When you or a loved one are
diagnosed with cancer, it is
important to know all of the
treatment options available in order
to make the best decision about your
cancer treatment. This may include
clinical trials, which are research
studies designed to evaluate new
cancer treatment options. Clinical
trials test the safety and
effectiveness of treatments, many of
which are only available through
participating in a clinical trial.
Trials evaluate new anti-cancer
drugs, unique approaches to surgery
and radiation therapy, and new
combinations of treatments. In the
United States, the Food and Drug
Administration (FDA) oversees the
conduct of clinical trials.
What does the FDA do?
The FDA is a government agency that
is responsible for making certain
that the food we eat and the drugs
we take are safe. The FDA does not
make drugs or directly test drugs to
determine if they are safe and
effective. The FDA's role is to
oversee the research conducted by
pharmaceutical companies, university
research centers, and physicians to
make certain that federal
regulations governing research are
being followed.
The FDA
requires that the drug company's
plan must be reviewed by community
research review board (IRB), and
that patients participating in the
clinical trial are informed about
the trial and consent to
participate. Once the drug company
has completed its clinical trials,
the data are tabulated and submitted
to the FDA in an application known
as a New Drug Application (NDA). The
FDA evaluates the outcomes reported
in the NDA and determines whether
the new drug will be approved and
made available to patients in the
United States. In order to be
approved, the drug must be safe and
effective.
Can I
get a drug before it is approved by
the FDA?
Until a drug
receives FDA approval, it cannot be
sold and the drug company may only
provide it to patients through
clinical trials. Furthermore, each
clinical trial has specific criteria
that patients must meet to be
included. Occasionally, a cancer
patient who is not eligible for a
clinical trial may receive a
promising unapproved drug, if the
patient's doctor, the drug company,
and FDA each agree. The FDA's
primary interest is helping to
ensure that the drug company's
research will not subject cancer
patients in the clinical trial to
undue risks. The FDA drug review
process guarantees that the risks
and benefits of a cancer drug have
been carefully considered before it
is approved and helps to ensure the
public that marketed drugs are safe
and effective.
How can
I learn more about a drug that has
been approved?
The FDA
requires that all drugs have an
information document for healthcare
providers and consumers called a
"package insert." This document is a
summary of the essential scientific
information needed for the safe and
effective use of the drug. You can
ask your doctor for this
information. Also, most package
inserts are available on the
internet. A package insert typically
includes the following information:
- Chemical structure -
Information about how the body
absorbs, distributes, metabolizes,
and excretes the drug - Results
from some clinical trials - What
specific circumstances the drug is
used for - Dosing and
administration schedules - Side
effects - Contraindications
It is important to understand
that once it has been determined
that a drug is safe and it is
approved by the FDA, physicians
often use the drug for the treatment
of medical conditions other than the
specific condition that the FDA has
approved it for.
Are all
clinical trials the same?
Development of new anticancer
drugs and treatment strategies
occurs in four phases. Each phase is
designed to determine specific
information about the potential new
treatment such as its risks, safety,
and effectiveness compared to
standard therapy. The hope is that
the new therapy will be an
improvement over the previous
standard therapy.
Phase I
Trials: This phase is
probably the most important step in
the development of a new drug or
therapy. Phase I therapy may produce
anti-cancer effects and a small
number of patients may benefit,
however, the primary goals of this
phase are to determine safety
issues, which include:
- The
maximum tolerated dose of the
treatment, - The manner in which
the drug works in the body, - The
toxic side effects related to
different doses, and - Whether
toxic side effects are reversible.
Phase I trials usually involve a
small number of patients for whom
other standard therapies have failed
or no known alternative therapy is
available. Upon completion of phase
I trials, the information that has
been gathered is used to begin phase
II trials.
Phase
II Trials: Phase II trials
are designed to determine the
effectiveness of the treatment in a
specific patient population at the
dose and schedules determined in
phase I. These trials usually
require a slightly higher number of
patients than phase I trials. In
general, all of the patients
participating in a phase II trial
will receive the treatment that is
being investigated. Drugs or
therapies that are shown to be
active in phase II trials may become
standard treatment or be further
evaluated for effectiveness in phase
III trials.
Phase
III Trials: Phase III trials
compare a new drug or therapy with a
standard therapy in a randomized and
controlled manner in order to
determine proof of effectiveness.
Phase III trials require a large
number of patients to measure the
statistical validity of the results
because patient age, sex, race, and
other unknown factors could affect
the results. To obtain an adequate
number of patients, several
physicians (investigators) from
different institutions typically
participate in phase III clinical
trials.
Phase
IV Trials: Once the drug or
treatment is approved and becomes
part of standard therapy, the
manufacturer of the drug may elect
to initiate phase IV trials. This
phase includes continued evaluation
of the treatment effectiveness and
monitoring of side effects as well
as implementing studies to evaluate
usefulness in different types of
cancers.
There is currently
no single source of all clinical
trials. The following are clinical
trial resources that patients may
wish to visit: NCI's Web site at
www.cancer.gov
ECancerTrials National
Clinical Trials Database
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