Clinical Trials: Is One Right for Me?
If you have been diagnosed
with skin cancer, you may be wondering if a clinical trial is an
option worth considering. After all, participating in a clinical
trial can place you in the care of some of the nation’s top
physicians, and you may find a treatment that works. Naturally, you
also are concerned about the risks involved. To help you decipher
the jargon and make an informed decision, the following provides
basic facts about clinical trials in a Q&A format and links to other
sources that may help.
What is a clinical trial?
A clinical trial is a medical investigational study in which humans
are observed and/or treated.
The National Institutes of Health (NIH) recognizes five types of
Treatment trials test new
treatments, such as a new medication or new combination of
medications, or a new approach to surgery or radiation therapy.
Prevention trials look for
better ways to prevent disease in people who have never had the
disease or to prevent a disease from returning. These approaches may
include medicines, vitamins, vaccines, minerals, or lifestyle
Diagnostic trials are conducted
to find better tests or procedures for diagnosing a particular
disease or condition.
Screening trials test the best
way to detect certain diseases or health conditions.
Quality of Life trials (or
Supportive Care trials) explore ways to improve comfort and the
quality of life for individuals with a chronic illness.
Treatment trials are generally
conducted in phases. The other types of clinical trials may not have
phases. This is what happens during each phase:
Phase I – This is the first time humans receive the new drug
or other treatment under investigation. Phase I occurs after the
basic science, laboratory, and animal studies indicate that an
approach, medicine, etc. may be safe and effective for treatment of
a specific condition. The NIH defines a
Phase I study as a study in which “researchers evaluate what dose is
safe, how a new agent should be given (by mouth, injected into a
vein, or injected into the muscle), and how often. Researchers watch
closely for any harmful side effects.”
The number of people who participate in a Phase I study is small,
generally 20 to 80 volunteers. A treatment may be recommended for
Phase II studies after researchers determine the highest dose that
can be taken with acceptable side effects.
Phase II – During this phase, the safety and effectiveness
are studied in volunteers who have the condition for which the
treatment is being considered. Several hundred volunteers may be
enrolled in a number of Phase II studies. Volunteers are selected
via the criteria outlined by the researchers in the study protocol
(study plan). If Phase II trials are satisfactorily completed, Phase
III studies are conducted.
Phase III – In Phase III, the investigational treatment is
compared to a standard therapy currently used to treat the condition
and the overall benefits and risks of the treatment under
investigation are evaluated. The data gathered from Phase III trials
provides a better understanding of the treatment’s effectiveness,
benefits and potential side effects.
During a Phase III trial, volunteers are divided into two groups:
Control group – The volunteers
in the control group do not receive the investigational treatment.
They receive either a currently accepted treatment or a placebo.
Study group - The volunteers in
the study group receive the treatment under investigation.
Volunteers are randomly assigned to a
control group or a study group. They do not get to choose which
group they want to join and are not told which treatment they
receive. Assigning volunteers by chance ensures that the groups will
be similar and the treatments can be compared objectively.
Since the volunteers do not know if they are receiving the
investigational treatment, standard treatment, or a placebo, they
are said to be “blinded.” This ensures unbiased results. The
researchers also may be “blinded,” (i.e., they do not know which
treatment each participant receives). When both the volunteers and
researchers are “blinded,” the study is termed “double-blinded.”
If you enter a Phase III study, you will likely be part of a
double-blinded, randomized control trial (RCT). In a double-blinded
RCT, volunteers are assigned by chance to each group and both the
volunteers and the researchers are blinded. Using chance to assign
volunteers allows the treatments and results to be objectively
compared. A double-blinded RCT is the ideal study for scientifically
demonstrating the effect of a therapy. In fact, pharmaceutical firms
often sponsor these types of studies to gather the data needed to
submit a drug to the U.S. Food and Drug Administration (FDA) for
A large number of volunteers are sought for Phase III clinical
trials. Several hundred to several thousand volunteers may be
enrolled in a Phase III trial. These trials are conducted over a
period of years in hospitals, medical centers, and private
physicians’ offices across the United States. They also are
conducted by large medical centers and government-supported
organizations, such as the NIH and the Veterans Administration.
Once Phase III testing is complete, a pharmaceutical company can
submit a drug to the FDA. The FDA uses the data to help determine
whether or not to approve the drug.
Phase IV – Phase IV trials are not always conducted. When
they are, they usually take place after the treatment receives FDA
approval and is available to the public. During a Phase IV trial,
long-term safety and effectiveness, side effects that may not have
been apparent during Phase III, and optimal usage are evaluated.
Hundreds to thousands of volunteers may participate in a Phase IV
What happens to the data collected during a clinical trial?
Data from all types of clinical trials, not just treatment trials,
is analyzed by the primary investigators who draw science-based
conclusions. The data and conclusions are then documented in papers.
These papers are usually referenced in a data bank, such as MedLine,
the data retrieval system of the National Library of Medicine.
Papers can be retrieved, usually for decades, by physicians,
non-physician scientists, and anyone else who is interested.
