Trials: Is one right for me?
If you are living with a serious or chronic (long-term) condition,
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 (5) types of
Treatment trials test new treatments, new combinations of
drugs, or new approaches 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 changes.
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
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 (2)
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
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. 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 approval.
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 trial.
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 U.S. Food
and Drug Administration (FDA) as described above.
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
contribute to medical research that may one day benefit many
Many costs may be covered by a federal program or pharmaceutical
Drawbacks to participating in a clinical trial include the
Unpleasant, serious, or even life-threatening side effects
Receiving a placebo or standard treatment rather than the
investigational treatment (Volunteers agree to this risk when they
consent to participate in the study.)
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 factors
If I participate in a clinical trial, will I have to be away from
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. Most dermatologic
illnesses do not require a hospital stay unless 24-hour monitoring
is a condition of the trial.
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 trial.
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
3. 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, you
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
Search the Internet. Visit the web sites of medical centers,
pharmaceutical companies, the Food and Drug Administration (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
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developed by the American Academy of Dermatology