The Alliance for Regenerative Medicine Outlines Recommendations for Increasing the Number of European-Based ATMP Clinical Trials

The Alliance for Regenerative Medicine Outlines Recommendations for Increasing the Number of European-Based ATMP Clinical Trials

• Europe has become less competitive than other regions in attracting new ATMP clinical trials.
• Faster and more streamlined review processes for clinical trials by regulatory authorities are fundamental to increase clinical research and development in Europe.
• There is considerable country-to-country variability within Europe: the UK has the highest absolute number of new ATMP clinical trials, but relative to their size, Belgium, Denmark, and Switzerland outperform other European countries as well as the USA and Canada.

WASHINGTON, D.C. and BRUSSELS, BELGIUM – 31 October, 2019

The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader advanced therapies sector, today published a report outlining the latest trends in clinical trials with advanced therapy medicinal products (ATMPs) based in Europe, and its recommendations for how to improve its competitiveness compared to other global regions.

The report, developed following a comprehensive global analysis of all new clinical trials with cell and gene medicinal products initiated between January 2014 to June 2019 and via an online survey with ARM therapeutic developer member organizations, provides detailed insights on clinical trials, including the number, type of technology, phase of development or approval process, specifically within Europe and also compared to other regions in the world.

Key Findings:

• Though the number of ATMP therapeutic developers based in Europe is approximately half of that based in North America, the number of clinical trials that have been initiated in Europe during the 2014-2018 period is only about a third of that in North America.
• During the same time period, the number of new clinical trials during the 2014-2018 period increased by 32% globally, 36% in North America, 28% in Asia, and less than 2% in Europe.
• There are proportionally more new gene therapy clinical trials (utilizing gene delivery, gene editing, and gene modified cell therapy technologies) in North America (71%) than in Europe (55%).
• There is considerable country-to-country variability in the number of clinical trials, speed of assessment, and time for approval of clinical trials in the different countries in Europe.
• In Europe, the UK, Spain, and France attracted the highest absolute number of new ATMP clinical trials (112, 102, and 101, respectively), followed by Germany and Italy (83 and 66, respectively).
• Within Europe, some individual countries have been particularly successful at attracting new clinical trials – with Belgium, Denmark, and Switzerland outperforming the USA and Canada in the number of new ATMP clinical trials per capita.
• ARM members said the most important criteria for selecting a clinical trial site and a country is the expertise and the skills of the clinical centers and healthcare professionals, followed by the speed of approval, the quality of the review, and the expertise of the regulatory authorities.

The fragmentation of regulatory bodies and ethical committees and the lack of harmonization on various aspects (e.g. donor testing requirements, patient information consent forms, contracting agreements) across the different countries may explain the complexity in starting new ATMP clinical trials  in Europe, however, other reasons may also contribute, such as the lower levels of investment capital available in the region. The complexity of European GMO (genetically modified organisms) requirements and approval process may contribute to fewer gene therapy trials in Europe.

ARM’s key recommendations (in Europe):

• Notwithstanding the quality of its clinical research centers, Europe could increase its attractiveness as a region for the development of ATMPs by improving timelines for approval of clinical trials and streamlining approval of multinational trials.
• For gene therapies, consistent approaches and conclusions for the GMO review and a faster approval process should be implemented across the region to not create a barrier to gene therapy-based clinical trials in Europe.
• Regulators should view the implementation of the Clinical Trial Regulation (EU) No 536/2014 as an opportunity to streamline the review process and significantly improve approval timelines across Europe.
• National competent authorities should allocate sufficient resources and ensure an adequate level of expertise for the review of applications for ATMPs.

Conclusions:

Previous work has shown that the European Clinical Trial Directive 2001/20/EC presented some shortcomings, particularly for trials carried out in several Member States, and was responsible for a decline in the number of clinical trials in the European Union. In view of the sharp increase of the number of ATMPs in development during the last few years, the Alliance for Regenerative Medicine, in this report, has characterized the attractiveness of Europe, including national disparities, for the clinical development of ATMPs, and trends over time compared to other regions. 

The implementation of the above recommendations will contribute to maintaining a strong innovation base and important R&D investments in Europe but, most importantly, this will be critical to ensure that European patients with serious diseases and only few or no alternatives can access these new transformative therapies at an early stage through participation in clinical trials.

Janet Lambert, CEO of ARM, commented: “The European advanced therapy space is rich in expertise and innovation, with some individual countries outperforming the U.S. in terms of per capita participation in clinical trials. This research shows that improving the speed of approval and streamlining requirements for European multinational trials with ATMPs are critical to attracting more clinical trials in Europe. Our hope is that regulators and governments across Europe will see the need to reduce barriers while continuing to ensure patient safety and maintaining its level of excellence for clinical research.”

Anne-Virginie Eggimann, M.Sc., Senior Vice President, Regulatory Science at bluebird bio, Inc., commented, “Time is an important driver when choosing a clinical trial location. In the USA, Investigational New Drug (IND) applications and subsequent protocols are reviewed by the FDA within a maximum of 30 days. The longer approval times and their related uncertainty make the conduct of multinational clinical trials in Europe more challenging than in some other regions.”

Dr Jacqueline Barry, Chief Clinical Officer at UK Cell and Gene Therapy Catapult, commented, “Europe has been at the forefront of innovation with ATMPs as the sector has matured and it is encouraging to see that there are so many potentially life-changing studies taking place across the continent. However, the number of clinical trials with ATMPs is variable from one country to another and we see that countries with shorter approval times, such as the UK, Belgium, Denmark, or Switzerland, attract more clinical trials. It is important to learn from these countries and replicate their performance across Europe. We remain excited by the prospects for the sector in Europe but to maintain its attractiveness and scientific leadership, we support the recommendations of ARM outlined in this report.”

A copy of the full report can be found here [link].

Press inquiries

For more information about the report or media requests, please contact Lyndsey Scull from the Alliance for Regenerative Medicine at lscull@alliancerm.org or Consilium Strategic Communications at arm@consilium-comms.com.

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory, and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. Founded in 2009, ARM works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its 350+ member organizations worldwide. ARM represents the interests of therapeutic developers, academic research institutions, major medical centers, investors, and patient groups that comprise the broader regenerative medicine community and is the prominent international advocacy organization in this field.

ARM has 70+ members across 15 countries in Europe. ARM aims to work closely with European stakeholders, leveraging its membership to create a supportive commercial and regulatory environment to create better conditions for the development and commercialization of ATMPs in Europe; develop strong stakeholder support around proposed solutions to improve patient access to ATMPs; promote clear, predictable and efficient regulatory framework across Europe; and promote international convergence of key regulations and guidance. For more information, visit alliancerm.org.