Investigational Drug FAQs
What is clinical research?
Some scientists do research in a labs and some scientists, including doctors and nurses, do research in a hospitals. Research involving patients in hospitals is called clinical research. Clinical research can study medicines, tests, and diseases with the goal of helping hospitals everywhere take better care of patients. Clinical research involves carefully planned studies that people volunteer to take part in.
How is medical research different from regular medical treatment?
Taking part in research is often a lot like the regular medical care your child receives at Cook Children's. In either case you will see the same providers because many of our doctors do research in their clinics. There are some differences in medical treatment and medical research.
In medical therapy parents agree to treatments to improve their own child's health. When children participate in medical research, parents give their permission to participate in the study. Parents may do this for several reasons some of which include the hope that it will benefit their child, but that it may also help other children suffering with the same medical issue. It is important to talk with your doctor and the research team about what medical care might be different if your child decides to be in a study.
When your child is in a research study, their doctor will still take the best care of them possible, but might do some things differently because they are following a study protocol. The protocol is a document that says exactly what will happen in the research study and it might ask doctors to give certain medicines, try certain treatments, or ask special research questions.
Does my child still see their regular doctor?
Yes. Your child will still see all of their regular doctors while in a study.
It's important to let your child's doctor(s) know your child is in a research study. Many studies at Cook Children's will ask you if you want the research team to send a letter to your child's doctor telling them that your child is in a research study. You can ask your child's doctor to talk with the research team to make sure there are no problems with the medicines or treatments your child is currently taking.
Can my child take part in a study if we don't live near Cook Children's?
Maybe, it depends on the study. Some studies can be as simple as filling out an online questionnaire. Other studies require you to come to regular, in-person, study visits.
Most of the time, patients will need to receive study treatments in-person at Cook Children's in order to be in a research study. Some studies can be done from your home or with your local doctor. If you are interested in your child being in a clinical research study, talk with a member of your research team about your options for participating in research outside of the Cook Children's campus.
What is a clinical trial?
Clinical trial: A research study that looks at if a medication, treatment, or medical device is safe and effective for people.
Why should my child participate in a clinical trial?
Clinical trials are important to help Cook Children's and other hospitals around the world give kids the best care. When children agree to be in clinical trials, doctors and scientists are able to gather more information on the newest treatment options for different diseases in kids.
Cook Children's goal is to only do studies that will help children. Sometimes this may mean your child will benefit from being in the study and other times it means the study may benefit many other children in the future.
What research studies or clinical trials are open at Cook Children's?
Cook Children's has studies open in nearly all of our departments. You can find the list of ongoing studies by clicing here. Please remember that not everyone is eligible for research studies. Even if your child has the disease or condition that the research is studying, they may not meet all of the study requirements. Talk to your child's doctor and/or research study team if you have questions about your child's eligibility for a study.
Who has reviewed and approved this study?
All research at Cook Children's is reviewed by an Institutional Review Board (IRB). The IRB is a committee that reviews research plans and documents to make sure that people in a study are informed and protected. They review studies both before they start and throughout the study. The IRB is made up of experts, like doctors and nurses, as well as people like parents and members of the community.
Who is asked to be in research studies?
Not everyone is asked to participate in research studies. If your child has been asked to participate, the investigator of the study thinks your child may qualify based on their condition or medical records.
What if we agree to be in research and then change our mind?
Being in a study is voluntary. Your child can stop participating in a study at any time without it affecting your regular medical care at Cook Children's. To stop being in a study you will need to let your research staff know and they will work with you to remove you from the study. You might be asked to fill out a survey to answer questions about why you decided to stop taking part in the study. Filling out the survey is voluntary and you will still be removed from the study if you don't want to fill it out.
Will I get to know the results of the research?
Most of the time, study participants don't know the results of a study before anyone else. At Cook Children's, we try to publish the results of all our studies in papers, posters, or presentations. Usually, because it can take quite a while to finish the study and write up the results, these publications will be available months or even years after you participate in a study. All the information we publish will be de-identified, meaning that any names, birthdays, or other information that could identify you or your child. Ask your research team how you can find the results of your study!
