More Than One Way to Measure? A Casuistic Approach to Cancer Clinical Trials.

Testing new oncological treatments in the era of personalized medicine is raising many challenges to the current regulatory paradigm. In particular, randomized controlled trials (RCTs) have proved to be inadequate for testing targeted therapies. Nonetheless, the Food and Drug Administration (FDA) still requires them to grant market approval. This article questions the idea that regulatory decision-making can be reduced to sound statistical inferences. The author discusses the implications of tumor heterogeneity for regulatory assessment of new medications, considering the challenges that the current paradigm is facing and addressing criticisms that could explain its resistance to change. To overcome those criticisms, the author proposes implementing casuistic method into regulatory decision-making.