Are there other ways to get investigational drugs?


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Less common ways that patients can receive investigational drugs include mechanisms such as an expanded access protocol or a 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.

NCI’s Treatment Referral Center (TRC) protocols are one type of expanded access protocol. NCI program staff establish 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.

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 on a compassionate basis for treatment with an investigational drug might not be able to obtain the drug if the supply is limited and the demand is high.