Journal: Nature Clinical Practice Rheumatology. (2007) 3, 59
Authors: Elizabeth D Mellins, Lisa G Rider
[Article has no abstract. Opening text supplied instead.]
Pediatric rheumatologists are often forced to make clinical decisions in the absence of robust evidence about the most effective therapies for their patients. A number of factors have contributed to the relative lack of practice-guiding information.
n Of an estimated 200,000 children with rheumatic diseases in the U.S. about 30 percent are under the care of pediatric rheumatologists, and it is likely that an equal number see adult rheumatologists (Mayer ML et al.  Arthritis Rheum 49: 759–765).
n With this dispersion of a relatively small patient population, adequately-powered studies can rarely be accomplished at a single site.
n The number of investigators appropriately positioned to carry out clinical research in pediatric rheumatology is also small. In total, there are 215 board-certified pediatric rheumatologists in the U.S.; 13 states don't have a pediatric rheumatologist at all.
n Many pediatric rheumatologists are 'one-person divisions' with substantial clinical responsibility.
n In addition to these barriers to investigator-initiated studies, pharmaceutical companies have historically seen the pediatric rheumatology population as unattractively small or too high risk.
n FDA policies to stimulate studies in children apply to pediatric forms of non-orphan diseases; only polyarticular juvenile idiopathic arthritis (JIA) qualifies and has benefited from increased industry-sponsored trials.