FAQ: Frequently Asked Questions

Clinical research is medical research that studies people to understand health and disease. Clinical research helps improve the way doctors treat and prevent illness. Through clinical research, researchers learn:

• How the body works
• How illness develops in people, such as how diseases get better or worse over time
• How the body handles a possible treatment
• Which behaviors help people stay healthy and prevent illness, and which behaviors raise the chance of illness

Clinical trials are research studies that test a medical, surgical, or behavioral intervention in people. These trials are the primary way that researchers determine if a new form of treatment or prevention, such as a new drug, diet, or medical device (for example, a pacemaker), is safe and effective in people. Often, a clinical trial is designed to learn if a new treatment is more effective or has less harmful side effects than existing treatments.

For children with rare conditions or who have not benefited from standard treatment options, participating in a clinical trial may provide an alternative that is not currently available to them. Additional benefits include getting expert medical care at leading health care facilities and helping others by contributing to medical research.

Prior to participating in any clinical trial, you will be asked for your permission to enroll your child and, depending on the age of your child, your child’s agreement, or assent, will be required. Below, we have listed the top points you should consider when determining if a trial is right for your child. If you have any questions, please email us and a Nicklaus Children’s Research Institute professional will get in touch with you.

• Inclusion and Exclusion Criteria: In clinical research, specific groups of relatively uniform patient populations need to be enrolled. Inclusion and exclusion criteria define who can be included or excluded from the study. The inclusion criteria identify the study population in a consistent, reliable, uniform, and objective manner. The exclusion criteria include factors or characteristics that make the recruited population ineligible for the study. For example, patients with liver disease would be excluded if the side effects of their disease would impact the results of the study.
• Benefits and Risks: All clinical trials are intended to help future patients with the disease being investigated, however, there is no guarantee the study participants will receive benefit for participating and may carry some risk. Make sure you fully understand the parts of the research designed to potentially benefit or improve your child’s condition, and the potential risks associated with the trial.
• Experimental vs Standard of Care: All clinical trials will have an experimental component, and most will include portions that are standard care. Experimental elements are the focal point of the clinical trial and will have some level of uncertainty about their effectiveness or potential side effects. Standard of care components are procedures, medications, or physician assessments that are a currently accepted practice for the care of your child.
• Cost: The amount of financial coverage will vary depending on the complexity of the trial. Be sure that you understand any potential financial obligations that you may be responsible for as part of your child’s participation in the trial. Typically, these will be standard care that your child would be receiving regardless of the trial.

Below, you will find potential benefits of participating in research. While these are typical across trials, there are trial-specific benefits outlined in each trial’s description, which you should review before giving your permission for your child to participate.

• Inclusion and Exclusion Criteria: In clinical research, specific groups of relatively uniform patient populations need to be enrolled. Inclusion and exclusion criteria define who can be included or excluded from the study. The inclusion criteria identify the study population in a consistent, reliable, uniform, and objective manner. The exclusion criteria include factors or characteristics that make the recruited population ineligible for the study. For example, patients with liver disease would be excluded if the side effects of their disease would impact the results of the study.
• Benefits and Risks: All clinical trials are intended to help future patients with the disease being investigated, however, there is no guarantee the study participants will receive benefit for participating and may carry some risk. Make sure you fully understand the parts of the research designed to potentially benefit or improve your child’s condition, and the potential risks associated with the trial.
• Experimental vs Standard of Care: All clinical trials will have an experimental component, and most will include portions that are standard care. Experimental elements are the focal point of the clinical trial and will have some level of uncertainty about their effectiveness or potential side effects. Standard of care components are procedures, medications, or physician assessments that are a currently accepted practice for the care of your child.
• Cost: The amount of financial coverage will vary depending on the complexity of the trial. Be sure that you understand any potential financial obligations that you may be responsible for as part of your child’s participation in the trial. Typically, these will be standard care that your child would be receiving regardless of the trial.

Phases of a clinical trial refers to the process of drug development regulated by the Food and Drug Administration (FDA). When a new drug is ready to be assessed using human participants, after initial laboratory testing, it will undergo potentially five phases of research.

• Pilot: A pilot study is a small-scale clinical trial that is conducted before a larger-scale study to assess the feasibility and acceptability of a new treatment or method. This phase of a clinical trial is not always required.
• Phase I (Phase 1): Phase I trials enroll 20-100 subjects for a short duration of several months. The purpose of trials in this phase of development is to assess the safety and appropriate dosage of the new drug. Typically, drugs in this stage of development will not be offered to pediatric participants unless the condition is life threatening and there are limited alternative treatments, the medication is meant for pediatric patients only, or preliminary safety data has been assessed in adult subjects. Prior to conducting a phase I trial, drugs have been approved to be assessed in humans after testing in a laboratory setting.
• Phase II (Phase 2): Phase II trials enroll several hundred subjects typically for a longer time period of months to 2 years. The purpose of clinical trials in this phase of development is to assess its effectiveness in treating the condition in question and identifying any unanticipated side effects. Prior to conducting a phase II trial, the safety and dosage of a new drug has already been assessed in a phase I clinical trial.
• Phase III (Phase 3): Phase III trials enroll hundreds to thousands of subjects typically for a period of 1-4 years. The purpose of trials in this phase of development is to continue monitoring the effectiveness of the drug in treating the condition in question and to screen for any adverse effects. Prior to conducting a phase III clinical trial, the investigational drug’s safety, dosage, effectiveness, and side effects will have been assessed during previous phases.
• Phase IV (Phase 4): Often referred to as Post Market Safety Monitoring, Phase IV trials typically enroll several thousand subjects and will continue for years and often decades. The purpose of this phase of development is to monitor the drug’s long-term safety and effectiveness. Drugs in this phase of development are available on the market and have been proven to be reasonably safe and effective during previous phases.