Access to Investigational Drugs: Questions and Answers
- 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
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
- 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
- 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
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
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
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
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
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:
What should patients do if they are interested in receiving
an investigational drug through a special exception or expanded access mechanism?
- 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
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
What are the costs involved in receiving an investigational
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.
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.
How can patients find out more information about a specific
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 NCI's Cancer Information Service at 1–800–4–CANCER (1–800–422–6237).
What other resources are available on this topic?
The following list of resources may be helpful:
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Related NCI materials and Web pages:
For more help, contact:
- NCI's Cancer Information Service
(toll-free): 18004CANCER (18004226237)
LiveHelp® online chat: https://cissecure.nci.nih.gov/livehelp/welcome.asp
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