Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Companion Diagnostics for Personalised Medicines – the Regulatory Framework for their (co-) Registration in the EU and the USA ***

Dr. Sandra Hennig (Abschlußjahr: 2017)

Summary
Language: English
Predictive, preventive, and precision care based on an individual patient’s need is often summarised by the term "personalised medicine". An integral tool for personalised medicine is the identification of clinically relevant biomarkers, which allow for the development of safe and effective therapeutic products for specific patient populations and/or the stratification of the patient population for existing treatments. These assays are described by the term "companion diagnostics".
The regulatory frameworks for the registration of companion diagnostics differ in the European Union and the United States of America. The FDA recognised early the importance of companion diagnostics for therapeutics. Due to their associated risks, companion diagnostics are classified as Class III devices requiring a premarket approval. In the USA, the co-registration of therapeutics and corresponding companion diagnostics is favoured and guidance on the co-development of therapeutics and corresponding diagnostics is available.
In the European Union in-vitro diagnostics have been regulated by Directive 98/97/EC where the term companion diagnostic has not been defined. For this kind of assay rules for general/other in-vitro diagnostics have been applied. Recently a new Regulation - (EU) 2017/746 - came into force. The regulation not only provides a definition but also clarifies the requirements for the conformity assessment by the manufacturer supported by a notified body.
In this thesis the regulatory frameworks for companion diagnostics in the European Union and US are summarised and the differences outlined. Since EU legislation is currently in a transition phase, the registration of in-vitro (companion) diagnostics as per Directive 98/97/EC and Regulation (EU) 2017/746 is illustrated. The main principles of the US framework and examples of companion diagnostics and therapeutics which were co-approved by the FDA are presented. Differences and the impact of the implementation of the new legislation on in-vitro diagnostic medical devices in the European Union are discussed and an overview on regulatory considerations for their registration in both regions is provided.
Pages: 63, Annexes: -