Clinical trials: The gold standard and so much more

Clinical trials are not intended just to assess treatmenteffects; they also provide a great deal of invaluableinformation, said Frederick Ferris III, MD, when he delivered theJackson Memorial Lecture during the opening session of theAmerican Academy of Ophthalmology annual meeting.

While having been termed as "indispensable ordeals," clinicaltrials can define the natural history of a disease, define therisk factors for disease progression, help standardizemethodology, and allow consideration of patients’ perspectives,according to Dr. Ferris, who is the clinical director of theNational Eye Institute, Bethesda, MD.

"Collaboration is the direction for the future as we strive toanswer increasingly difficult clinical questions," he emphasized.An example of this is the collaboration needed to addressphenotyping and sequencing, with a publicly available databasethe first step. In line with this, investigators need toestablish common definitions that will allow the combining ofepidemiologic and clinical trial data in the search for geneticrisk factors.

"It is apparent that the value of clinical trials goes far beyondthe assessment of the treatment effect," Dr. Ferris said. "Welearn about the natural history and the risk factors of thedisease, but equally importantly, the collaboration of clinicalinvestigators as they develop and carry out protocols facilitatesincorporation of new ideas into the practice of medicine."

Support for the vision research community will be necessary toadvance the rich tradition of clinical trials in ophthalmology.The rewards of this type of research are well worth theinvestment.

"Clinical trials remain the indispensable ordeal," Dr. Ferrisconcluded.