Translation of academic medicinal products towards clinical practice

Abstract

This

thesis focuses on drug development and drug manufacturing for special (small)

patient populations in academic institutions. Reaching the patient via the

commercial route of marketing approval (licensing) does mostly not apply to these academic products. The first category of these drugs, the advanced therapy medicinal products (ATMPs), belongs to a new complex group of medicinal products, of which the process of acquiring marketing authorization is relatively unexplored. The second category is the unlicensed tailor made pharmaceutical preparations, which have been used in clinical care in hospitals for decades. Despite long-term experience, it seems that commercialization of these pharmaceutical preparations, via licensing, has never been the main goal for academia. The aim of this thesis is to explore the development field of these academic medicinal products and the role of the academic developers and manufacturers for providing these products for regular patient care whether commercialized or not.