What is informed consent/assent?
Informed consent explains risks and potential benefits about a clinical trial before someone decides whether to participate. A member of your research team will sit down with you and your child and go over the consent in a "consent conference." You'll be encouraged to ask questions and consider whether or not you want your child to be in that 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. It also helps them 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.
Who is the sponsor of a trial?
The sponsor is the organization or person who initiates the study and who has authority and control over the study. Cook Children's is the sponsor of many of our studies, but we also participate in studies sponsored by other hospitals or organizations.
What is a protocol?
A protocol is a carefully designed study plan. The document says how the study will be done, how long it will last, who can be in the study, what safety plans are in place, how the results will be studied, and many more details. The information in a study's protocol is included in the consent and assent documents and helps explain what it means for your child to be in a study. The protocol, and the consent and assents, all have to be checked and approved by the IRB.
What is an IRB?
The IRB is a committee that reviews research plans and documents 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. The IRB is made up of experts, like doctors and nurses, as well as people like parents and members of the community.
What is the FDA?
FDA stands for Food and Drug Administration. The FDA monitors some research, including research on new medicines, therapies, or uses of medicines. After a research study is complete, the FDA approves medicines for standard medical care. Cancer.gov scientific review
What is randomization?
Some studies use randomization and it decides the treatment plan your child will get. Randomization means that the treatment your child gets is decided by chance. It's like flipping a coin or pulling numbers from a hat, except that a computer does it. This is to make sure that there are about the same number of people on each treatment plan. You and your investigator do not get to pick your child's treatment plan if they are on a study with randomization.
What is a placebo?
A placebo looks like a drug being used in a study, but does not contain the actual ingredients used to treat patients.
What does "expanded access" mean?
Most use of new medicine takes place in controlled clinical trials designed to determine how safe and effective the medicine is. Sometimes, patients do not qualify for these trials because of other health problems, age, or other factors. In these patients that may benefit from the medicine but do not qualify for the trials, the FDA has created special exceptions. These exceptions allow the makers of investigational medicines to provide "expanded access" use of the drug meaning, the drug may be able to be used outside of the clinical trial. These "Expanded access protocols" are only allowed if researchers are actively studying the new medicine or treatment in well-controlled studies, or all studies have been completed. There must also be evidence that the medicine may be an effective treatment in these patients, and finally cannot expose patients to unreasonable risks.
What is a "Treatment IND" or treatment protocol?
A treatment IND (Investigational New Drug) or treatment protocol is a relatively unrestricted study. Its main purpose is to provide access to a new medicine for people with a life-threatening or serious disease for which there is no good alternative treatment. A secondary purpose for a treatment IND is to obtain additional information about the medicine, especially its safety.
Does research involving children include special requirements?
Yes, there are special requirements in place to protect children in research. The IRB carefully reviews the risks and benefits to make sure that the research is safe for children. Since children are not old enough to provide their own consent to be in research studies, we get consent from a parent as well as "assent," or agreement, from the child as well. The assent will explain the study to them in terms that they can understand, and ask clearly if they want to be in that study or not. We only enroll children who give a clear "yes" to being a part of research.
Studies will sometimes use a safety monitor or safety board (usually a doctor who is not participating in the study) to keep an eye on research activities to make sure that they're safe for the children participating.
How is a "child" defined?
Generally, all people under the age of 18 are considered children. However, researchers may want to include children of a specific age range, or adults with a condition that began during childhood. The specific age range of study participants will be described in the study protocol.
How is parental permission involved in research?
For a child to take place in research at Cook Children's, we require both their permission and the permission of their parent or legal guardian. Before your child is enrolled in a study, a member of your research team has to get your consent, or permission. Before asking for your permission, a member of your research team will go over the study with you in detail and give you, your child, and your family time to consider if participating in that study is right for you.
How is a child's permission involved in research?
