Masterstudiengang "Drug Regulatory Affairs"


Voluntary post-authorisation studies: differentiation, regulatory requirements and value ***

Andrea Brischle (Abschlußjahr: 2019)

Language: English
At the time of the launch of a medicinal product, it is not possible to predict to what extent the efficacy and safety profile known from the controlled clinical trials will be confirmed in everyday clinical practice. The launch is therefore not the end of the clinical development of a medicinal product, but merely a milestone. In terms of gaining knowledge after marketing authorisation, voluntary clinical studies are an indispensable tool. Currently, clinical trials as well as non-interventional studies are the possible options for conducting clinical studies after marketing authorisation. In the future, low-intervention clinical trials will be an additional option. In this thesis, based on the legal definitions of the different types of studies, their sometimes difficult demarcation process is outlined. On this basis, a comprehensive overview of the regulatory requirements level, which varies greatly from study type to study type, is compiled and linked to the study type-related risks. The focus here is limited to clinical studies that are conducted voluntarily by the responsible party after marketing authorisation in the European Economic Area. Clinical studies imposed by regulatory authorities are explicitly not part of this thesis. Furthermore, the focus is limited to the extent that this thesis deals exclusively with the legal requirements valid throughout the European Economic Area and the additional requirements valid in Germany. In addition, this thesis provides an overview of the publicly accessible databases on clinical studies. In this context, the different structure and contents of the EU Clinical Trials Register, PharmNet.Bund, EU PAS Register and the databases of BfArM and PEI on observational studies ('Anwendungsbeobachtungen') are discussed. Besides the general consideration of the topic, data from post-authorisation studies voluntarily started in Germany between 01 January 2014 and 01 January 2019 will be examined. In terms of quantity, observational studies account for by far the largest proportion of these studies. Finally, the strengths and weaknesses of the different types of studies are discussed and it is shown that they should be seen as complementary. The choice of the appropriate type of study depends on the question to be clarified.
pages: 70, Annexes: 2, pages: 10