The Facts about Clinical Trials

A clinical trial is a research study in human volunteers to answer specific health questions. Choosing to participate in a clinical trial is an important personal decision. It is often helpful to talk to a physician, family members, or friends about deciding to join a trial.

Ideas for clinical trials 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 trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.

Control Groups and Placebos
A control is the standard by which experimental observations are evaluated. In many clinical trials, 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.

A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, 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.

Participation
Before joining a clinical trial, a participant must qualify for the study. All clinical trials have guidelines about who can participate. These guidelines are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. The criteria are used to identify appropriate participants and keep them safe. The guidelines help ensure that researchers will be able to answer the questions they plan to study.

The Clinical Trial Process
The clinical trial process depends on the kind of trial being conducted. The clinical trial 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 trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed.

Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the study plan, called a protocol, is carefully followed and there is frequent contact with the research staff.

Each clinical trial is based on its own specific protocol. This 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 trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, 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.

Participants continue to work with their primary health care provider while in a trial. Most clinical trials provide short-term 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 ensure that other medications or treatments will not conflict with the protocol.

Types of Clinical Trials

Treatment trials test experimental 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.

Benefits and Risks of Participating in a Clinical Trial

Benefits: Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

• Play an active role in their own health care.
• Gain access to new research treatments before they are widely available.
• Obtain expert medical care at leading health care facilities during the trial.
• Help others by contributing to medical research.

Risks: There are risks to clinical trials:

• There may be unpleasant, serious or even life-threatening side effects to experimental treatment. Side effects are any undesired actions or effects of the experimental drug or treatment. Experimental treatments must be evaluated for both immediate and long-term side effects.
• The experimental treatment may not be effective for the participant.
• 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.

Meeting with the Research Coordinator or Doctor
People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial.

• 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.

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 trial affect my daily life?
• How long will the trial last?
• Will hospitalization be required?
• Who will pay for the experimental treatment?
• Will I be paid 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 trials be provided to me?
• Who will be in charge of my care?

Informed Consent
Informed consent is the process of learning the key facts about a clinical trial 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 trial explain the details of the study. If the participant's native language is not English, translation assistance can be provided.

The research team provides an informed consent document that includes details about the study, such as its purpose, length, risks, and potential benefits. The participant then decides whether or not to sign the document. Informed consent is not a contract; a participant can leave a clinical trial at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.

The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built-in safeguards to protect the participants. The trial follows a carefully controlled protocol that details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial 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 trial 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 ensures that a clinical trial is ethical 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.

Sponsorship
Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veteran's Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.

To learn about current clinical trials, see the Clinical Trials website of the US National Institutes of Health.

Each year, Medical College of Wisconsin physicians care for more than 180,000 patients, representing nearly 500,000 patient visits. Medical College physicians practice at Children's Hospital of Wisconsin, Froedtert Memorial Lutheran Hospital, the Milwaukee VA Medical Center, and many other hospitals and clinics in Milwaukee and southeastern Wisconsin.