Access to 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). 

1. 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 

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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.

2. 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, 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. 

3. 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 
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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 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. 

4. 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. 

5. 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. 

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. 

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.) 
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7. 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 

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 which outlines the known risks and benefits of the treatment, as well 

as the rights and responsibilities of the patient.

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. Before beginning treatment, patients should check with 

their insurer about coverage of these costs. 

 

10. 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.

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 NCI’s 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: 

• The NCI’s Web site has a feature titled Understanding the Approval Process for 

New Cancer Treatments: Special Needs, which can be found at 

http://www.cancer.gov/clinicaltrials/learning/approval-process-for-cancerdrugs/page4 on the Internet. 

• Another NCI Web site titled Developing Cancer Therapies can be found at 

http://ctep.cancer.gov on the Internet. 

• The FDA’s Center for Drug Evaluation and Research Web site has Oncology 

Tools, which contains a variety of information related to cancer, including a 

section on access to unapproved drugs. This resource is at 

http://www.fda.gov/cder/cancer/index.htm on the Internet. 

# # # 

Related NCI materials and Web pages: 

• National Cancer Institute Fact Sheet 1.4, NCI’s Clinical Trials Cooperative 

Group Program (http://www.cancer.gov/cancertopics/factsheet/NCI/clinicaltrials-cooperative-group) 

• National Cancer Institute Fact Sheet 1.17, The NCI/VA Agreement on Clinical 

Trials: Questions and Answers

(http://www.cancer.gov/cancertopics/factsheet/NCI/VA-clinical-trials) 

• National Cancer Institute Fact Sheet 1.22, Cancer Clinical Trials at the National 

Institutes of Health Clinical Center: Questions and Answers

(http://www.cancer.gov/cancertopics/factsheet/NCI/clinical-center) 

• National Cancer Institute Fact Sheet 2.11, Clinical Trials: Questions and 

Answers (http://www.cancer.gov/cancertopics/factsheet/Information/clinicaltrials) 

• National Cancer Institute Fact Sheet 8.14, More Choices in Cancer Care: 

Information for Beneficiaries on Medicare Coverage of Cancer Clinical Trials

(http://www.cancer.gov/cancertopics/factsheet/support/medicare) 

• If You Have Cancer and Have Medicare ... You Should Know About Clinical 

Trials (http://www.cancer.gov/clinicaltrials/resources/medicare-and-cancer-trials)

• Taking Part in Cancer Treatment Research Studies

(http://www.cancer.gov/clinicaltrials/Taking-Part-in-Cancer-Treatment-ResearchStudies)
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For more help, contact: 

NCI’s Cancer Information Service 

Telephone (toll-free): 1–800–4–CANCER (1–800–422–6237) 

TTY (toll-free): 1–800–332–8615

LiveHelp® online chat: https://cissecure.nci.nih.gov/livehelp/welcome.asp

This fact sheet was reviewed on 11/19/04