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

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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.

What is NCI’s role in providing access to investigational drugs?

NCI acts as the sponsor for many, but not all, investigational drugs. When acting as sponsor, 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 NCI. These requests are reviewed on a case-by-case basis.

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.