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Our goal is to provide information that might help you decide whether or not to take part in a clinical trial. This handout reviews key information to consider and contains additional questions that you may want to ask.
We hope that this information is useful as you consider whether or not a clinical trial is right for you.
- What is a Clinical Trial?
- How Are Patients Protected While on a Study?
- Eligibility, or Do You Fit the Group of People to be Studied?
- Safety Monitoring, or How Will Side Effects be Found Early?
- What are the Phases of Clinical Trials?
- What Might Help You Decide if Entering a Clinical Trial is Right for You?
A clinical trial is a research study that is done to answer important questions about medical care. They are research studies in which new therapies and treatments are tested in patients to determine if they are safe and effective. If you choose to take part, you and the study team will work together to find out if a new method of treatment, diagnosis, or prevention is effective. Carefully conducted clinical trials are the safest and fastest way to find treatments that work in people, and new ways to improve health.
There are different kinds of clinical trials:
- Prevention trials that 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 to test the best way to detect certain diseases or health conditions.
- Quality of Life (or Supportive Care) trials, that explore and measure ways to improve comfort and the quality of life for individuals with a chronic illness.
Clinical trials are conducted in phases. Usually, they compare a new product or therapy to something else to see if it works as well or better to treat or prevent a disease or condition. In a blinded study, a participant may be randomly assigned to receive the test product, or an existing, approved therapy. In some studies, participants may be assigned to receive a placebo (a product with no therapeutic action that looks like the test product). Comparison with a placebo can be the fastest and surest way to demonstrate therapeutic effectiveness of new products. However, placebos are not used where a patient would be put at risk, particularly in the study of treatments for serious illnesses, by not having effective therapy.
Participating in well-designed and well-executed clinical trials is one approach for eligible patients/volunteers to:
- get actively involved in their health care;
- receive regular and careful medical attention from a research team that includes doctors and other health professionals;
- gain access to potentially new research treatments;
- have access to expert medical care for the condition being studied, since investigators are often specialists in the disease area being studied;
- help others by contributing to medical research.
Taking part in a clinical trial is voluntary. Anyone can decide not to participate. If you decide to take part in a trial and then change your mind, you can also drop out of the trial at any time. Whatever decision you make, your doctors and nurses will make sure you get the best care possible for you.
There are three systems in place to keep patients as safe as possible while taking part in a study: protocol review, eligibility, and monitoring.
When a researcher wants to perform a clinical trial, he or she must prepare a detailed plan. This is called a protocol. The trial cannot be started until a formal committee people, called the Institutional Review Board (IRB) has reviewed the protocol for safety and effectiveness. The IRB are made up of physicians, nurses, scientists, pharmacists, administrators who may work at the facility offering the trial, and at least one person who is not connected with the health care facility.
Federal institutions such as the U.S. Food and Drug Administration (FDA) or the National Cancer Institute (NCI) may also review the study. While the study is going on, the IRB receives regular updates to make sure the study is being carried out as planned and that any safety issues are addressed promptly.
Like all clinical research, each clinical trial must be based on the following principals:
- Respect for the person – requires full voluntary agreement to participate.
- Beneficence- ensures the clinical trial provides the maximum benefit to the patient while minimizing their risk.
- Justice – entrances into a trial is guarded against a patient being vulnerable, or accepted into the trial for wrong reasons.
A patient must meet certain requirements to take part in a clinical trial. First, if your risk from being in the trial is greater than any possible benefit, you might not be able to join the study. Second, the people taking part in some trials must be similar to one another so the study team can learn if the new treatment works the same as or better than the standard treatment. Some examples of study criteria are age, disease type, the ability of body organs to work, and the ability of the person to carry out the normal activities of daily living.
Often, the study treatment has not been used in many people. In these cases it may be hard to predict what side effects might occur. During a study, the team will try to identify any potential side effects as quickly as possible. They may do more tests and physical exams than they would if someone were not taking part in a clinical trial. You would be informed to report any potential side-effects, even minor ones, quickly to receive the extra care provided during a clinical trial. If you develop unexpected health problems, you would be treated promptly and may have to withdraw from the study.
Phase I: How much of the new treatment can be given with reasonable safety?
The goal of a Phase I study is to find out three things:
- How much of a new drug can be given safely?
- How often the drug needs to be given?
- What are the side effects of the new drug?
Researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. Generally, if you are one of the first patients to take part in a Phase I study, you will be given a small amount of the drug. This amount is based on animal testing or the safe dose of a similar medication given to other people. In some cases, it is based on the safe dose of the new drug given to people who took part in earlier studies of the drug. If there are no serious side effects, people who enroll later in the same study will receive more of the drug, until the study team learns the maximum amount of the drug that can be given and still be reasonably safe. The studies are most often carried out at academic medical centers or large hospitals.
