PET/CT in cancer: moderate sample sizes may suffice to justify replacement of a regional gold standard

PURPOSE: For certain cancer indications, the current patient evaluation strategy is a perfect but locally restricted gold standard procedure. If positron emission tomography/computed tomography (PET/CT) can be shown to be reliable within the gold standard region and if it can be argued that PET/CT also performs well in adjacent areas, then sample sizes in accuracy studies can be reduced. PROCEDURES: Traditional standard power calculations for demonstrating sensitivities of both 80% and 90% are shown. The argument is then described in general terms and demonstrated by an ongoing study of metastasized prostate cancer. RESULTS: An added value in accuracy of PET/CT in adjacent areas can outweigh a downsized target level of accuracy in the gold standard region, justifying smaller sample sizes. CONCLUSIONS: If PET/CT provides an accuracy benefit in adjacent regions, then sample sizes can be reduced and the conduct of trials accelerated, leading to earlier decisions on the use of PET/CT while exposing fewer patients and reducing overall costs.