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Patient safety

Patient Safety in clinical trials

There are certain rules that help ensure the safety and ethics of clinical trials. Especially in the case of a child participating in a clinical trial, the rules are extra strict. Before enrolling in a study, your doctor will discuss elements of the trial such as: the length of the study, the number and interval of visits required, the lab work and other tests or questionnaires required during the study. Any study related physical exam or outcome measures will be explained here as well.

Below, more information on:

  • Giving consent & assent
  • Randomization
  • Trial safety
  • Trial timeline

Giving consent & assent

Your principal investigator will talk you through the consent process. This can be done by telephone call or in person. In this talk, you will receive more information about the trial itself and alternative options, what kind of tests your child has to do, and the duration and intensity of the clinical trial. Possible risks and benefits are also discussed. Make sure you receive this information in a language you can understand, and discuss this later with another peer, your general practitioner, or your patient organization.

After this conversation, you will receive an informed consent form. This contains all the information that you just discussed and describes which kind of patient data will be collected and how this medical record will be stored. You can always ask additional questions to the principal investigators, members of the research team or someone of your patient organization. Sometimes, this consent form is dynamic, meaning that you always decide how your medical information should be used.

If you have read this document through and agree with this, you sign the form. Although this is final, you are free to withdraw from the clinical trial at any time. The study consent (for a parent, if participant will be under age 18) and assent (for participants age 7 -17) will be described, mailed and later discussed prior to enrolment.

Legally, children are not able to give consent until they turn 18. Instead, they are asked for their assent: they agree to take part. Children must be old enough to understand the trial and what they have to do. They will receive this information from the research team. Additionally, this app is an easy-going way to learn what is going to happen when a child participates in a clinical trial.

Randomization

In most cases, a clinical trial is randomized. This means patients are placed in a specific group, usually automated, to make sure the different groups are as similar as possible. Each group than receives a different kind of treatment (active compound or placebo/standard treatment). This is called the control group. Researchers randomize trails to ensure they have no bias that could affect the results.

A placebo may only be used when no standard treatment is available. From an ethical perspective you cannot withdraw care according to the latest Standards of Care and it is unethical to give a placebo to a group of patients who are in need for treatment, so ethics committees will not grant permission to trials designed this way.

More information about:

Trial Safety

Long before the clinical trial starts, experts have reviewed the protocol to make sure the trial is safe and justified for participants to enrol in. In most cases, there is a board that reviews protocols before the study can begin. The board closely monitors the progress of the trial and its outcome. This board is capable to change the protocol, or even stop the trial for the sake of the patients’ safety.

The European Medicines Agency (EMA) in Europe, and the Food and Drug Administration (FDA) in the United States approve new drugs before they can be sold. They protect people in research studies and ensure the data from the trial is used correctly. The can also decide to remove researchers from conducting trials when the rules are not followed.

Trial Timeline

Most of the time, clinical trials run from the beginning to end. However, a clinical trial can be stopped early for several reasons:

  • A new study answers the research question
  • Side effects are too severe, so the harms outweigh the benefits
  • There is clear evidence the drug is effective, trial can be stopped to make treatment widely available
  • There are not enough patients recruited to conduct the study

In rare cases, an extension can be asked if these criteria are not met yet. A committee has to review this proposal.

After the trial has ended, it’s not self-evident that study participants will have access to the treatment, or if this treatment is being reimbursed in their country or by their government. In some cases, compassionate use enables access to the treatment after a trial has ended. This is not always the case and should be made clear before enrolling in the study.

European Neuromuscular Centre (ENMC)

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