How are Clinical Trials Designed and Reviewed?
The larger the organization carrying out the trial, the greater the review of the plan of therapy.
For example, a cooperative group or consortium trial that involves multiple hospitals and research centers would be developed and reviewed by many pediatric oncologists, surgeons, pathologists, radiation oncologists, nurses, statisticians, and patient advocates in the organization, as well as by the National Cancer Institute's Cancer Treatment Evaluation Program (CTEP), and by Institutional Review Boards (IRBs) responsible for protection of human subjects enrolling in clinical trials.
IRB review ensures that the rights of the patient will be upheld and protected and that the patient will not be exposed to any unnecessary or extreme risk if he/she agrees to participate in the clinical trial.
The reviews of clinical trials in high-risk neuroblastoma address a number of questions, including :
1. Based on pre-clinical and clinical data, is the therapy to be tested in the trial likely to be effective against the disease?
2. Is the risk of serious toxicity during therapy appropriately balanced by the potential benefit to the patient?
3. What is known about the risks of long-term complications (called "late effects")? Examples of such risks include decreased intellectual ability, impaired growth and development, increased risk of cancers other than neuroblastoma, and hearing loss.
In the case of a new therapy, these risks may be relatively unknown compared to standard therapy. When there are data to suggest approaches to minimize such risks (without adding other risks, such as therapy failure), does the clinical trial employ such approaches?
4. Is the option of being treated with a known standard therapy available to those patients who choose not to enter onto the clinical trial?
The above questions are appropriate for parents to address with their oncologist when evaluating any therapeutic approach for neuroblastoma.