+ -Text Size

Compassionate Drug Use

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 tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigational drugs. These drugs are generally available only to people who are taking part in a clinical trial (a research study that is testing the drug). 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’s 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. For people who aren’t in clinical trials, there are 2 ways a drug company can allow use of their unapproved drug: through expanded access programs (EAPs) or through single patient access.

Expanded 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. (See Clinical Trials: What You Need to Know to learn more about the phases of clinical trials.)

The FDA generally approves these EAPs if the drug has shown that it works at least somewhat to treat cancer in the clinical trials that are being done. This can allow a lot of people access to the unapproved drug, as long as they meet the requirements of the EAP.

Single patient access

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 on this). The length of time it takes to get single patient access varies. But if it’s an emergency, the FDA can complete the paperwork in 24 hours.

Who might benefit from using unapproved drugs?

According to guidelines from the National Cancer Institute, most compassionate drug use is for patients who meet all of these conditions:

  • Have advanced disease
  • Have used standard treatments and they have not worked
  • Are not eligible for any clinical trial that’s in progress
  • Have no other treatment options
  • Have a type of cancer for which there’s reason to expect the investigational drug will help
  • Are likely to have benefits that outweigh the risks involved

In a case like this, the doctor may consider trying to get a new, unapproved drug for a patient to see if it will help.

Is it difficult to get drugs for compassionate use?

Perhaps the biggest problem with compassionate drug use is that it’s 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 can’t find suitable clinical trials, live far from cancer research centers, or are not eligible for any studies being done.

Getting the drug through expanded access programs (if one is offered by the drug company) or single-patient compassionate use is possible for some people. But going through all the steps needed to get single-patient compassionate use of an unapproved drug can be frustrating and take a lot of time. For instance, drug companies have different policies and processes. And there’s no way to force 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 when there’s a chance the drug might never 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 drug outside of a clinical trial. Drug companies, patient advocacy groups, and the FDA all may use different terms for the same things. The FDA provides definitions for the terms they use in their regulations. But most drug companies use different terms that are unique to their specific compassionate access programs.

Another big problem is cost. Some drug companies will supply the drug for free, but others charge patients. Most insurance companies will not pay for investigational drugs. There may also be other costs, such as the clinic’s cost of giving the drug and monitoring your response, that might not be covered by your health insurance.

How common is compassionate drug use?

Before a patient or group of patients can get an unapproved new cancer drug outside of a clinical trial, 2 things MUST happen:

  • 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 approve the use of the drug for that person or group.

Still, despite these hurdles, compassionate drug use does happen. Because actual use is not well-documented, there are no numbers or statistics on how often it’s done, who’s doing it, or how well it’s 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 haven’t 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 other costs will I have to pay to get the drug? Will my insurance cover any costs?
  • 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?

To learn more

More information from your American Cancer Society

We have selected some related information that may also be helpful to you. These materials may be read on our website, www.cancer.org, or ordered from our toll-free number, 1-800-227-2345.

Clinical Trials: What You Need to Know (also in Spanish)

A Guide to Chemotherapy (also in Spanish)

Oral Chemotherapy: What You Need to Know

Caring for the Patient With Cancer at Home: A Guide for Patients and Families (also in Spanish)

National organizations and web sites*

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

US 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 an FDA-regulated product or program.” You may have to continue through the menu to reach someone to ask about the Emergency Use Investigational New Drug Program for Oncology Drugs

Website: www.fda.gov/Drugs/default.htm

    Emergency Use Investigational New Drug Program for Oncology Drugs
    Through this FDA program, patients with severe or life-threatening illnesses who are in an urgent medical crisis but aren’t eligible for clinical trials 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.

    Your 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, depending on the type of product:

      1. Division of Drug Oncology Products: 301-796-2330 (Ask your doctor to call this number about an investigational drug.)
      2. Division of Biological Oncology Products: 301-796-2320 (Ask your doctor to call this number about any product that uses cells or tissue extracts.)

National Cancer Institute
Toll-free number: 1-800-4-CANCER (1-800-422-6237)
TTY: 1-800-332-8615
Website: www.cancer.gov

    Has accurate, up-to-date information about cancer for patients, their families, and the general public; also can help you find specific clinical trials for your situation

Pharmaceutical Research and Manufacturers of America (PhRMA)
Telephone number: 202-835-3400
Website: 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 July 3, 2013.

US Food and Drug Administration. Access to Investigational Drugs Outside of a Clinical Trial (Expanded Access). Accessed at www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm on July 3, 2013.

US Food and Drug Administration. Physician Request for an Individual Patient IND under Expanded Access for Non-emergency or Emergency Use. Accessed at www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm107434.htm on July 3, 2013.

National Cancer Institute. Access to Investigational Drugs. Accessed at www.cancer.gov/cancertopics/factsheet/Therapy/investigational-drug-access on July 3, 2013.

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


Last Medical Review: 07/09/2013
Last Revised: 07/09/2013