Cancer: Access to Investigational Drugs, Part 2

by National Cancer Institute

(Page 2 of 2)

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.

In some cases, even patients who qualify for treatment with an investigational drug on a compassionate basis might not be able to obtain the drug if the supply is limited and the demand is high.

4. Are all investigational drugs available through an expanded access or special exception mechanism?

No. The sponsor decides whether to provide an investigational drug outside a clinical trial. Availability may be limited in part by drug supply, patient demand, or other factors.

5. What is the NCI's role in providing access to investigational drugs?

The NCI acts as the sponsor for many, but not all, investigational drugs. When acting as sponsor, the NCI provides the investigational drug to the physicians who are participating in clinical trials or TRC protocols. A physician who wishes to treat a patient with the investigational drug as a special exception must request the drug from the NCI. These requests are reviewed on a case-by-case basis.

6. 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.)

7. 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

8. 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.

9. 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.

10. 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.

11. How can patients find out more information about a specific investigational drug?

Patients can find out more about a specific drug by contacting the drug company that is developing the drug. Information may also be available from the NCI's Cancer Information Service at 1-800-4-CANCER (1-800-422-6237).

About the Author

www.nci.nih.gov
The National Cancer Institute's research programs are extensive and contain many innovative initiatives. I invite you to explore our Web site to find out more about the exciting work being conducted here at NCI and by NCI-supported scientists throughout the country.