Researchers also report their findings at medical meetings, to
scientific journals and to various government agencies. Articles may
be written that appear in scientific journals, such as the
Journal of the American Academy of Dermatology. At the American
Academy of Dermatology’s annual and summer meetings, researchers
present their findings and allot time for fellow dermatologists to
ask questions. The goal is to provide information that helps
Data collected about new drugs also is submitted to the FDA as
When a study is deemed well-designed and its results are
scientifically and statistically valid, it may be referenced in
future studies. It also is possible that future studies may seek to
build upon or critically examine its results. Such a study becomes a
building block in a constantly evolving information base.
Who is eligible to volunteer for a clinical trial?
Clinical trials have eligibility guidelines for volunteers. This
ensures that the study’s questions can be effectively answered. The
eligibility guidelines are published in a public notice. Eligibility
criteria include age, gender, and medical history. Within the
eligibility guidelines, researchers write “inclusion criteria” and
“exclusion criteria.” Inclusion criteria are factors that the
researchers seek, such as having a certain medical condition.
Exclusion criteria identify factors that preclude a volunteer from
participating. For example, the study may exclude people who are
already participating in another clinical trial.
What are the major benefits and drawbacks of being in a clinical
The primary benefits of participating in a clinical trial are:
Gaining access to new treatment before
it is widely available
Possibility of being placed in the care
of some of the nation’s top physicians
Helping contribute to medical research
that may one day benefit many patients
Many costs may be covered by a federal
program or pharmaceutical sponsor
Drawbacks to participating in a
clinical trial include the possibility of:
Unpleasant, serious, or even
life-threatening side effects
Receiving a placebo or standard
treatment rather than the investigational treatment (Volunteers
agree to this possibility when they consent to participate in the
Receiving treatment that is ineffective
Assuming responsibility for many costs
(Costs are not always covered by health insurance or a sponsor.)
A greater time commitment since
participation may require more of the volunteer’s time than a
standard treatment due to trips to the study site, possible need for
more frequent observation or treatment, hospital stays, and other
If I participate in a clinical
trial, will I have to be away from home?
This depends on a number of factors, including the study
protocol, the severity of your illness, and where a trial is
conducted. When patients are very ill or 24-hour monitoring is
required, the trial may be conducted at one, or even a few, large
medical centers and require the volunteers to remain at the center
during the trial. Other trials may have many dispersed locations and
only accept volunteers who can get from location to location.
How long does a clinical trial last?
Duration varies and depends on the time required to test the
study hypothesis adequately. A clinical trial can last a few days,
several weeks, or years. In a randomized control trial, a volunteer
may be asked to return for follow-up testing for several weeks or
months after the treatment phase is over. The volunteer’s time
commitment will be specified in the public notice for the clinical
Is the volunteer liable for any of the costs associated with a
This varies from trial to trial. Health insurance may not pay
for some, or all, of the costs of treatment received during a
clinical trial since some insurance plans regard clinical trials as
"investigational" treatment. Investigational treatments are
typically not a covered cost. Trials underwritten by a
pharmaceutical firm usually pay some, but not all, costs of
treatment. In such a clinical trial, the volunteer may be liable for
some expenses, such as those for tests. Before entering a clinical
trial, a prospective volunteer should learn what costs will be
covered by the sponsor and health insurance as well as what costs
will become the volunteer’s responsibility.
How can I find out what will be required during a clinical trial?
This, too, is published in the public notice. Each and every
clinical trial must adhere to federal and institutional rules and
regulations. Federal regulations, and the rules of each institution
involved in a clinical trial, require that:
Every trial must be approved by an
Institutional Review Board (IRB) to assure that volunteer rights and
safety are adequately protected.
A volunteer must give “informed
consent.” Informed consent means that the volunteer agrees to
participate in the trial under conditions explained by the
investigators. However, the volunteer is not legally bound to remain
in the trial, and may leave at any time without penalty. It is hoped
that volunteers will remain in the trial so that study results are
not skewed by dropouts.
Informed consent requires the study’s researchers to thoroughly
inform volunteers about:
The study’s plan
Treatment to be given during the trial
Tests that will be carried out
Follow-up procedures after the trial
If the study is blinded and
placebo-controlled, the volunteer must be told that he or she may
receive a placebo or the standard treatment rather than the
Once a potential volunteer knows what
will happen during a clinical trial, the person is strongly urged to
carefully think about whether or not to participate and to discuss
the study with family and physicians before making a final decision.
How do I find clinical trials?
To find clinical trials conducted by an authoritative sponsor,
Talk with your dermatologist about
participating in a clinical trial. Ask if a clinical trial is a
treatment option you should consider, and if so, which one(s) would
be best for you.
Contact large medical centers and
pharmaceutical firms regarding any trials they may be conducting or
planning in the area of your interest.
Search the Internet. Visit the web
sites of medical centers, pharmaceutical companies, the FDA, and
government-sponsored research centers, such as the National
Institutes of Health (NIH). Information on clinical trials sponsored
or approved by the NIH and FDA is available at
content solely developed by the American Academy of Dermatology