Investigational drugs are those under evaluation in the clinical trial system, but not yet approved by the Food and Drug Administration to be legally marketed and sold. Most commonly, patients obtain investigational drugs by participating in a clinical trial sponsored by the National Cancer Institute, a cooperative group or a pharmaceutical manufacturer. In some cases, however, these drugs may be obtained through other channels such as expanded access programs or special or compassionate use exemption. If obtained in this manner, the sponsoring entity must agree to provide the drug.
Given under these circumstances, the following criteria must be met:
- There must be substantial evidence that the drug may benefit patients with a specific type of cancer.
- The drug must be able to be given safely outside the clinical trial setting.
- The drug must be manufactured in sufficient supply to accommodate all ongoing and future clinical trials.
Expanded Access Programs
The purpose of an expanded access program is to allow investigational drugs that have shown activity against specific cancers and do not carry unreasonable risks, to be available to larger patient populations before the drug has reached FDA approval. The sponsor of the drug must apply to the FDA to make this type of program available, and the FDA generally gives their approval only if no other satisfactory treatment for the disease exists. The chemotherapy drug, Alimta, which was the first drug approved specifically for pleural mesothelioma in 2004, was available to patients through an expanded access program. Many mesothelioma patients received the drug under this program, and benefitted from its early use.
Special or Compassionate Exemption
Patients who do not meet eligibility criteria for a clinical trial of an investigational drug, may be eligible to receive the drug under a special or compassionate exemption. In these cases, the patient’s doctor is responsible for contacting the drug’s sponsor, must provide the patient’s medical and treatment history and be of the opinion that receiving the drug will prolong the life of the patient or improve their quality of life. The sponsor evaluates the request on a case-by-case basis. If the drug is in short supply or there is high demand, the request may be denied.
In order to be considered for treatment outside the clinical trial setting, in general, patients must meet the following criteria:
- They have previously undergone standard treatment for their cancer that has proven unsuccessful.
- They are ineligible for any current clinical trials of the investigational drug.
- They have no acceptable treatment alternatives.
- They have a diagnosis for which the investigational drug has demonstrated effectiveness.
- They are likely to obtain benefits that outweigh the risks.
Patients interested in obtaining access to an investigational drug should discuss all available options with their doctor. If a drug is to be given outside of a clinical trial, physicians must follow certain guidelines including approval from their Institutional Review Board (IRB), and obtaining informed consent from the patient. Drugs are normally given free of charge, however, there may be other costs associated with the treatment. Patients should check with their insurance carrier to be sure they have a complete.