Masterstudiengang "Drug Regulatory Affairs"


The challenge of filing a drug application for an NCE for human use for the US and EU in parallel - An overview of the potential differences between the US and EU CTD dossiers and essential pre-application activities

Cordula Grandpierre-Verhees (Abschlußjahr: 2017)

Language: English
A medicinal product has to be authorised by the competent authority before it is placed on the market. The pharmaceutical industry is highly regulated with the goal of ensuring the quality, safety and efficacy of the medicinal product for protecting human health. In order to request a marketing authorisation of a new medicinal product, a dossier, structured in five modules, has to be submitted to the competent authorities. In the US and EU the mandatory dossier format is the Common Technical Document (CTD).
This master thesis gives an overview of the differences between the US and EU dossiers. The Module 1 is region-specific. The US and EU versions of this module are compared and the labelling, paediatric information, risk management and environmental risk assessment described in more detail. The differences between Modules 2 to 5, despite inter-regional alignment by the International Council for Harmonisation (ICH), are considered and highlighted.
Additionally this master thesis gives an overview of the required regulatory activities, starting approximately one and a half years before filing an Marketing Authorisation Application (MAA) for the EU via the Centralised Procedure (CP) and a New Drug Application (NDA) for the US for a New Chemical Entity (NCE).
Due to differences in culture and medical practices in the EU and US, the different therapeutic and regulatory guidelines have to be observed. The differences in the dossier are also the result of variances in the review processes of the EMA and FDA.
In the EU, the dossier is reviewed from "top to bottom" (Modules 1, 2, 3, 4, 5). First of all, the reviewers examine the overviews, then the summaries and at last the reports provided in Modules 3, 4 and 5. This is in contrast to the US, where the “bottom-up” (Modules 5, 4, 3, 2, 1) approach is applied. The FDA prefers to analyse raw data and to reach its own conclusions, not relying on the applicant’s argumentation.
The FDA requires an Integrated Summary of Safety (ISS), an Integrated Summary of Effectiveness (ISE) with datasets and, in addition datasets relating to the studies. The compilation of BIMO files for the pivotal studies are also mandatory.
Pages: 71, Annexes: 1 pages: 10