Study of Infrequent Tumors: Sustained Commitment Necessary.

Abstract

Appropriate development of clinical trials has been essential to improved cancer survival rates. In the hierarchy of trial design, the randomized controlled trial with classic statistics has the greatestweight; however, such trials are impractical, if not impossible in rare cancers. Other research methods may be more applicable to the study of rare tumors. These include the relaxationof statistical error ratesor selection of population or outcome measures to minimize sample size. One could argue that all pediatric tumors are rare tumors. There has been a continued emphasis on the adolescent and young adult age group and the disturbing fact that, in some instances, adolescent and young adult patients may have inferior outcomes compared with children and adults with the same disease. One complicating factor may be that when tumors cross the boundaries of childhood, adolescence, and young adulthood, the distribution of cases to different providers further reduces the patient pool available to different specialists. Too often, barriers exist between these groups that limit collaboration. Finally, there are pediatric tumors that pediatric oncologists label as so infrequent that they are never part of national clinical trials.