Over the last decade, many exciting developments and treatment approaches for kidney disease have emerged.1 There are many underlying reasons for this, including rapid advances in understanding the pathogenesis for specific kidney diseases. The increased availability of high-throughput genetic sequencing, single-cell gene expression analysis, and ever more powerful bioinformatic techniques are just a few of the contributing factors. Equally important are efficient yet rigorous methods to conduct clinical trials to assure the safety and efficacy of new treatments in a cost-efficient and timely manner.