Key Points

An investigational drug is one that is under study and is not approved by the U.S. Food and Drug Administration for sale in the United States.

The most common way patients receive investigational drugs is by taking part in clinical trials.

Patients sometimes receive investigational drugs through mechanisms such as expanded access protocols and special exception programs.

Specific criteria must be met to receive an investigational drug outside a clinical trial.

Patients interested in receiving investigational drugs should talk to their physician.

In general, investigational drugs are provided free of charge.

1. What is an investigational drug?

An investigational drug is one that is under study but does not have permission from the U.S. Food and Drug Administration (FDA) to be legally marketed and sold in the United States.

FDA approval is the final step in the process of drug development. The first step is for the new drug to be tested in the laboratory. If the results are promising, the drug company or sponsor must apply for FDA approval to test the drug in people. This is called an Investigational New Drug (IND) Application. Once the IND is approved, clinical trials can begin. Clinical trials are research studies to determine the safety and measure the effectiveness of the drug in people. Once clinical trials are completed, the sponsor submits the study results in a New Drug Application (NDA) or Biologics License Application (BLA) to the FDA. This application is carefully reviewed and, if the drug is found to be reasonably safe and effective, it is approved.

2. How do patients get investigational drugs?

By far, the most common way that patients get investigational drugs is by taking part in a clinical trial sponsored under an IND. A patient's doctor may suggest a clinical trial as one treatment option. Or a patient or family member can ask the doctor about clinical trials or new drugs available for cancer treatment.

Another way patients and their families can learn about new drugs being tested in clinical trials is through the National Cancer Institute's (NCI) PDQ® database. This database contains information on a large number of ongoing studies. Individuals can search this database at http://www.cancer.gov/clinicaltrials, or they can call the NCI's Cancer Information Service at 1-800-4-CANCER (1-800-422-6237). Information specialists can search the database and provide a list of trials for individuals to discuss with their doctor.

3. Are there other ways to get investigational drugs?

Less common ways that patients can receive investigational drugs include mechanisms such as an expanded access protocol or as special or compassionate exception. The sponsor must agree to provide the drug for this use.

Investigational drugs given under these mechanisms must meet the following criteria:

• There must be substantial clinical evidence that the drug may benefit persons with particular types 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.

Expanded Access

The purpose of an expanded access protocol is to make investigational drugs that have significant activity against specific cancers available to patients before the FDA approval process has been completed. Expanded access protocols allow a larger group of people to be treated with the drug.

The sponsor must apply to the FDA to make the drug available through an expanded access protocol. There must be enough evidence from studies already completed to show that the drug is likely to be effective against a specific type of cancer and that it does not have unreasonable risks. The FDA generally approves expanded access only if there are no other satisfactory treatments available for the disease.

The NCI's Treatment Referral Center (TRC) protocols are one type of expanded access protocol. The NCI establishes a TRC protocol when clinical evidence suggests that an investigational drug should be made more widely available to patients, even though the FDA approval process has not been completed. The TRC protocol is made available at NCI-designated cancer centers and other institutions selected to provide wide geographic availability of the drug to patients.

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Tags: Cancer

About the Author

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