Phase II: Does the new treatment work for a specific type of disease or condition?
The goals of a Phase II study are to:
- continue to gather information about the safety of the drug
- test whether or not the drug works against a specific disease or condition.
In Phase II studies, the experimental study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety. Patients with a specific disease or condition are given the highest dose of the new drug that is thought to be reasonably safe. This dose is based on results from Phase I studies. Generally, these studies are often carried out at major hospitals or academic medical centers, but they may also be conducted at local community hospitals.
Phase III: Is the new treatment better than standard treatment for a specific disease or condition?
In Phase III, the experimental study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the experimental drug or treatment to be used safely. Patients are assigned at random to a group that receives standard treatment or to a group that receives the new treatment. This means that the patient is assigned to a group by chance, like rolling dice or flipping a coin, and not by his or her doctor. The patient has an equal chance of being assigned to any one of the groups. If the study team learns that one of the drugs is not effective or is too toxic, the study might be changed or stopped earlier than planned.
Phase IV trials are conducted after a drug or device has been approved for consumer sale and often called post marketing studies.
Phase IV trials help: (1) to compare a drug with other drugs already in the market; (2) to monitor a drug's long-term effectiveness and impact on a patient's quality of life; and (3) to determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies. Phase IV studies can result in a drug or device being taken off the market or restrictions of use could be placed on the product depending on the findings in the study.
If you are eligible and are thinking about taking part in a study, it is important that you find out what options are available and that you think about them carefully. Don’t make a decision without getting as much information as you reasonably can.
Understand your choices.
Bring a trusted family member or friend with you when you meet with the study team to discuss options. That person can:
- ask questions
- write down the study team’s answers
- discuss the pros and cons of the study with you after the doctor’s visit
Meet with a financial counselor at the study site.
He or she can contact your insurance company to find out if it will pay for the extra costs of taking part in the trial. Many insurance companies will pay for a lot of clinical trial-related services.
Request a professional medical interpreter if English is not your first language.
That person will:
- make sure that the translation is accurate
- make it possible for family members or friends to act as advisors in the decision-making process
- know the medical terms so he or she can translate the medical information clearly and accurately
Never feel pressured to participate.
If you are feeling pressured by anyone, even a family member, to make a certain decision, you may always discuss your options with someone else. Possible sources of help include:
- spiritual leaders
- health care team members, such as your physician, nurse, or social worker
- psychologists and support group members
Remember that you don’t have to take part in research. You should not take part in the trial unless you feel it is right for you.
Questions You Might Ask the Study Team or Hospital Staff
- What should I know about the study?
- Why is the study being done?
- Have there been other studies that this drug, device or treatment? If so, what were the results?
- Is it possible that I will be treated with an inactive substance (placebo) if I participate in the study?
- What financial issues I should consider?
- Will my insurance company pay for any extra costs of participating in the trial?
- If I don’t have insurance, can I still be in the study?
- If I need long-term treatment for a health problem that might have been caused by the new treatment, will my insurance company pay for it?
- How will my health care study team follow my care?
- Who are the health care professionals who will take care of me during the study?
- Will my primary care physician or primary oncologist be involved?
- If I withdraw from the study or if I change studies, will my health care team change? •
- When the study is over, will the same doctors and nurses take care of me?
- How will the study affect my overall health care?
- How are the study treatments and procedures different than the regular medical care that I might get if I decide not to take part?
- Will the study require that I be hospitalized? If so, for how long?
- Can I take my regular medications while I am in the study?
- If I enroll in the study, will I be able to find out my test results?
- If someone else in the study has an unexpected and severe health problem, will I be told?
- If I think the new treatment worked for me, will I be able to get the same treatment after the study ends?
- Will I need to keep getting tests done after I stop taking the study drug?
- Where will I be treated if I participate in the study?
- What happens if I withdraw from the study?
- If I withdraw, will I continue to be cared for by the same health care team?
- Can I switch from one trial to another?
Questions You Might Ask Yourself
Because each person is unique, what feels right to someone else might not feel right to you. In other words, when it comes to a clinical trial, you should make a decision based on your personal feelings and needs. Below is a list of questions you might want to ask yourself as you think about whether or not to take part in a clinical trial.
- Am I able to make the extra trips to the doctor or hospital that might be needed for the study?
- Do I have family or friends who can help me if I experience special problems during the trial?
- Do I feel comfortable receiving a treatment whose risks and benefits are somewhat unknown?
- Will I be able to manage if taking part in the trial means I might miss more work?
- If taking part in the study might cost me more money than standard treatment, would I be able to afford it?
- If I am very sick and have exhausted all other treatment options, would I rather receive treatment aimed at making me live comfortably and help me stay at home instead of being in a clinical trial that might cause side effect.
- Am I satisfied that my decision is right for me, and not a decision that is being made to please someone else?