32nd EURORDIS Round Table of Companies Workshop
The impact of the EU regulatory network strategy 2020-2025 on the development of orphan medicines
20-21 October 2021
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The pandemic has changed all our lives, but it has also led to many new lessons that can hopefully be of benefit for generations to come. Those lessons will inform and shape every aspect of healthcare delivery in the coming years, which were already set for major changes long in the planning before the pandemic was even imagined. Not least in the regulation of the sector. The latest European medicines agencies network strategy to 2025 was published by EMA and the HMA at the end of last year and has set out six priority focus areas which are in line with the earlier Pharmaceutical Strategy for Europe from the European Commission that formed the basis of our roundtable meeting earlier this year in March.
These are both complimented by EMA’s own Regulatory Science Strategy to 2025, published earlier last year and after a lengthy and rigorous consultation process. As we might say, “You wait 5 years for a regulatory strategy and then three come along all at once!”
These three documents all herald important changes in the way that healthcare is delivered right through to the end of the decade and not just the end date of the title. The planned changes will have a profound impact on the development of treatments for rare diseases and will affect all of us. Some of those changes may be relatively predictable, such as the entry into force of the clinical trials regulation following the commencement of the Clinical Trials Information System (CTIS) single point clinical trial application system in just a few months time, but they will nevertheless lead to fundamental changes in the way trials are conducted throughout the EU and beyond. EMA has assembled four task forces to oversee their activities in pursuance of these strategies and an introduction of and discussion on three of these highly interconnecting task forces will form the basis of the workshop.
The Regulatory Science and Innovation Task Force will set the agenda for the rest of the session with a special focus on research and innovation that emphasises collaborative working and a strong commitment to greater use of real world evidence (RWE). This leads naturally into the second session focusing on the work of the Data Analytics and Methods task force and with particular attention given to the Data Analysis and Real World Interrogation Network (DARWIN) which aims to provide a consistent and robust means to access the vast wealth of RWE data sources that exist across the EU.
Data has the potential to transform healthcare, but also to complicate it and we must be cautious to ensure that the correct tools are applied to allow us to benefit and not suffer from the profusion of data that will be available to us in the coming years. At the heart of any discussion of data in rare diseases, indeed, any discussion at all about rare diseases in Europe, is the role and potential of European Reference Networks (ERNs), who will unquestionably be one of the major providers of data and we aim to examine this role in our discussion.
On Day 2 the focus shifts to clinical trials (the subject of one of our first ever ERTC meetings) with a discussion of the proposed changes in EU CT oversight and practice, and bringing into the debate the proposed revisions for ICHe6 good clinical practice guidelines on trials and data sources. The session will end with an examination of the performance of adaptive trial designs, their benefits and challenges for both regulators and payers.
This is a very busy schedule but it can be seen that these topics all fit together very closely and they will become the new reality in a very short timeframe. It is imperative that we all understand these changes and the opportunities and challenges they present, while this is also an important opportunity to provide feedback to the key officials with direct oversight and responsibility for planning and delivery of the initiatives. Even the experts will find something of value in this workshop, and there are relatively few of those in reality.
This workshop provided valuable information to its participants on the following points:
- Get insights into the future of the regulatory strategy for data acquisition and analysis
- Learn about the new EMA task forces established to prepare the regulatory network for modern methods
- Understand how all stakeholders can prepare and plan for the future and benefit from the changes
- Learn how clinical trials will be conducted in the next 20 years with the revision of the ICHE6
- Understand how patients will benefit from shorter and more innovative clinical trials, more robust HTA assessments, new digital solutions to measure efficacy and how more real world evidence data will help to reduce risk and make decisions more certain
Please send any questions concerning this workshop to Martina Bergna