The scientific establishment of a new therapeutic intervention for developmental conditions: practical and ethical principles.

BACKGROUND: Although the randomized controlled trial (RCT) is a major methodological breakthrough extending the limits of objectivity in clinical medical science, clinical trials of surgery have seldom included placebo surgery as a control, for ethical reasons. Especially in clinical studies intended eventually to establish a new intervention for developmental conditions, it has been recognized that there is huge examiner bias. In addition, the many miraculous cases that have been reported in nonsurgical open trials for developmental conditions and have eventually been evaluated as nonspecific positive outcomes in RCTs suggest that empirically promising interventions must be subjected to scientific scrutiny as soon as possible in the field of developmental conditions. APPLICATION TO PEDIATRIC NEUROSURGERY: Therefore, in childhood neurosurgery, clinical studies to establish a new therapeutic measure for developmental conditions should be designed as rigorously as possible using optimized scientific methods. The worldwide ethical guideline, the Declaration of Helsinki issued by the World Medical Association, can provide principles for the establishment of a new intervention in the treatment of a patient when proven therapeutic methods do not exist or methods used thus far have been ineffective. Physicians' discretion to use unproven or new therapeutic measures for such patients is approved in the presence of efforts of an ethical and scientific approach. Even if the measure is a very promising intervention, the research aspects must completely be demonstrated for informed consent and review by the ethical committee and the trial must be regarded as a clinical research. Especially when an RCT is not possible for ethical reasons, appropriate epidemiological data or animal experiments should suggest that the new measure is effective before a clinical trial. In a clinical setting, where neither epidemiological studies nor animal experimentation can be introduced, if necessary the researcher should collaborate with experts to obtain multidisciplinary justification for clinical testing.