What is a clinical study?
Clinical studies are a way to test investigational drugs to help
develop medicines for people with diseases.
Why participate in a clinical study?
Participants in clinical studies can play a more active role in
their own health care, gain access to new research
treatments before they are widely available, and help others
by contributing to medical research.
Who can participate in a clinical study?
All clinical studies have guidelines about who can participate.
Using inclusion/exclusion criteria is an important principle of
medical research that helps to produce reliable results. The
factors that allow someone to participate in a clinical study
are called "inclusion criteria" and those that disallow
someone from participating are called "exclusion criteria."
These criteria are based on such factors as age, gender, the
type and stage of a disease, previous treatment history, and
other medical conditions.
Before joining a clinical study, a participant must qualify for
the study. Some research studies seek participants with
illnesses or conditions to be studied in the clinical study,
while others need healthy participants. It is important to note
that inclusion and exclusion criteria are not used to reject
people personally. Instead, the criteria are used to identify
appropriate participants. The criteria help ensure that
researchers will be able to answer the questions they plan to
What happens during a clinical study?
The clinical study process depends on the kind of study
being conducted. The clinical study team includes doctors
and nurses as well as social workers and other health care
professionals. They check the health of the participant at the
beginning of the study, give specific instructions for
participating in the study, and monitor the participant
carefully during the study.
Some clinical studies involve more tests and doctor visits
than the participant would normally have for an illness or
condition. For all types of studies, the participant works with
a research team. Clinical study participation is most
successful when the protocol is carefully followed and there
is frequent contact with the research staff.
What is informed consent?
Informed consent is the process of learning the key facts
about a clinical study before deciding whether or not to
participate. It is also a continuing process throughout the
study to provide information for participants. To help
someone decide whether or not to participate, the doctors
and nurses involved in the study explain the details of the
If the participant's native language is not English, translation
assistance can be provided. Then the research team
provides an informed consent document that includes details
about the study, such as its purpose, duration, required
procedures, and key contacts. Risks and potential benefits
are explained in the informed consent document. The
participant then decides whether or not to sign the
document. Informed consent is not a contract, and the
participant may withdraw from the study at any time.
What are the benefits and risks of participating in a clinical study?
According to the National Institutes of Health, there
are benefits and risks associated with participating in a
clinical trial, including:
Clinical studies that are well-designed and well-executed are
the best approach for eligible participants to:
- Obtain medical care at health care facilities during the
- Help others by contributing to medical research.
- There are risks to clinical studies.
- There may be unpleasant, serious or even life-
threatening side effects to experimental treatment.
- The experimental treatment may not be effective for
- The protocol may require more of their time and
attention than would a non-protocol treatment,
including trips to the study site, more treatments,
hospital stays or complex dosage requirements.
How is the safety of the participant protected?
The ethical and legal codes that govern medical practice also apply to clinical studies. In addition, most clinical research is federally regulated with built-in safeguards to help protect the participants.
The study follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical study progresses, researchers report the results of the study at scientific meetings, to medical journals, and to various government agencies. Individual participants' names will remain secret and will not be mentioned in these reports.
Every clinical study in the US must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that helps ensure that a clinical study is conducted in an ethical manner and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.
What should people consider before participating in a study?
People should know as much as possible about the clinical study and feel comfortable asking the members of the health care team questions about it, the care expected while in a study, and the cost of the study. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.
- What is the purpose of the study?
- Who is going to be in the study?
- Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
- What kinds of tests and experimental treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this study affect my daily life?
- How long will the study last?
- Will hospitalization be required?
- Who will pay for the experimental treatment?
- Will I be reimbursed for other expenses?
- What type of long-term follow up care is part of this study?
- How will I know that the experimental treatment is working?
- Will results of the studies be provided to me?
- Who will be in charge of my care?
What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
- Plan ahead and write down possible questions to ask.
- Ask a friend or relative to come along for support and to hear the responses to the questions.
- Bring a tape recorder to record the discussion to replay later.
