Information for Participants

UNDERSTANDING CLINICAL TRIALS

Choosing to participate in a clinical trial is an important personal decision. The following questions provide detailed information about clinical trials. In addition, it is often helpful to talk to a physician, family members, or friends about deciding to join a trial. After identifying some trial options, the next step is to contact the study research staff and ask questions about specific trials.

What should people consider before participating in a trial?
What kind of preparation should a potential participant make for the meeting with the research coordinator or physician?
Does a participant continue to work with a primary health care provider during a trial?
Where do the ideas for clinical trials come from?
Who sponsors clinical trials?
What is a control or control group?
What are the phases of clinical trials?
What is "expanded access"?
Can any individual patient qualify for expanded access to any drug? 

What should people consider before participating in a trial?

People should know as much as possible about the clinical trial and feel comfortable asking questions of the healthcare team, the care expected during the trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the healthcare team. Some of the answers to these questions are found in the informed consent document:

  • What is the purpose of the study?
  • Why do researchers think the approach may be effective?
  • Who will fund the study?
  • Who has reviewed and approved the study?
  • How are study results and safety of participants being checked?
  • How long will the study last?
  • What will my responsibilities be if I participate?

Possible risks and benefits

  • What are my possible short-term benefits?
  • What are my possible long-term benefits?
  • What are my short-term risks, such as side effects?
  • What are my possible long-term risks?
  • What other options do people with my disease have?
  • How do the possible risks and benefits of this trial compare with those options?

Participation and care

  • What kinds of therapies, procedures and /or tests will I have during the trial?
  • Will they hurt, and if so, for how long?
  • How do the tests in the study compare with those I would have outside of the trial?
  • Will I be able to take my regular medications while in the clinical trial?
  • Where will I have my medical care?
  • Who will be in charge of my care?

Personal issues

  • How could being in this study affect my daily life?
  • Can I talk to other people in the study?

Cost issues

  • Will I have to pay for any part of the trial such as tests or the study drug?
  • If so, what will the charges likely be?
  • What is my health insurance likely to cover?
  • Who can help answer any questions from my insurance company or health plan?
  • Will there be any travel or child care costs that I need to consider while I am in the trial?

Source: Cancer.gov

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

  • Consider taking a family member or friend along, for support and for help in asking questions or recording answers.
  • Plan ahead what to ask — but don't hesitate to ask any new questions you think of while you're there.
  • Write down your questions in advance, to make sure you remember to ask them all.
  • Write down the answers, so that you can review them whenever you want.
  • Ask about bringing a tape recorder to make a taped record of what's said (even if you write down answers).

Source: NIH.gov

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

Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary healthcare. In addition, by having the healthcare 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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Where do the ideas for trials come from?

The idea for a clinical research study—also known as a clinical trial—often originates in the laboratory. After researchers test new therapies or procedures in the laboratory and in animal studies, the most promising experimental treatments are moved into clinical trials, which are conducted in phases. During a trial, more information is gained about an experimental treatment, its risks, and its effectiveness. (For more information about the role of IRBs in overseeing clinical trials, see our question about participant safety on our Information for Parents page.)

Source: NIH.gov

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Clinical trials are sponsored or funded by various organizations or individuals, including physicians, foundations, medical institutions, voluntary groups, and pharmaceutical companies, as well as federal agencies such as the National Institutes of Health and the Department of Veterans Affairs.

Source: NIH.gov

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What is a control or control group?

In many clinical trials, researchers often administer the drug or treatment under study to one group of study participants, while giving the standard treatment or placebo (an inactive pill, liquid, or powder that has no treatment value) to another group that serves as the control. This allows researchers to accurately measure the drug's effectiveness by comparing data from the group that received the drug against that of the control group. 

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What are the phases of clinical trials?

Clinical trials are conducted in “phases”. Each phase has a different purpose and helps researchers answer different questions.

  • Phase I trials: Researchers test an experimental drug or treatment in a small group of people (20–80) for the first time. The purpose is to evaluate its safety and identify side effects.
  • Phase II trials: The experimental drug or treatment is administered to a larger group of people (100–300) to determine its effectiveness and to further evaluate its safety.
  • Phase III trials: The experimental drug or treatment is administered to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or equivalent treatments, and collect information that will allow the experimental drug or treatment to be used safely.
  • Phase IV trials: After a drug is approved by the FDA and made available to the public, researchers track its safety, seeking more information about a drug or treatment’s risks, benefits, and optimal use. 

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What is "expanded access"?

Expanded access, sometimes called "compassionate use," is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options. 

FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial. They also permit expanded access for large groups of patients who do not have other treatment options available, once more is known about the safety and potential effectiveness of a drug from ongoing or completed clinical trials. 

Just as in clinical trials, these investigational drugs have not yet been approved by the FDA as safe and effective. They may be effective in the treatment of a condition, or they may not. They also may have unexpected serious side effects. It is important for you to consider the possible risks if you are interested in seeking access to an investigational drug. 

Source: FDA.gov

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Can any individual patient qualify for expanded access to any drug? 

No. In order for a patient to gain access to an investigational drug outside of a clinical trial, the patient must have a serious or immediately life-threatening disease or condition and no comparable or satisfactory therapeutic alternatives. Additionally, the drug manufacturer and the patient’s doctor must make special arrangements to obtain the drug for the patient. These arrangements must be authorized by the FDA. These safeguards are in place to avoid exposing patients to unnecessary risks. 

Manufacturers may not always be willing or able to provide access to a drug outside of their clinical trials. Physicians may not always be able to seek expanded access for patients, depending on a patient’s medical history and the risks associated with taking an investigational drug. The physician must determine that the probable risk from the drug is not greater than the probable risk from the disease. Not all physicians are willing to manage the use of an investigational drug for patients in their care. 

Source: FDA.gov

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