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.

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 Investigational New Drug. 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.

Are there other ways to get investigational drugs?

Less common ways that patients can receive investigational drugs include mechanisms such as the special or compassionate exception. The sponsor, such as the pharmaceutical company 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.

Special Exception/Compassionate Exemption
Patients who do not meet the eligibility criteria for a clinical trial of an investigational drug may be eligible to receive the drug under a mechanism known as a special exception or a compassionate exemption to the policy of administering investigational drugs only in a clinical trial. The patient's doctor contacts the sponsor of the investigational agent and provides the patient's medical information and treatment history. The sponsor (the drug company or NCI) evaluates the requests on a case-by-case basis. There should be reasonable expectation that the drug will prolong survival or improve quality of life.

Who can provide access to investigational drugs being developed by pharmaceutical companies?

In the case of investigational drugs sponsored by a drug company, the drug company in collaboration with the FDA provides access to the drug. The process is similar to that described above. The patient's physician must submit a request to the drug company and to the FDA. The drug company can provide the name of the appropriate reviewing division at the FDA. (FDA reviewing divisions are prohibited from divulging proprietary information such as whether a sponsor has filed an IND or the status of an IND.)

Are there specific criteria used to determine whether patients can receive an investigational drug outside a clinical trial?

To be considered for treatment with an investigational drug outside a clinical trial, generally patients must meet the following criteria:

• have undergone standard treatment that has not been successful
• be ineligible for any ongoing clinical trials of this drug
• have no acceptable treatment alternatives
• have a cancer diagnosis for which the investigational drug has demonstrated activity
• be likely to experience benefits that outweigh the risks involved

What should patients do if they are interested in receiving an investigational drug through a special exception or expanded access mechanism?

Patients interested in gaining access to investigational drugs should talk to their physician about available options. Physicians can make requests for special exceptions by contacting the study sponsor. Physicians will be required to follow strict guidelines, including gaining approval from their Institutional Review Board and obtaining informed consent from the patient. Informed consent is a process that includes a document to be signed by the patient which outlines the known risks and benefits of the treatment, as well as the rights and responsibilities of the patient.

What are the costs involved in receiving an investigational drug?

In general, the drug is provided free of charge. However, there may be other costs associated with the treatment. Before beginning treatment, patients should check with their insurer about coverage of these costs.

What are some of the potential drawbacks to receiving an investigational drug?

It is not known whether an investigational drug is better than standard therapy for treating a disease, and a patient may not receive any benefit. Side effects (both long-term and short-term) from the drug may not be fully understood, especially if the drug is in early phases of testing. Finally, a patient's health insurance company may not pay expenses associated with receiving the investigational drug.