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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 are called investigational
drugs. Access to 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, including 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 programs if the drug has shown
some effectiveness against a specific cancer in the clinical trials
that are being done.
Patients who are not eligible 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 request permission for access to
the drug from the drug company. 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 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 are compassionate drugs
used?
Compassionate drug use is mostly obtained for patients with
advanced disease who have tried all of the available treatment options
and whose disease has not responded, or for patients with diseases that
have no approved treatment options and no clinical trials that meet the
patients' needs. There must also be reason to expect some benefit from
the investigational drug. In cases such as these, the doctor may
consider trying to get a new, unapproved drug for a patient who is not
in a clinical trial.
What problems are associated
with compassionate drug use?
Perhaps the biggest problem with compassionate drug use is
that it is hard to get. The simplest way to get access to 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.
Expanded access programs (if offered by the drug company) or
access through single-patient compassionate use is possible for some.
But working out single-patient compassionate use of an unapproved drug
is often time-consuming and frustrating. For instance, there is no
single policy or process followed by the FDA and drug companies. As of
2007, no one publishes a list of all the drugs that are available
through compassionate use in the United States. There is no way to
require the drug company to supply the drug. Producing extra medicine
for people who are not in clinical trials can be expensive 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. There are many terms and definitions
that 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 the 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, and how well it
is working for patients.
What questions 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 be more
effective than the use of 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-ACS-2345).
National Organizations and Web
Sites*
In addition to the American Cancer Society, other sources of
patient information and support include:
Emergency Use Investigational New Drug Program for Oncology Drugs
Telephone: 888-463-6332
Description of Service: Through this 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.
A patient's doctor should contact the FDA with questions regarding
compassionate use of an investigational drug. The program only allows
for doctors to make emergency IND requests. There are two direct lines
(for doctors' use only):
- Division of Drug Oncology Products: (301) 796-2330 (Ask
your doctor to call this number)
- Division of Biological Oncology Products: (301) 796-2320
(Ask your doctor to call this number)
National Cancer Institute
Telephone: 1-800-4-CANCER (1-800-422-6237)
Internet Address: www.cancer.gov
Pharmaceutical Research and Manufacturers of America (PhRMA)
Telephone: 202-835-3400
Internet: www.phrma.org
Description of Service: Provides information about member pharmaceutical companies and drugs that are currently available in clinical trials or are under development.
US Food and Drug Administration, Center for Drug Evaluation
and Research
Telephone: 1-888-463-6332
Internet Address: www.fda.gov/cder/
*Inclusion on this list does not imply endorsement
by the American Cancer Society.
The American Cancer Society is happy to address almost any
cancer-related topic. If you have any more questions, please call us at
1-800-ACS-2345 at any time, 24 hours a day.
References
Compassionate Use and Expanded Access. Available at http://www.canceractionnow.org/living/cat_compassionate_use_and_expanded_access.php. Accessed February 7, 2007.
National Cancer Institute. Access to Investigational Drugs: Q & A. Available at http://www.cancer.gov/cancertopics/factsheet/Therapy/investigational-drug-access. Accessed 3/19/07.
Okie S. Access before Approval -- A Right to Take Experimental Drugs? N Engl J Med. 2006:355(5);437-440.
Revised: 03/30/2007
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