Deciding to take part in clinical trials is something only you can decide with information from the study doctor (investigator) and team. There are possible benefits as well as drawbacks so you should discuss these and any concerns fully before making your decision.
Participation allows you the benefit of a treatment option that is not otherwise available. Your participation allows you an active role in a decision that affects your life or the life of your child. You have a chance to help others by helping science advance.
Unknown Side Effects and Risks
There is always a chance the treatment or medicine will not work. Even if side effects are known, you may have other side effects that are not known by the study team at the time you start the study. You may need more frequent monitoring than is called for in the trial, depending on your response and possible side effects.
This is a process used in some trials to prevent favoritism with one treatment versus another. One group receives the actual study treatment or medication, while the other will receive the most widely accepted treatment (standard approved treatment). Comparing the results from these two groups often shows which treatment is more effective or has fewer side effects. Before you decide, you should understand your chance of being in either group is about equal, like the flip of a coin. The doctor does not decide on what treatment or drug you get, it is usually done randomly by a study statistician or pharmacist.
Placebos, which are inactive ‘look-alike’ drugs, are rarely used in primary immunodeficiency drug studies, but there is the possibility that you may not be selected to be in the group receiving the actual drug and will not know until later in the trial.
Your Rights as a Research Subject
As a participant in any kind of research study, you have rights. If you approached about participating in a study, you can decide to participate or not without any effect on your regular care. If you decide to participate, you will be told about important risks and benefits that are known about what is being tested. You will learn how long participation will take and what is expected of you, including any costs, or if you will receive any payment for participating.
As a research volunteer, there are also benefits, such as treatment with experimental medications not available elsewhere, the opportunity to learn more about an illness and how to take care of it, access to care from expert physicians in the field who are familiar with the most advanced treatment available, possible care and medication at no cost to you, and the satisfaction of helping others by contributing to medical knowledge.
All research trials have supervision and monitoring by an Institutional Review Boards (IRB). This board is made up of doctors, nurses and non-medical people who make sure that the research is fair and ethical and that the risks of being in the study are not greater than the expected benefit. They closely monitor the process of the trial from beginning to end. Additional supervision for some trials is the Data Safety Monitoring Board (DSMB), an independent group made up of statisticians, physicians and other experts. The DSMB makes sure that risks are reduced as much as possible, and that the study design and data are sound. They have the ability to stop a trial if safety concerns arise.
You can withdraw from any research protocol at any time without any consequences to you or your child’s care. Before you are given any new drug or treatment, you will always be asked to review and sign a written informed consent. This is a legal document that says you give your permission to be involved in the research and understand the risk and benefits. Though some risks may not be known yet for new products, you should only sign the consent after all the known risks and benefits have been explained to you and all of your questions have been answered.
Once you or your child has decided to participate in a study, you are always considered a volunteer and can withdraw at anytime. You can continue to ask questions and be assured your privacy is maintained. Any publications that result from the study will protect your or your child’ s identity and medical information.
Each study has its own entry criteria. Before you decide to participate, it is important that you fully understand the reason for the study and the expected outcomes. The investigator will explain what the expectations are for you as a participant and fully explain the risks and benefits. Once all of your questions have been answered to your satisfaction, you will be asked to sign the consent form. Often, children age 7 and older will be asked to sign an assent form, a similar document to the informed consent, written in age-appropriate language. Your child will not be forced to participate. You may ask for the consent form to be translated into your language or ask for an interpreter to help with understanding the consent. You will be given a copy of the signed consent form.
Even after you sign a consent form, you may still withdraw at any time without consequences to your care. If you have any further questions, you have options to contact: the physician, your nurse, the patient advocate of your institution and the IRB for answers.