  • Not be eligible for a clinical trial

How to Obtain a Drug through Expanded Access

The only way to obtain a drug through expanded access is with the help of your doctor. He or she will need to:

  • Ask the company that is developing and testing the experimental drug if it is willing to provide it. The company may not be willing to provide the drug if it believes that doing so is against its interests or if there isn’t enough drug available.
  • Apply to the FDA, if the drug company agrees to supply the drug. Your doctor should refer to Expanded Access: Information for Physicians on the FDA site for complete information.

How Your Safety Is Protected if You Obtain a Drug through Expanded Access

Several measures help protect your rights and safety if you are treated with a drug through Expanded Access:

  • An Institutional Review Board or its representative must approve the use.
  • The FDA must approve the use.
  • You will go through an informed consent process, which ensures that you understand the potential risks and benefits of taking the experimental drug.

Deciding on Treatment with an Experimental Drug

Your doctor is in the best position to help you understand all your treatment options.  

As you think about your options, keep in mind that there may be unknown serious side effects with experimental drugs. Also, cost may be an issue. Federal law requires most insurance plans to cover routine patient care costs related to taking part in a clinical trial. But your insurance may not cover the cost of an experimental treatment outside of a trial.

Our list of Questions to Ask Your Doctor about Clinical Trials may help you talk with your doctor about taking part in a clinical trial.