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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).
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
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