For a child to take place in research at Cook Children's, we require both their permission and the permission of their parent or legal guardian. Before your child is enrolled in a study, a member of your research team has to get their assent, or permission. Before asking if they want to be in the study, a member of your research team will go over the study with your child in terms that they can understand, and give them time to ask questions and consider whether or not they want to participate.
Will my child's information be shared?
Most studies will only share patient information in a "de-identified" form, meaning that your child's name, birthday, or any other information identifying them will be removed. Your research team will discuss exactly how all information will be used during the consent conference before you and your child decide to participate in the study.
Do participants get paid a stipend for being in a research study?
Each research study is different. Some studies pay participants for their time and effort related to taking part in a research study, and some do not. Stipends are given to cover the costs of parking, travel, childcare, lost wages, or time lost from work.
Do research studies pay for travel?
Some clinical trial sponsors will pay for participants' travel costs. Some studies may offer a stipend to participants that may cover transportation costs such as gas money or taxi services. Some sponsors of a research study may even pay for air travel.
Does participating in research cost anything?
In most cases, study participants do not have to pay for the costs of anything that is done only for the research study, including the investigational treatment and any special tests or extra doctor visits. However, study participants or their insurance companies are responsible for the cost of anything that is standard of care.
If my child is harmed while participating in research, will the cost of their medical care be covered?
Some studies have money to cover the cost of medical care if a child is harmed while participating in research. Some do not.
Investigational Drug Service
What is an Investigational Drug Service (IDS) Pharmacy?
The IDS Pharmacy is a service that employs pharmacy staff responsible for a variety of tasks required to prepare and dispense your child's study medication.
Who works in the Investigational Drug Service Pharmacy?
Two investigational pharmacists and an investigational drug service administrator.
What steps does the IDS pharmacy staff follow to prepare my child's study medication?
There are a number of steps involved in preparing to dispense your child's study medication. These steps may take between 20 minutes to 1 hour or more depending on the type of study. These steps are explained in further detail below.
- The IDS pharmacist receives a prescription for your child's study medication and reviews the order for accuracy. The pharmacist will verify the dose based on your child's height and weight, and according to the protocol. They will then review your child's medication history to verify there will be no interactions with other medications. Finally, they will review laboratory test results, as necessary.
- Once the order is verified, the study medication is entered into the computer system which creates a prescription label for your child's medication.
- If your child is taking a study medication by mouth, it will be prepared and labeled in the main pharmacy. If your child is receiving a study medication through an IV, it will be prepared and labeled in a special mixing area using sterile techniques.
- The pharmacist must then record every vial, tablet, capsule, or liquid medication into the investigational drug inventory log. This log is required to be provided to the pharmaceutical company sponsoring the study and the Food and Drug Administration (FDA) upon request. The IDS pharmacist spends several hours each week documenting inventory and dosages dispensed to patients on Drug Accountability Logs.
Why am I asked to return empty and partially used containers of my child's study medication?
We ask you to return empty and partially used containers in order to comply with requirements of the pharmaceutical company sponsor, as well as state and federal regulations regarding the accountability of study medications. These returns are recorded in the drug accountability log which is then provided to the regulatory agencies.
Why do studies use a placebo instead of actual drug in some patients?
A placebo is used to help researchers separate "placebo effects" from the actual effects of the drug or treatment being studied. This is explained in greater detail below. Our mind is a powerful thing and sometimes our own expectations and beliefs that a medication or treatment will make us feel better, can actually affect how our body responds to the treatment. In easier terms, "if we believe the treatment is going to make us feel better, then we will actually start to feel better." This phenomenon is called the "Placebo Effect" When studying a new medicine or intervention, it is important for researchers to compare these "placebo effects" to the effects seen from the actual drug. This comparison allows researchers to judge whether the actual drug being studied is effective.
Will I continue to receive a placebo if it is found that the study drug is more effective?
If during a study, an investigational medicine seems to work well, the researcher may stop using the placebo. This decision depends on many factors including the interventions being studied, the medicine, and the illness. These decisions will vary greatly from one study to another. You can ask the doctor this question when you are considering participation in a study.