What is compassionate drug use?

Medical professionals use the term "compassionate use" to refer to the treatment of a seriously ill patient using a new, unapproved drug when no other treatments are available. Drugs that are being scientifically tested but have not yet been approved by the United States Food and Drug Administration (FDA) are called investigational drugs. Being able to use one of these drugs when you are not in a clinical trial has many names, but is most commonly referred to as compassionate use.

Is compassionate drug use legal?

Compassionate drug use is legal, but it is tightly restricted to people who meet certain conditions. The FDA first approved investigational drugs to be used in this way for critically ill patients in 1987. There are 2 ways a drug company commonly gives access to their unapproved drug to a person who is not in a clinical trial:

• expanded access program (EAP)
• single patient access

A company sponsoring a drug in the late stages of drug development, such as Phase III clinical trials, can offer expanded access programs for patients who are not able to enroll in a clinical trial. The FDA generally approves these EAPs if the drug has shown that it works at least somewhat to treat a specific cancer in the clinical trials that are being done.

Patients who don't qualify for either clinical trials or an expanded access program (if one exists) may be able to get the unapproved new drug by applying for single patient access. In this case, the patient's doctor must first ask the drug company if the drug can be used for the patient and see if the drug company will supply it. If the company agrees, the patient's doctor works with the drug company to ask the FDA to approve the drug for use by this one patient. The FDA requires the doctor to send information about the patient, why the request is being made, the proposed treatment plan, and a signed informed consent from the patient (see our document Informed Consent for more information). The length of time it takes to get single patient access varies. But if it is an emergency, the FDA can complete the paperwork in 24 hours.

Why use drugs that aren't approved?

Most compassionate drug use is for patients with advanced disease who have tried all of the available treatment options and whose disease has not responded. Or it is used for patients with diseases that have no approved treatment options and there no clinical trials that meet the patients' needs. There must also be reason to expect that the investigational drug will help. In cases like these, the doctor may consider trying to get a new, unapproved drug for a patient who is not in a clinical trial, just to see if it will help the patient.

Are there problems with compassionate drug use?

Perhaps the biggest problem with compassionate drug use is that it is hard to get the drug. The simplest way to get an unapproved drug is through a clinical trial. But many people with life-threatening diseases either cannot find suitable clinical trials, or they live far from cancer research centers, or they are not eligible for any studies being done.

Getting the drug through expanded access programs (if offered by the drug company) or single-patient compassionate use is possible for some people. But working out single-patient compassionate use of an unapproved drug can take a lot of time and can be frustrating. For instance, the FDA and drug companies follow no single policy or process. As of 2009, no one publishes a list of all the drugs that are available through compassionate use in the United States. There is no way to make the drug company to supply the drug. There may be very limited amounts of the drug, and producing extra medicine for people who are not in clinical trials can be costly for the drug company, especially since there is a chance the drug will not be approved.

Compassionate drug use can also be very confusing. There are several programs that regulate it. Many terms and definitions are used to describe how a patient may get access to an unapproved, new drug outside of a clinical trial. Drug companies, patient advocacy groups, and the FDA all may use different terms for the same things. For example, the terms used by the FDA are defined in their regulations, while most drug companies refer to their compassionate access programs with terms that are unique to their specific programs and not in line with the FDA language.

How common is compassionate drug use?

Before a patient or group of patients is allowed to have access to an unapproved new cancer drug outside of a clinical trial, 2 things MUST be in place:

• The owner (sometimes referred to as the sponsor -- most often a drug company) of the new, unapproved drug must agree to allow the use of their drug outside of a clinical trial.

• The FDA oncology medical officer in charge of overseeing the new drug's development must grant approval before the patient or patients can start taking the drug.

Still, despite the difficulties, compassionate drug use does happen. Because actual use is not well-documented, there are no numbers or statistics on how often it is done, who is doing it, or how well it is working for patients.

What should I ask my doctor about compassionate drug use?

Here are some questions you may want to ask if your doctor is thinking about compassionate drug use at this point in your cancer treatment.

• Are there any approved treatments that I have not tried?
• Is there any evidence to support the use of this drug to treat my type of cancer?
• What makes you think this drug could help me?
• In what way do you think this drug is likely to work better than an approved drug?
• What are the known risks and benefits of treatment with this drug?
• Will the drug company give me the drug for free? If not, how is it to be paid for?
• What will I have to do to get access to this drug?
• How long do you think it will take for me to get access to this drug?

Additional resources

More information from your American Cancer Society

We have selected some related information that may also be helpful to you. These materials may be found on our Web site or ordered from our toll-free number (1-800-227-2345).

• Caring for the Patient with Cancer at Home: A Guide for Patients and Families

• Choosing a Doctor and a Hospital (also available in Spanish)

• Clinical Trials: What You Need to Know

• Oral Chemotherapy: What You Need to Know

• Understanding Chemotherapy: A Guide for Patients and Families (also available in Spanish)

National organizations and Web sites*

Along with the American Cancer Society, other sources of information and support include:

U.S. Food and Drug Administration, Center for Drug Evaluation and Research
Toll-free number: 1-888-463-6332 (This is the main FDA number. Select the option to "request information or speak with an FDA representative about FDA-regulated products or programs." You may have to continue through the menu to reach someone if you want to ask about the Emergency Use Investigational New Drug Program for Oncology Drugs )
Web site: www.fda.gov/cder/

Emergency Use Investigational New Drug Program for Oncology Drugs
Through this FDA program, patients with severe or life-threatening illnesses who are not eligible for clinical trials, and are in an urgent medical crisis, may be able to receive drugs not yet approved by the FDA to be used right away. The program is also known as the Compassionate Use IND Program.

The patient's doctor should contact the FDA with questions regarding compassionate use of an investigational drug. There are 2 direct lines for doctors to use for emergency IND requests:
1. Division of Drug Oncology Products: 301-796-2330 (Ask your doctor to call this number.)
2. Division of Biological Oncology Products: 301-796-2320 (Ask your doctor to call this number.)

National Cancer Institute
Toll-free number: 1-800-4-CANCER (1-800-422-6237)
Web site: www.cancer.gov

Pharmaceutical Research and Manufacturers of America (PhRMA)
Telephone number: 202-835-3400
Web site: www.phrma.org
Offers information about member pharmaceutical companies and drugs that are currently available in clinical trials or are being developed.

*Inclusion on this list does not imply endorsement by the American Cancer Society

No matter who you are, we can help. Contact us anytime, day or night, for information and support. Call us at 1-800-227-2345 or visit www.cancer.org.

References

Cancer Action Now. Compassionate Use and Expanded Access. Accessed at: www.canceractionnow.org/living/compassionate.php on June 17, 2009..

US Food and Drug Administration. Physician Request for a Single Patient IND for Compassionate or Emergency Use. Accessed at: www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm107434.htm on June 17, 2009.

National Cancer Institute. Access to Investigational Drugs: Q & A. Accessed at www.cancer.gov/cancertopics/factsheet/Therapy/investigational-drug-access on June 17, 2009.

Okie S. Access before Approval -- A Right to Take Experimental Drugs? N Engl J Med. 2006:355(5);437–440.

Last Medical Review: 07/09/2009
Last Revised: 07/09/2009