Discussion of the papers by Niels Keiding and Nanny Wermuth

Abstract

Problems of clinical trials associated with human beings are discussed. They are characterized by very inhomogeneous and limited numbers of samples, and by periods of observation too short to allow efficacy and safety to be evaluated. Avoiding human error associated with subjective measures and ensuring the generalizability of the result of a limited trial to the true target population are crucial. The regulatory science is therefore an important factor in making appropriate decisions and gaining maximal benefit from insufficient information.