[Main characteristics of the new authorisation procedure for clinical trials with medicinal products according to regulation (EU) no 536/2014 and the cooperation between the member states]. / Grundzüge des neuen Genehmigungsverfahrens für klinische Arzneimittelprüfungen im Rahmen der Verordnung (EU) Nr. 536/2014 und der Zusammenarbeit zwischen den Mitgliedstaaten.

With Regulation (EU) No. 536/2014 on clinical trials on medicinal products for human use, which became applicable on 31 January 2022, full harmonisation of the authorisation and monitoring procedures of clinical trials with medicinal products in the European Union (EU) and the European Economic Area (EEA) has been achieved. In addition to an entirely paperless application procedure, communication between all parties involved is done through the Clinical Trials Information System (CTIS), which was developed specifically for the Regulation and through which all non-proprietary information and content of the clinical trial application and results are also made available to the public. As was already the case under the old legal framework, the authorisation of a clinical trial is granted by each Member State concerned; however, the assessment of the common part of the dossier of a clinical trial that is conducted in more than one Member State is jointly done by the respective Member States under the coordinating lead of a reporting Member State. The present article outlines the authorisation procedure with its deadline concept and addresses further aspects of the Regulation, such as details on the protection of the trial subjects, safety reporting and transparency rules.

[The EU Portal: Implementation, importance, and features]. / Das EU-Portal: Aufgaben, Bedeutung, Funktion.

The European Medicines Agency (EMA) is developing a web-based EU portal with a database "at Union level as a single entry point for the submission of data and information relating to clinical trials in accordance with" the new EU regulation No. 536/2014. The specifications are mostly published, but some documents are still missing. Because the project is integrated and has dependencies on other projects, this could result in other specification upgrades. The IT solution is under ongoing development until project completion in quarter III of 2019. The EU Portal and the database will be audited. If the audit is successful, the new regulation will come into force in October 2018. The use of the EU Portal will then be mandatory with some transition rules. The software development of the portal is restricted to the regulation and the derived requirements. It is not possible to implement any national requirements. We describe in this paper the current key functionalities of the portal and try to derive requirements for a national IT system.On 16.06.2017 the EMA Management Board announced that the development of the new portal has been delayed and it is foreseen that the new regulation can come into effect in 2019 at the earliest. The press release can be found here: http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2017/06/news_detail_002764.jsp%26mid=WC0b01ac058004d5c1 (accessed: 12.07.2017).

Clinical Development and Commercialization of Advanced Therapy Medicinal Products in the European Union: How Are the Product Pipeline and Regulatory Framework Evolving?

The research and development of advanced therapy medicinal products (ATMPs) has been active in Europe and worldwide during recent years. Yet, the number of licensed products remains low. The main expected legal change in the near future in the European Union (EU) concerns the regulation on clinical trials (536/2014), which will come into force in 2018. With this new framework, a more harmonized and swift process for approval of clinical trials is anticipated, which is expected to support the entry of new innovations into the EU market. A survey on ATMPs in clinical trials during 2010-2015 in the EU was conducted in order to study the trends of ATMP development since the earlier survey published in 2012. According to the results, the number of clinical trials using ATMPs is slowly increasing in the EU. Yet, the focus is still in early development, and the projects are mainly carried out by small and medium-sized enterprises, academia, and hospitals. Oncology is the main area of clinical development. Yet, the balance between cell-based products and gene therapy medicinal products in this area may be changing in the future due to the new T-cell technologies. Many limitations and challenges are identified for ATMP development, requiring proportionate regulatory requirements. On the other hand, for such a novel field, the developers should be active in considering possible constraints and actively engage with authorities to look for solutions. This article provides up to-date information on forthcoming regulatory improvements and discusses the main challenges hampering the commercialization of ATMPs in the EU.