By Lindsay McNair, MD, MPH, MSB
For many years, the randomized, parallel-group, double-blinded clinical trial was considered the optimal study design to assess the efficacy and safety of new investigational therapies. As the science behind therapeutic interventions has deepened and grown, the clinical trial designs through which those interventions can be best tested have evolved as well.
One of the therapeutic areas in which this has been seen most dramatically is oncology. “Precision medicine” includes the development of agents targeted to specific molecular profiles, including specific genetic mutations that may be driving cancer growth. These genetic mutations may appear in more than one type of cancer. For example, cancers with the TRK fusion protein may be found in the colon, breast, or lung. To study therapies directed against these specific abnormalities, it may make sense to include anyone with the target abnormality in the trial population, regardless of the location of their cancer.