Statistical Challenges in the Design and Analyses of Clinical Trials in Solid Organ Transplantation
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*LaRee Ann Tracy, FDA/CDER/OB/DB4 

Keywords: solid organ tranplantation, immunosuppressant, clinical trial issues

The evolution and improvement of immunosuppressive regimens in organ transplantation has led to improved patient and graft survival rates and reduced rates of acute rejection; however, these improvements may be leading to greater incidence of severe adverse events, such as infections, nephrotoxicity and diabetes. The evaluation of new regimens therefore requires detailed risk/benefit analyses and consideration of more relevant clinical outcomes. Additionally, there is tremendous variation in treatment combinations, doses, and concentration monitoring leading to challenges in designing and interpreting active-controlled clinical trials. Lastly, recent failures of confirmatory clinical trials have raised concern regarding the absence of sufficient phase II dose-finding. This talk will outline the critical issues surrounding clinical trials in organ transplantation with case examples.