What is a clinical trial?
What are clinical trial phases?
Who can participate in a clinical trial?
What is informed consent?
How am I protected if I participate?
Who sponsors clinical trials?
What happens during a clinical trial?
What are the benefits and risks of being in clinical trials?
What should I know before I join a clinical trial?
How should I prepare for the meeting with the research team representative or doctor?
What questions should I ask?
Can I continue working with my primary healthcare provider if I participate in a trial?
Can I leave a clinical trial after it has begun?
Will I be paid for participating in a clinical trial?
Will my insurance cover the costs involved if I join a clinical trial?

What is a clinical trial?

Clinical trials are research studies involving people. These studies try to answer the safety and efficacy of a new treatment, therapy or medical device under highly controlled conditions.

A clinical trial is one of the final stages of a long research process. Before any new treatment is tested on people, extensive review of safety and effectiveness is done in the laboratory and on animals. Only after these results show potential benefits exceed any risks or side effects are human studies carried out.

Trials often require large numbers of participants to best evaluate effectiveness and long-term safety of a drug or device.

Clinical trials follow strict requirements (protocols) of how a study is carried out. Protocols may require a treatment to be examined alone or compared with a placebo (sugar pill) or a treatment that is already known. Or, a trial may be designed to show how an already known treatment can be used in a new way. Protocols also describe who may participate in the trial, the schedule of tests, procedures, medications and dosages, and the length of the study.

While a trial is in effect, participants are regularly seen by doctors and research staff who monitor the participants' health and closely follow the safety and effectiveness of the treatment.

What are clinical trial phases?

Clinical trials of a new drug go through four phases:

• Phase I (20 to 80 patients) - These studies, which typically last several months, are primarily designed to determine safe doses and how a new drug should be given (by mouth, injected into the blood, inhaled, etc.). About two-thirds of drugs tested in Phase I move to Phase II trials.

• Phase II (100 to 300 patients) - Continues to test the safety of the drug and how well it works.

• Phase III (1,000 to 3,000 patients) - To further determine safety and effectiveness of a drug in a large number of people over a longer time period, and at times to compare the new treatment with known treatments.

• Phase IV (post-marketing studies) - These studies involve ongoing monitoring of large patient groups after a drug or device has been approved and marketed. Phase IV studies monitor long-term safety and watch for any problems that develop after distribution of the drug or device.

Who can participate in a clinical trial?

Because each clinical trial looks for answers to different questions, each trial has specific guidelines to describe who can participate. Guidelines describe characteristics such as age, type of disease, medical history and medical condition. If you wish to participate, you must meet these characteristics or inclusion criteria, to qualify for the study. There are also exclusion factors, which may prevent you from participating in a study.

Keep in mind these factors are in place before you are made aware of the study, and they are not used to reject people for personal reasons. Instead, these factors are necessary to identify appropriate patients suitable for the specific design of the study and to keep them safe.

What is informed consent?

Before entering a trial, you will learn the key facts about the trial through the process of informed consent. Your rights as a participant in a trial entitle you to know:

• Why the research is being done.
• What the researchers want to accomplish.
• What will be done during the trial and for how long.
• What risks are involved in the trial.
• What benefits you can expect from the trial.
• What other treatments are available.
• The fact that you have the right to leave the trial at any time.

As you consider whether to enroll or participate in a clinical trial, the research staff gives you detailed written information known as the informed consent form, which describes the study. Before you agree to participate and sign the informed consent form, make sure you read all documents thoroughly and write down your questions. If you feel comfortable sharing the information, discuss it with your family or friends. They may have good questions or comments to suggest.

Talk with your doctor or the research team member who will be working with you. If English is not your native language, you can ask for the consent documents to be written in your native language. You should receive a copy of the informed consent so that you can review it at any time.

Your informed consent to participate in a clinical trial makes you a team member throughout the study. More than just a form, it's a process that continues through the study. You should feel free to ask the research team questions before, during and after the study.

How am I protected if I participate?

Before any trial is carried out with human participants, the protocol is reviewed, approved and monitored by an Institutional Review Board (IRB). The IRB follows strict government guidelines and safeguards to protect you as a participant, and makes sure the risks are as low as possible and are worth any potential benefits.

An IRB is an established group of physicians, statisticians, community members and clergy that ensures your clinical trial is ethical and protects your rights as a patient and participant. A research center involved in human research is required by federal law to have an IRB in place to approve and review a trial during the time it is active. Large research institutions, which perform many clinical trials, have their own IRB, while smaller centers and individual doctor's offices may use a nationally based IRB. Members are selected from the institutional staff, local citizens, attorneys and clergy members. IRB members are not paid. They volunteer their services.

Who sponsors clinical trials?

Many organizations, hospitals, pharmaceutical companies and governmental agencies sponsor clinical trials. Even insurance companies will evaluate certain treatments to consider if they are effective enough to make it part of their plan. Trials generally take place in a clinical setting, such as a university teaching hospital, a local hospital center, community clinics and even a doctor's office.

Most often, either your doctor or a research staff member will approach you to enroll in a particular clinical trial. Some studies (phases III to IV) require a large number of participants, so doctors and researchers are frequently paid for enrolling each participant in a study. Ask you doctor if he/she is receiving payment for your participation in the study, so that you are fully informed and aware of the sponsorship arrangement. Your doctor considers your best interests and would not ask you to participate unless you were an appropriate candidate. That is another reason for the strict guidelines and the need for participants to give their informed consent.

