Controlled clinical trials are now necessary for the approval of new therapies. Besides academic centers and large pharmaceutical companies, individual practicing physicians may also be involved in clinical trials. This clinical summary reviews the basics of clinical trials, including design and methodology, with special emphasis on neurologic disorders. The role of the United States government in the database of clinical trials is described, along with problems and limitations of clinical trials.
Clinical trials are essential for new drug development as evidence of efficacy and safety is required for approval by regulatory authorities.
Biomarkers and pharmacogenomics are playing an important role in the design and monitoring of clinical trials.
Basic methods of clinical trials are applicable in neurology, but there may be specific problems associated with clinical trials of disorders such as multiple sclerosis.
Some problems with clinical trials, such as the lack of publication of negative results, still need to be resolved.