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Home > Resources > Access to Care Resources > Clinical Trial Info > Investigational Drugs Q&A

Questions and Answers about Investigational Drugs, continued

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.

A request to treat a patient with an investigational drug outside a clinical trial must be made to the drug company and to the FDA. The request to the FDA is sent as general correspondence to the appropriate reviewing division where the IND application is filed. The drug company can provide the name of the appropriate reviewing division. (FDA reviewing divisions are prohibited from divulging proprietary information such as whether a sponsor has filed an IND or the status of an IND.)
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7. Are there specific criteria used to determine whether patients can receive an investigational drug outside the clinical trial setting?
Generally, patients must meet the following criteria to be considered for treatment with an investigational drug outside the clinical trial setting; they must:
  • have undergone standard treatment that has not been successful;
  • be ineligible for any ongoing clinical trials; and
  • have a cancer diagnosis for which an investigational drug has demonstrated activity and is being studied in ongoing Phase 2 or Phase 3 protocols.
The potential benefits of receiving the drug should outweigh the risks involved.
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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 that outlines the known risks and benefits of the treatment, as well as the rights and responsibilities of the patient.
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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. Patients should check with their insurer about coverage of these costs prior to beginning treatment.
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10. What are some of the potential drawbacks to receiving an investigational drug?
There are some 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 who is receiving an investigational drug may not receive any benefit from it. 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.
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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 Cancer Information Service at 1-800-4-CANCER (1-800-422-6237).
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12. What other resources are available on this topic?
The following list of resources may be helpful:
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