Clinical studies often include terms that are unfamiliar to you. You should ask your research team to explain each term until you are comfortable that you understand what it means to you. This section discusses some terms very common to clinical studies.
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Assent
Assent
“The term used when a child agrees to be in a study.”
Even though parents and guardians must consent for their child to join a study, children should have a part in making a decision to join a study, if they are capable of doing so. When a child is asked to have a part in the decision, this is called “assent.”Children do want to have a say in what happens to them and they want to ask questions and have them answered. When children are asked if they want to join a study, it shows respect for them. And they will feel good about being in the study and more committed to doing what the study requires.
It has been found that most children from age 7 can understand basic information if it is given at their level. So, in most studies, children are now asked if they agree (assent) to be in a study and are asked to sign an assent form. These forms are usually a simpler version of the consent form that parents sign. They have also been reviewed by the same safety group, the Institutional Review Board, to assure that the forms are accurate and at a child’s level. Making sure children have a say is important, but remember—not all studies require assent, and the age when assent is requested can vary depending on the study.
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Blinding Or Masking
Blinding Or Masking
“That’s when patients, and usually doctors too, don’t know which study treatment each child is getting.”
This study term can sound scary. But it is really pretty simple. In some studies, it is important that certain people do not know (or are blinded to) which study group your child is in. You may be blinded, or your doctor may be blinded or the research team itself could be blinded.Why? Researchers want to make sure that anything you are filling out, like surveys or diaries—as well as anything the doctors or research team may be doing, like physical exams or tests—is not affected by knowing which group anyone is in. Let’s imagine that your child is in a study of a new medicine. If your doctor knew that your child was taking the new drug, maybe the doctor would go in to the study thinking this is a great drug, and then might, unknowingly, evaluate results based on that opinion. Or you may think that because your child is suddenly tired, it has to do with the drug. Researchers want to make sure that the data that is collected is true and not affected by how any one person feels about the treatment.
One important thing to know is that in every study there is someone who knows what drug or treatment each participant is taking. In an emergency, the research team and doctors can find out quickly.
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Informed Consent
Informed Consent
“A parent’s guide to learn key facts about a research study.”
This is the way that you as a parent are given details about a study so that you can decide if your child should join a study. You are “informed” so that you can give your “consent” or okay. Nothing can happen until you consent to it.Most people think about “informed consent” as a long document that is given to them when they are asked to be in a study. And while there is a form, the discussion you have with a member of the research team is vital. The study team wants you to know about every detail of the study; you should know everything about what will happen when and what your and your child’s involvement will be.
Take the time to ask them questions.
Sit down and read the full informed consent document.
After that, if you still have questions, make notes to yourself and go back to the research team.
Take your time! The informed consent form can be long and may have words that are hard to understand. Ask your research team for any help you might need. Remember, always ask questions!
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IRB
IRB
“A group of experts who monitor clinical studies to make sure people are safe.”
Most research done in the United States must have what’s called “independent review.” This means that a group of experts in research on people, who are separate from the research, reviews it. In the United States these groups of experts are usually called Institutional Review Boards (IRBs). Outside the United States, the groups of experts who review research can be called Research Ethics Boards or Ethics Committees. They review research studies to decide whether or not to allow the studies to be done at the hospital, doctor’s office, clinic, or other place for which the IRB is responsible. IRBs are made up of different types of people: doctors, nurses, ethicists, community people, attorneys, patients, pharmacists, and others.The IRB’s role is to help ensure that the study is well designed, that the risks are as low as possible, and that the rights of study participants are protected. When IRBs review studies in children, they take extra care and follow special rules.
The IRB continues to monitor the study throughout its duration for safety and to make sure it’s continuing to be run properly.
You can usually tell if an IRB has reviewed the study by a stamp on the consent form. If the consent form doesn’t contain this information, ask your research team if the IRB has reviewed and approved the study.
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Placebo
Placebo
“Placebo is a pill, liquid, or powder that has no active medicine in it. It’s a fake.”
There are lots of questions parents have about a placebo. A placebo can be a sugar pill or a look-alike procedure or device that has no curative effects. Parents might worry that their child is going to be denied effective treatment if a placebo is part of a study.But it is important to know that there are ethical rules that help researchers decide if a placebo is okay to use in a study with children. A placebo must pose little risk to participants and the harms and benefits of being in the placebo group should be similar to those in the treatment group.
A lot of times, studies compare a new treatment to an older treatment. But sometimes it is necessary to see if a new treatment is better than doing nothing. This isn’t as silly as it sounds because some treatments have side effects that are harmful. And believe it or not, some patients who get the placebo in studies do improve.
Sometimes there is a standard therapy and researchers want to see what happens if a new therapy is added. This is commonly done in child cancer studies in which all children get the standard therapy, half get a new treatment, and half get the placebo to see if adding a drug will have more benefits or not.
Most importantly, risks must be minimal.
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Protocol
Protocol
“A detailed plan that says who can be in a study, for how long, and what will happen. It’s like the rules.”
The Clinical Study Protocol is the document that describes a study, why the study is being done, how the study will be conducted, and how the results will be analyzed. If the study is taking place at many different locations, study teams at each site will use the protocol to perform the study in exactly the same way.The protocol will describe
- why the study is being done;
- what types of children can be in the study;
- when the visits, tests and procedures will take place;
- the types of medicines and dosages to be used;
- how long the study will last;
- how children will be kept safe;
- possible benefits and risks;
- how privacy will be protected;
- how side effects will be monitored and reported;
- who will monitor the safety of the study participants and how often; and
- how the results will be analyzed.
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Randomization
Randomization
“Since researchers don’t know which treatment is better, this is a way to determine who gets what like flipping a coin.”
Randomization is a fancy way of saying “by chance.” It’s similar to rolling dice or flipping a coin. It is often the best way that researchers have to end up with study groups that will be similar in age, ethnicity, and other characteristics in order to better compare the results at the end of a study.Picture a bowl filled with the same number of green and blue marbles. You cannot see them, but you are going to pick one. Green marbles go to DRUG A and blue to DRUG B. You can’t know which color you’ll grab before you do it, so there’s no determining beforehand which color might get picked at any time. Similarly, which children are selected into treatment groups remains “random.”
If we know who is getting what drug or treatment, we are more likely to make judgments about what is better before we really see the results.
