What is a clinical trial?

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Clinical trials are research studies performed in people that are aimed at evaluating a medical, surgical, or behavioral intervention. They are the primary way that researchers find out if a new treatment, like a new drug or diet or medical device (for example, a pacemaker) is safe and effective in people. Often a clinical trial is used to learn if a new treatment is more effective and/or has less harmful side effects than the standard treatment.

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Other clinical trials test ways to find a disease early, sometimes before there are symptoms. Still others test ways to prevent a health problem. A clinical trial may also look at how to make life better for people living with a life-threatening disease or a chronic health problem. Clinical trials sometimes study the role of caregivers or support groups.

 

 

Why participate in a clinical trial?

 

There are many reasons why people choose to join a clinical trial. Some join a trial because the treatments they have tried for their health problem did not work. Others participate because there is no treatment for their health problem. By being a part of a clinical trial, participants may find out about new treatments before they are widely available. Some studies are designed for, or include, people who are healthy but want to help find ways to prevent a disease, such as one that may be common in their family.

Many people say participating in a clinical trial is a way to play a more active role in their own health care. Other people say they want to help researchers learn more about certain health problems. Whatever the motivation, when you choose to participate in a clinical trial, you become a partner in scientific discovery. And your contribution can help future generations lead healthier lives. Major medical breakthroughs could not happen without the generosity of clinical trial participants—young and old.

 

 

Who can participate in a clinical trial?

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All clinical trials 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 trial 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 trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, 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 and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

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What happens during a clinical trial?

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The clinical trial process depends on the kind of trial being conducted (See What are the different types of clinical trials?) The clinical trial team includes doctors and nurses as well as 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 protocol is carefully followed and there is frequent contact with the research staff.

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What is informed consent?

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Informed consent is the process of learning the key facts about a clinical trial before deciding whether to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether 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. 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 to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

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What are the benefits and risks of participating in a clinical trial?

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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. 

There are risks to clinical trials:

There may be unpleasant, serious or even life-threatening side effects to experimental treatment

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.

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What are side effects and adverse reactions?

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Side effects are any undesired actions or effects of the experimental drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

    

 

How is the safety of the participant protected?

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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. 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.

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What should people consider before participating in a trial?

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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. 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 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 trials be provided to me?

• Who will oversee my care?

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What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?

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Plan and write down possible questions to ask. It may be helpful to ask your personal physician for input.

Ask a friend or relative to come along for support and to hear the responses to the questions.

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Does a participant continue to work with a primary health care provider while in a trial?

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Yes. 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.

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Can a participant leave a clinical trial after it has begun?

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Yes. 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.

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Where do the ideas for trials come from?

The ideas for clinical trials often originate in the laboratory. Researchers develop a clinical trial protocol (the plan for a trial) after laboratory studies indicate the promise of a new drug or procedure.

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Who sponsors clinical trials?

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Clinical studies can be sponsored, or funded, by pharmaceutical companies, academic medical centers, voluntary groups, and other organizations, in addition to Federal agencies such as the National Institutes of Health, the U.S. Department of Defense, and the U.S. Department of Veterans Affairs.

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What is a protocol?

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A protocol is a standard set of rules that allow electronic devices to communicate with each other. These rules include what type of data may be transmitted, what commands are used to send and receive data, and how data transfers are confirmed. You can think of a protocol as a spoken language.

 

 

What is a placebo?

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A placebo is anything that seems to be a "real" medical treatment -- but isn't. It could be a pill, a shot, or some other type of "fake" treatment. What all placebos have in common is that they do not contain an active substance meant to affect health.

 

 

What are the different types of clinical trials?

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There are two main types of trials or studies - interventional and observational. Interventional trials aim to find out more about a particular intervention, or treatment. People taking part are put into different treatment groups, so that the research team can compare the results.

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What are the phases of clinical trials?

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Phase 0. Phase 0 trials are the first clinical trials done among people. Phase I. Phase I trials aim to find the best dose of a new drug with the fewest side effects. Phase II. Phase II trials further assess safety as well as if a drug works. Phase III. Phase IV.

 

 

What is an "expanded access" protocol?

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Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials.