Investigational Drugs

Investigational Drugs: Questions and Answers

Key Points
An investigational drug is one that is under study and is not approved by the U.S. Food and Drug Administration for sale in the United States (see Question 1).
The most common way patients receive investigational drugs is by taking part in clinical trials (see Question 2).
Patients sometimes receive investigational drugs through mechanisms such as expanded access protocols and special exception programs (see Question 3).
Specific criteria must be met to receive an investigational drug outside a clinical trial (see Questions 3 and 7).
Patients interested in receiving investigational drugs should talk to their physician (see Question 8).
In general, investigational drugs are provided free of charge (see Question 9).
What is an investigational drug?
An investigational drug is one that is under study but does not have permission from the U.S. Food and Drug Administration (FDA) to be legally marketed and sold in the United States.
FDA approval is the final step in the process of drug development. The first step is for the new drug to be tested in the laboratory. If the results are promising, the drug company or sponsor must apply for FDA approval to test the drug in people. This is called an Investigational New Drug (IND) Application. Once the IND is approved, clinical trials can begin. Clinical trials are research studies to determine the safety and measure the effectiveness of the drug in people. Once clinical trials are completed, the sponsor submits the study results in a New Drug Application (NDA) or Biologics License Application (BLA) to the FDA. This application is carefully reviewed and, if the drug is found to be reasonably safe and effective, it is approved.
How do patients get investigational drugs?
By far, the most common way that patients get investigational drugs is by taking part in a clinical trial sponsored under an IND. A patient’s doctor may suggest a clinical trial as one treatment option. Or a patient or family member can ask the doctor about clinical trials or new drugs available for cancer treatment.
Another way patients and their families can learn about new drugs being tested in clinical trials is through the National Cancer Institute’s (NCI) PDQ® database. This database contains information on a large number of ongoing studies. Individuals can search this database at http://www.cancer.gov/clinicaltrials/search, or they can call the NCI’s Cancer Information Service at 1–800–4–CANCER (1–800–422–6237). Information specialists can search the database and provide a list of trials for individuals to discuss with their doctor.
Are there other ways to get investigational drugs?
Less common ways that patients can receive investigational drugs include mechanisms such as an expanded access protocol or as 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.
The NCI’s Treatment Referral Center (TRC) protocols are one type of expanded access protocol. The NCI establishes 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 ExemptionPatients 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.
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.
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.
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.
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.
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.

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