Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Patient Registries as Specific Obligations for Marketing Approvals of Orphan Drugs in the European Community ***

Dr. Barbara Siebertz (Abschlußjahr: 2015)

Summary
The approval of a marketing authorisation for orphan medicinal products (OMP) and for advanced therapies of medicinal products (ATMP) of orphan diseases increasingly includes the establishment of patient registries. This is either already part of the Risk Management Plan already included in the Marketing Authorisation Application (MAA) or an obligation by the Agency, since orphan medicinal products follow the centralised procedure. The reason for this obligation is the authorisation of the product despite incomplete clinical data, which results from on the major challenges of orphan diseases, their low prevalence. For the purpose of epidemiology and post-marketing surveillance, the establishment and implementation of registries and databases actively support the possibility of delivering significant data, which clearly represents the risk versus the benefit of the new medicinal product on the market.
Since 1999 the European Community actively delivered solutions to the challenging situation of orphan drugs. The first step was the implementation of the European legislation regulation 141/2000 in 1999 and the commission regulation No 847/2000 in 2000. The EC uses two regulatory procedures for marketing approval of orphan medicinal products, which are "conditional marketing authorisation" or "approval under exceptional circumstances".
With the implementation of directive 2011/24/EC it became clear that it was necessary to develop a closer interaction between health care systems in all Member States. The overall purpose was the creation of a European Reference Network and a European register platform accompanied by the development of guidance to this platform. Development of standards in data collection and databases for pre- and post-marketing studies are first  steps towards a European harmonisation process in establishing a platform for patient registries. One of the first important steps was achieved with the creation and launch of the ENCePP E-Register (EU-PAS Register).
Use of the EU-PAS register is free of charge, voluntary for MAHs conducting voluntary PASS´s, but obligatory for marketing authorisations with an SOB to implement patient registries.
This master thesis provides information and guidance for the establishment of patient registries, combined with a reflection of the legal basis according to the latest legislation in the EC and compares the process and efficacy in the establishment of patient registries of four medicinal products related to orphan diseases.
Pages: 54, Annexes: 3, pages: 16