| Frequently Asked Questions (FAQ)
is a clinical trial?
A clinical trial is a scientific experiment that is done with people
who volunteer to participate. Like any scientific experiment, the
conditions of a clinical trial are carefully controlled and closely
monitored. For example, study volunteers must fulfill enrollment criteria
in order to be eligible for participation. The criteria are determined
with consideration to the validity of the research results and the
safety of the volunteers.
In general, the purpose of clinical trials is to evaluate the safety,
effectiveness or best dosage schedule of experimental drugs or medical
devices in persons having a specific disease or health condition.
New drugs have to be evaluated before they are given to the general
population. The safest and most effective way to evaluate drugs is
in the format of a clinical trial.
What is this I've heard about research volunteers
being blinded and taking placebos?
Often a clinical trial is designed so that neither the study volunteer
nor the medical staff knows if the study volunteer is actually receiving
the experimental drug. This would be called a "blinded study." A study
is blinded in order to eliminate the possibility of unfairness in
the assessment of the effectiveness of the study drug.
In a blinded study, the study volunteers are randomized, that is they
are randomly assigned to receive the study drug or something that
looks like the study drug. This might be a standard drug that has
been reformulated to look like the study drug, or it might be a placebo.
A placebo looks like a drug but actually does not contain
any active substance.
How can I be sure that I am assigned to the
study drug that I want?
Well, you actually can't. The study group you are assigned to is decided
randomly, like flipping a coin or drawing a card. Often, a computer
program randomizes patients. The study doctor has no influence as
to which study group you are assigned within a given clinical trial.
Would I be the first person ever to take the experimental drug?
In order for a drug to be approved by the Food and Drug Administration,
it must go through different phases of drug development. You can find
out what phase your clinical trial is in by asking the study doctor
or any member of the clinical trial staff. There are either Phase
I, II, III, or IV clinical trials.
A Phase I
trial involves giving the experimental treatment to healthy people
in order to generate preliminary information on safety.
A Phase II
trial is usually the second stage in testing a new drug in human beings.
Generally, the phase II study is carried out on people having the
disease or condition for which the experimental treatment has been
developed. The main purpose is to provide preliminary information
on effectiveness of the drug and to provide additional information
on safety and side effects.
Usually the Phase
III trial is the third and final stage in testing
a new drug in people. The Phase III trial is concerned with assessment
of dosage effects, effectiveness, and safety and aimed at providing
information for labeling in relation to use by the general public.
Once this phase is completed the drug manufacturer may request permission
to market the drug for the disease or condition for which it has been
A Phase IV
trial is performed after the drug has been FDA approved for and is
typically done under circumstances approximating real world conditions
instead of carefully controlled conditions. Often it measures the
long-term effects of a new drug.
Does anyone monitor clinical trials?
The federal government has in-depth regulations regarding the protection
of research study volunteers. The Food and Drug Administration
(FDA) is a federal agency with which large drug companies
must comply in order to have their drugs FDA approved and licensed
for sale to the general public.
According to the FDA, any drug must pass rigorous safety tests in
animals before it can be approved for clinical trials. Once the clinical
trial begins, the FDA closely monitors its progress. In addition to
approval by the FDA, every clinical trial requires approval and monitoring
by Institutional Review Board (IRB). The IRB is a committee
made up of doctors and community representatives. The purpose of the
IRB is to review research and determine if the rights and welfare
of research volunteers are adequately protected.
The study doctor is directly responsible for your safety.
He or she will be watching for any abnormalities in test results.
Why would someone want to join a clinical trial?
Each clinical trial is different, therefore the benefits vary. Some
clinical trials offer volunteers the opportunity to try treatments
that appear to be promising but are not available yet to the general
public. Often clinical trials have rigorous health monitoring. Patients
meet with the study staff on a regular basis and as a result, become
motivated to take better care of themselves. For example, many clinical
trials involving patients with diabetes may offer dietary counseling
and blood sugar monitoring. Study volunteers find that they are motivated
to watch their diet more carefully when they have to report what they
eat at their next visit to the research center. And of course, if
you are a volunteer in a clinical trial, you are giving of yourself
and helping society make medical progress.
What should I be aware of as I consider joining a clinical trial?
Before you enroll in a study you will be given a consent form
to read and sign. This form gives you all the information about the
clinical trial you are considering. Be sure that you have read the
consent form thoroughly. Some people read it two or three times. If
something is not clear on the consent form, or you want additional
information, be sure to ask all of your questions to the study doctor
or the study coordinator, whoever reviews the consent form with you.
Every clinical trial is different, and the benefits and risks of participating
vary. The consent form is your best source of information.
What if I read something on the consent form
that I don't like?
Although previously you might have talked to the study doctor or study
nurse, you might read the consent form and find out something about
the clinical trial that you didn't expect. Perhaps it never occurred
to you to ask about the risks of a certain procedure, or a placebo
period. Maybe you didn't know that there would be an overnight study
visit, and maybe any one of these details is enough for you to decide
that you don't want to join after all. You can thank the study staff
for their time and efforts, but at the same time politely decline
to sign the consent form and leave the research center. There will
be no hard feelings.
Will it cost me anything to participate? Will I be paid?
In general, there is no cost to volunteers or their insurance agencies
for any research related costs. Often insurance agencies are billed
for procedures that would be done regardless of participation in the
research study. Some studies offer to pay the volunteer for their
participation. This is usually a modest amount meant to cover travel
and parking expenses and any inconvenience the study might cause you.
For example, studies that require overnight visits to the research
center offer a more generous remuneration than other studies.
Meinert, Curtis L. Clinical Trials Dictionary: Terminology
and Usage Recommendations. The Johns Hopkins University; School
of Hygiene and Public Health; Center for Clinical Trials, © 1996.