Patient Participation in Clinical Trials

Clinical research is medical research involving people. The goal of clinical research is to translate scientific observations and laboratory discoveries into new ways to diagnose, treat and prevent disease.

Research participants include healthy volunteers and patient volunteers.

A healthy volunteer is a person with no known significant health problems who participates in clinical research to test a new drug, device, or intervention. Research with healthy volunteers is designed to develop new knowledge, not to provide direct benefit to study participants. Healthy volunteers have always played a vital role in medical research.

A patient volunteer has a known health problem and participates in research to better understand, diagnose, treat, or cure that disease or condition. Research procedures with a patient volunteer help develop new knowledge. Involvement in such research may or may not benefit the study participants.

Why Do Research In Children?

Medicines, devices, and treatments are often not tested in children.

At nearly half of medical visits, children are given a medicine, and 70% of those medicines have only been tested in adults.

The simple truth is…children are not little adults.

But without research involving children, we have no choice but to treat them that way.

Doctors and advanced practice providers (e.g. nurse pratictioners and physicians assistants) often give medicines to children even though they have not been studied and approved by the Food and Drug Administration (FDA) for use in children. This is known as “off-label” use. Most of the time, this works well, but when the adult dose is adjusted to the weight of a child, there is a chance that the dose used could be ineffective or even harmful.

This approach may sound like guesswork, but without research in children, it’s all we have. We need to think about how a child’s brain and body are developing…as well as the way a child’s body handles medicines and other treatments over time.

Why Clinical Studies Are Important

Clinical research in children helps us to treat our children like children, rather than as little adults, in several ways:

  • it uncovers the best dose of medicines to prevent harmful effects or under-treatment;
  • it leads to the development of chewables, liquids, or tablets that are easier for children to take, yet still safe;
  • it results in treatments for problems that occur only in children, like prematurity;
  • it leads to treatments for diseases or conditions that occur in both children and adults but that act differently in children and adults, like arthritis or heart disease;
  • it results in treatments for new or existing diseases that improve the health of children in the future, like vaccine studies that were done years ago and help children stay healthier today; and
  • clinical research in children help us understand how medicines affect children’s brains and bodies as they grow and develop.

Pediatrician Clinician-researchers, doctors, and nurses talk about the importance of conducting clinical trials for children while addressing common questions that parents and caregivers face. www.nhlbi.nih.gov/childrenandclinicalstudies/whyclinical.php

Stages of Growth

Children are growing. They are changing and maturing all the time. An 8-month-old is completely different from an 8-year-old, who is completely different from an 18-year-old, so even among children, everyone is different. And at each of the stages of growth below, children may need different doses of medicine, different sizes of devices, or different types of therapy.

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For example, testing of one antibiotic showed that babies needed higher doses than older children to get eradicate their infection. Many medicines are filtered out of the body and handled differently by a child’s developing liver or kidneys—and because there is limited research, we don’t know what the long-term effects on these organs may be. So, we need to conduct studies to find out.

How Research is Different than Care

Clinical research can look a lot like regular, or standard, medical care. Sometimes it is hard to tell the difference.

Here are some of the ways they may be similar:

  • The researcher and your health care provider can be the same person.
  • The setting may be your regular clinic.
  • The treatments may seem the same.

Research is done to help find out if a treatment or procedure is good for a large group of people with a certain disease or condition. Research helps to answer questions for the future health of those populations. Standard medical care, however, focuses on individual needs in the present.

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When considering enrolling your child in a study, make sure you understand the difference between the regular care your child gets from the doctor and what’s involved in research. Even when the place and health care providers are the same as your regular health care team, find out what makes it a research study.

Make sure you ask: How is this different from standard care?

  • Will I see different doctors and nurses for the study?
  • Will I go to a different hospital or clinic for the study?
  • Will the doctors and nurses ask me a lot more questions about my child’s condition?
  • Will there be more paperwork or additional tests when we are in the study?
  • Will there be more rules and deadlines in the study?

Here is an example of how some things in a clinical study are the same as in regular care, while some are different.

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How Children Benefit

Parents who are asked to enter their child into a study will want to know, “Will being in this study help MY child?” It is very important to understand that research is done to gain information about a disease, condition, drug, or treatment that will benefit children in the future. It is different from regular medical treatment that is given to help a specific child.

So, while most studies are not done to help a specific child, does this mean there are no benefits to being in a study?

In fact, there can be potential benefits when entering a clinical study.

Helping Future Generations

One reason that parents say that they join a study is to help other families in similar situations. Today our children have more protection from death, disability and discomfort from many childhood diseases like polio or measles because parents in the past made a decision to allow their child to be in a study to test vaccines.

But, there are still many medicines and procedures that have not been tested in children. And before they can be accepted for use, they must be tested to see if they are safe and effective.

Having Access to New Drugs or Treatments

Researchers test new drugs and treatments because they have reason to believe they might work better or be safer than the standard care. In a study, your child may have access to something that is not available yet. If the drug or treatment is found to be helpful, your child may be among the first to benefit.

Sometimes enrolling in a clinical study can give your child a chance to see extra doctors or find out more facts about your child’s condition. The study team may be able to tell you about organizations, groups, or websites that deal with your child’s condition. A clinical study may be able to put you in touch with families going through what you are going through.

Having Closer Monitoring

A clinical study may offer closer monitoring or additional testing for your child, which may not be part of regular care. Sometimes a study asks parents to keep a diary or to bring a child in to be seen more often, such as weekly visits. Children in a clinical study will be watched closely for side effects and to understand how the treatment is working.