Does a participant continue to work with a primary health care provider while in a study?
Yes. Most clinical studies provide short-term investigational treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can help ensure that other medications or treatments will not conflict with the protocol.
Can a participant leave a clinical study after it has begun?
Yes. A participant can leave a clinical study at any time. When withdrawing from the study, the participant should let the research team know about it, and the reasons for leaving the study.
Where do the ideas for studies come from?
Ideas for clinical studies usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical studies. During a study, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.
A protocol is a study plan on which all clinical studies are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the study; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical study, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical studies, experimental treatments are often compared with placebos to assess the experimental treatment’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or experimental treatment.
What is a control or control group?
A control is the standard by which experimental observations are evaluated. In many clinical studies, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
What are the phases of clinical studies?
Clinical studies are classified under four phases:
Phase I: Researchers test an investigational drug in a small number of people by giving it to either healthy volunteers or patients to evaluate its safety, determine a safe dosage range and identify side effects.
Phase II: The investigational drug is given to a larger number of people who have a particular disease or condition to further evaluate its safety and often to assess its effectiveness.
Phase III: Several hundred to several thousand people with the appropriate disease or condition participate in these studies. The investigational drug undergoes additional testing to further determine its safety and effectiveness, monitor side effects, and compare it to commonly used treatments or placebo.
Phase IV: After a drug is available by prescription, additional information about a drug’s risks, benefits and use may be gathered.
What is a screening process?
All potential clinical study participants must be evaluated by the study doctor to make sure they meet the eligibility requirements. Most clinical study screenings require a physical examination to assess height and weight, temperature, blood pressure, and blood and urine tests. Often, an electrocardiogram is conducted, which is a painless, noninvasive test that records the electrical activity of the heart.
It is also common for a screening to include blood and urine testing as well as other diagnostic evaluations.
What is meant by study design?
Many clinical studies are designed to prevent anyone (including study doctors, nurses, or participants) from affecting the study’s scientifically accurate results. Blinded studies are often designed so that participants do not know which study medication they are taking, so they can describe their reactions without being biased.
Single- and double-blind studies
In single-blind studies, only the participant does not know which study medication he or she was given. In a double-blind study, doctors, nurses, participants, and other healthcare staff are not informed as well.
Some studies are placebo-controlled, which means that one group of study participants takes a placebo while another group takes an investigational medication. Other studies are active-controlled, where one group of participants receives a recognized effective treatment and the other group receives the investigational medication.
In a medical emergency, it is always possible to find out which study medication group a participant has been assigned to, and to determine which study medication the participant has been taking.
In open-label studies, both the researchers and participants know which drugs are being used.
Randomization occurs when a participant is selected by chance (not by choice) to join a study treatment group. This is similar to being chosen by flipping a coin. For example, if an investigational medication is being compared to a currently available drug or placebo (an inactive drug), there may be 2 study groups. One group of participants may receive the investigational medication, while the other would receive a placebo.
The standard drug may be covered with a capsule or the placebo will be made to look the same as the investigational medicine so that study participants are unable to tell the difference between the drugs.
Will there be any cost to me if I participate in a clinical study?
Study medications, procedures and tests, as well as visits, may be provided without cost to participants. Information about costs is included in the informed consent form and the study doctor will answer any questions about cost that you may have.
Whom can I contact regarding clinical studies sponsored by Bristol-Myers Squibb?
I am not eligible to participate in any clinical studies sponsored by Bristol-Myers Squibb – where can I learn more about other studies?
Why can't I find a study listing I previously saw on Bristol-Myers Squibb Study Connect?
When a study has completed enrolling participants, additional people are not permitted to enter the study. At that time, the study listing is automatically removed from Bristol-Myers Squibb Study Connect. Studies that have completed enrollment can be found on the Bristol-Myers Squibb Clinical Trials Transparency website at http://www.bms.com/clinical_trials/Pages/home.aspx or visit www.ClinicalTrials.gov.