Cookies on this website

We use cookies to ensure that we give you the best experience on our website. If you click 'Accept all cookies' we'll assume that you are happy to receive all cookies and you won't see this message again. If you click 'Reject all non-essential cookies' only necessary cookies providing core functionality such as security, network management, and accessibility will be enabled. Click 'Find out more' for information on how to change your cookie settings.

© 2015 Elsevier Inc. All rights reserved. Early clinical trials are pivotal steps in the humanization of a drug development project. Their design has to accomplish both regulatory aspects as well as scientific gain regarding the drug target, human mechanism of action, and viable biomarkers; they should ultimately support proof-of-concept as early as possible. This chapter details the basic characteristics of such trials and shows the opportunities elicited by smart designs of exploratory trials, exploratory investigational new drug (eIND) filing, microdosing studies, early decision making, adaptive trial design, and the combination of regulatory and exploratory trials. All this aims at accelerating proof-of-concept as a major value inflection point of drug development.

Original publication





Book title

Principles of Translational Science in Medicine: From Bench to Bedside: Second Edition

Publication Date



217 - 225