What happens during a clinical trial?

Once you are enrolled in a trial, the research team checks you health at the beginning of the trial. This evaluation becomes your baseline for monitoring the progress of the trial. Different protocols require a health checkup and/or tests and doctor visits at various times throughout the course of the trial -- more procedures than you may be used to. Your work and contact with the research team assures the most successful participation.

Here are some of the words, terms or expressions you may hear throughout the course of your trial:

• Placebo: This is what you may think of as a "sugar pill." It is an inactive pill, liquid or powder that is used as a baseline treatment so that any group of patients in a clinical trial appears to take the same preparation. In some studies, the participants in the control group receive a placebo instead of an active drug or treatment. In that way, researchers can remove any emotional reaction to a treatment.

• Control group: This group of participants usually receives either a placebo or the standard treatment, so that an experimental drug or treatment can be compared with a baseline. Participants will not know if they are in the control group or the experimental group.

• Blinded or masked study: Participants in a blinded or masked study are not aware of which treatment, if any, they are receiving. They are blinded to any knowledge of their treatment. In this situation, the study is called a single-blinded study, because participants are unaware of their treatment.

• Double-blind or double-masked study: When neither the participants nor the research team knows which participants are receiving treatment or placebo, the study is called double blind, because both parties are unaware of the conditions of treatment. This type of study is designed to try to remove both the patients' and the doctors' expectations for the results.

• Side effects or adverse reactions: Clinical trials are also conducted to learn about the undesired actions or side effects of a drug or treatment. Side effects are recorded and considered in determining if the treatment risk is acceptable, in relation to its benefit. Negative or adverse reactions are side effects that are may be unexpected, but may occur in a significant number of participants. Some adverse reactions may include headache, nausea, hair loss, skin irritation or other physical problems.

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

As a participant in a clinical trial, you may benefit by being able to:

• Take an active role in your own health care.
• Gain access to new treatments or drugs that are not available to the public.
• Obtain expert medical care at leading health care facilities during the trial.
• Help others by contributing to medical knowledge.

Some of the risks involved are:

• There may be side effects or adverse reactions to medications or treatments.
• The treatment may not be effective for you.
• The trial may require a lot of your time for trips to the study site, treatments, hospital stays or complex dosage requirements.

What should I know before I join a clinical trial?

Familiarize yourself with the research team responsible for your care during the trial. Being comfortable with them allows you to freely ask questions and have them answer you in a way you can understand. Try to learn as much as possible about the study.

How should I prepare for meeting with the research team representative or doctor?

• Plan ahead and write down your questions. You may want to keep a personal journal to record your feelings, reactions and questions that may arise during your care.
• Ask a friend or relative to come with you for support and to hear the responses to your questions.
• Bring a tape recorder so you can replay the discussion after you get home.

Here are some questions you might ask about the research:

• Why is this research being done?
• What is the purpose of the study?
• Who is sponsoring the study?
• Who has reviewed and approved this study?
• Why does the research team feel the treatment, drug or medical device will work?
• How many hospitals or institutions are participating in the study and where did the study originate?

Some questions you might ask about your participation in the study:

• Where is the study site?
• What kinds of therapies, procedures and/or tests will I have during the trial?
• Will they hurt? If so, for how long?
• How will the tests in the study compare to tests I would have outside the study?
• How long will the study last?
• How often will I have to go to the study site?
• Who will provide my medical care after the study ends?
• Will I be able to take my regular medications during the trial?
• What medications, procedures or treatments must I avoid while in the study?
• What are my responsibilities during the study?
• Will I have to be in the hospital during the study?
• Will the study researchers work with my doctor while I am in the study?
• Can anyone find out that I am participating in a study?
• Can I talk to other people in the study?
• Will I be able to find out the results of the trial?

Questions you might ask about risks and benefits:

• How do the possible risks and benefits of the study compare with approved treatments for me?
• What are the possible immediate and long-term side effects?
• How well am I covered for the costs of any treatment or hospitalization I might need for side effects from the study?

Other questions include:

• What other treatment options do I have?
• Will I have to pay anything to participate in the study?
• What are the charges likely to be?
• Is my insurance likely to cover those expenses?

Can I continue working with my primary health care provider if I participate in a trial?

Sure. Most clinical trials look at short-term treatments for a specific illness or condition. Trials do not usually involve a primary health care physician. If your primary doctor is working with the research team, your other medications or treatments will not be disturbed.

Can I leave a clinical trial after it has begun?

Yes. Though you are able to leave a clinical trial at any time during the treatment period, discuss your reasons with the research team.

Will I be paid for participating in a clinical trial?

Most clinical trials do not offer payment to participants, although some pay you a certain amount for joining and additional money if you complete the trial. Other programs may cover expenses such as transportation costs, childcare, meals and accommodations.

Will my insurance cover the costs involved if I join a clinical trial?

Before joining a clinical trial, contact your insurance company to find out if there is any type of information they might need to provide coverage for your care during the trial. Depending upon your coverage, you may be completely covered, partially covered or not covered at all, because certain companies may not reimburse treatments that are considered experimental, even if there is a small, but known, chance of success.

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External Sources National Cancer Institute The American Cancer Society

This article was reviewed and updated June 2007.