Whatever the reason, remember that clinical studies are designed to test if a drug or procedure works and is safe. There may be benefits for your child, but there may not be.

Safety and Protections

Every parent wants to know Who is watching out for my child in a study?

The answer is lots of people.

While research can have risks, there is a lot that goes on “behind the scenes” to ensure safety before families are invited to be in a study.

  • Many people review a study in detail before a single person is enrolled. These people include
    • the scientists who focus on identifying the right treatment,
    • the statisticians who help to design the right study with the right number of participants to get good results, and
    • the medical investigators (nurses, doctors, pharmacists, psychologists, and technicians) who will make sure that the study is done in the right way with the right participants.
  • Informed consent documents are developed to describe why a study is being done, what will happen, and risks and benefits.
  • Institutional Review Boards(IRBs) are independent committees that review research plans and consent forms to make sure that people in a study are informed and protected when in studies. They review studies both before they start and throughout the study. They are also known as Research Ethics Boards (REBs) or Ethics Committees (ECs).

Safety measures

There are safety measures in place to protect children while they are in a study.

  • There are many clearly defined government rules for protecting human subjects in clinical research with special protections for children (this link will take you to the Office for Human Research Protections for more information about Special Protections for Children as Research Subjects).
  • Each study has a research team responsible for running the study and making sure people are safe.
  • Some studies have a Data and Safety Monitoring Board that regularly monitors safety. This impartial Board oversees studies and says if a study should be changed or closed at any time for safety issues.
  • Some studies have a Medical Monitor, usually a doctor or nurse not associated with the study, who reviews side effects that occur in a study.

Be comfortable

As a parent, you must be comfortable with what your child will be doing in a study.

  • Even with efforts to make your child safe, remember there may still be risks. Make sure you understand the possible risks and benefits and understand how they apply to your child.
  • Get familiar with the study team whose job it is to protect your child.
  • Find out what resources are available to help you understand your rights.
  • Ask questions.

Clinical Studies don’t just “happen.” Before your child is even asked to participate, the procedure or medication being researched has likely been through a series of steps:

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Rights of Families in Studies

You’re being asked to enroll your child in a study.

You’ve seen a lot of information; you’ve talked to the research team. You know you have rights but would like to know a little more about them.

When you are thinking about joining a study, remember that you have the following rights:

  • You do not have to join a research study unless you want to. You should never feel pressured or forced to join.
  • The clinical researchers must tell you about all the known or possible risks and benefits of the treatments in the study.
  • You have the right to obtain full and complete information about what the study means for your child, what is expected of you, and how the study will be done.
  • You have the right to ask questions about the study before you start and as the study goes on.
  • You have the right to know about any costs to you or your insurance company.
  • You have the right to know about other treatment options, if any exist, and their risks and benefits.
  • You have the right to know how your personal information will be shared and protected.
  • You have the right to obtain a copy of the signed and dated written informed consent form when one is required.
  • You have the right to know about any new information that the researchers may learn about the treatments while the study is going on.
  • You have the right to leave a research study whenever you want. You should never be pressured to stay in a study. You cannot lose access to your regular medical care if you leave a study.

These rights are in place to make sure that you feel that your child is protected and that you have control over the decisions made about medical care and being in a study.

Good Questions to Ask

It is difficult enough to decide to enroll in a study as an adult, but it is even harder to make that decision for a child, especially if the child is sick.

Clinical study documents have a lot of information, but there are questions that you may still have. Here is a list of possible questions you might want to consider asking your research team. Remember that you should ask questions until you are comfortable with the information provided

There are many precautions in place to protect children in clinical studies. Safety is the most common concern for parents, along with what risks and benefits they can expect. But there are many other factors involved when joining a study, which parents need to look at and ask questions about. And sometimes when you ask a question, it might cause the research team to think about how to make the study better or easier for you and others. Don’t forget, the research team will expect you to ask! Here are some questions:

  • What will happen and how much time will it take?
  • How will it affect other family members?
  • Are there costs?
  • How do I know what questions to ask?
  • What if I have questions during the process?

Searching Clinical Trials

Parents most often find out about clinical studies from a health care provider who is conducting a study, and sometimes your doctor or nurse may recommend a study to you. If you are seeking a clinical study, there are a few ways to search.

ClinicalTrials.gov is a database of trials in many different diseases for different age groups. Here are a few tips to help you get started:

  1. In the search box, enter the medical condition, disease, or term you are interested in searching, such as “epilepsy.” This might turn up many studies.
  2. To help narrow your search, click “Modify this search.” The most important fields to complete might be the following:
    1. Location: You may pick up to three locations.
    2. Age Group: Trials for children will fall into the “Child” category.
  3. Once the form is complete, click the Search box at the bottom of the page.
  4. The next page presents a full list of the trials. The trials are listed based by best match first.
  • Recruiting means the trial is looking for volunteers
  • Unknown means that the search system is not sure if the trial is open.
  • Completed means the trial has closed and no new participants are being taken.

ResearchMatch.org  is a tool to match patients with appropriate studies. You must register to receive notifications about relevant clinical studies. The site will walk you through the process to register you or your child. Once registration is complete, any matches will be emailed to you with further instructions about how you can get more information about participation.

Visit our Clinical Trials Directory to explore links to current clinical trials sponsored by our partners.

Source: National Heart, Lung, and Blood Institute, National Institutes of Health: www.nhlbi.nih.gov/childrenandclinicalstudies