CARES Foundation does not counsel individual patients either for or against participation in any specific research study.  Prospective volunteers should always carefully review the study’s informed consent documentation and discuss the pros and cons of their participation with trusted advisers, including their health care providers and family members.  For more information on research participation, check out https://www.hhs.gov/ohrp/education-and-outreach/about-research-participation/index.html

Reprinted with permission from the National Organization of Rare Diseases Newsletter

A study from the Rainbow Babies and Children’s Hospital in Cleveland, Ohio reveals that parents of children who participate in clinical trials generally do not understand the concept of experimental treatments. The study of 137 parents of children with leukemia was published in JAMA. When children were randomly assigned to an experimental treatment or the standard treatment, even though this was explained to them 50 percent of the parents did not fully understand the concept. Nor did they understand that the effectiveness of experimental treatments was unproven, nor that a computer and not a doctor would make the decision about which treatment the child would get. Parents are supposed to give their informed consent before a child participates in research, but apparently many parents continue to expect that their child will benefit from the experimental treatment.

What to Expect from Clinical Trials

When a person considers participating in a clinical trial, there are several things he or she should be aware of, in order to have realistic expectations and adequate knowledge of possible risks and benefits:

• What is the purpose of the trial? Is the intent to find a cure, or simply a treatment, or perhaps preventive measures or a diagnostic test? Don’t sign up for a clinical trial unless your expectations are in line with the study’s objective.
• Is an experimental clinical trial appropriate for you? Have you tried the standard treatments and failed to benefit from them? Are you willing to try a treatment for which little is known, rather than try a treatment that has risks and possible benefits that are well understood?
• What are the known risks and benefits of the experimental treatment? How many people have been in the experiment before you? Did they experience any harm?
• Is the experimental product only slightly different from other available treatments (e.g., a slightly revised version of an existing drug), or is it a completely new approach to treatment?
• Who is paying for the study? Is it supported by government funds or a commercial firm? Does your doctor have a commercial interest in the therapy (e.g., a patent, royalties, etc.)? Note: Some universities have “Conflict of Interest” committees who review these factors and decide whether a commercial conflict may influence the outcome of a study.
• Is this a “placebo controlled” trial, or will the experimental treatment be compared to the standard treatment for your disease? Is there a chance that you will receive the placebo and if so, how much risk does this represent?
• If you are harmed by the experimental treatment, who will pay for your medical care?
• What will happen when the study is finished? Will you be able to continue using the treatment, or will it be stopped even though you feel you are benefiting from the product? Will the manufacturer promise you can have continued access?
• While the vast majority of experimental treatments are free, will you or your insurance be liable for any costs related to your medical care while you are participating in the clinical trial?