ARM Reports Europe Less Competitive in Attracting Advanced Therapy Clinical Trials

Rare Daily Staff

The Alliance for Regenerative Medicine (ARM) finds that Europe lags North America and Asia in terms of the initiation of clinical trials for advanced therapy medical products such as cell and gene-based therapies.

It’s recently published 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 through 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.

“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,” said Janet Lambert, CEO of ARM. This research shows that improving the speed of approval and streamlining requirements for European multinational trials with advanced therapy medicinal products (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.”

The report outlines the latest trends in clinical trials with ATMPs based in Europe, and ARM’s recommendations for how to improve its competitiveness to other global regions.

Among the reports key findings is the fact that though the number of ATMP therapeutic developers based in Europe is about half of those 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 that same time period, while the number of new clinical trials increased by 32 percent globally, it increased less than 2 percent in Europe compared to increasing 36 percent in North America, and 28 percent in Asia. ARM also found that of new gene therapy clinical trials, those utilizing gene delivery, gene editing, and gene modified cell therapy technologies, there were more proportionally in North America than 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 European country-to-country variability in the number of clinical trials, speed of assessment, and time for approval of clinical trials. In Europe, the United Kingdom, Spain, and France attracted the highest absolute number of new ATMP clinical trials, followed by Germany and Italy. Some countries, such as Belgium, Denmark, and Switzerland, outperform by the U.S. and Canada in attracting new ATMP clinical trials per capital.

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.

“Time is an important driver when choosing a clinical trial location. In the USA, Initial new drug applications and subsequent protocols are reviewed by the FDA within a maximum of 30 days,” said Anne-Virginie Eggimann, senior vice president of regulatory science at bluebird bio. “The longer approval times and their related uncertainty make the conduct of multinational clinical trials in Europe more challenging than in some other regions.”

The fragmentation of regulatory bodies and ethical committees and the lack of harmonization on various aspects such as donor testing requirements, patient information consent forms, and 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 genetically modified organisms (GMOs) requirements and approval process may also contribute to fewer gene therapy trials in Europe.

ARM provides some recommendations to increase Europe’s attractiveness for the initiation of ATMPs, which include 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.

ARM also recommends that regulators 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, allocating sufficient resources and ensure an adequate level of expertise for the review of applications for ATMPs.

ARM says that the implementation of its recommendations will contribute to maintaining a strong innovation base and important R&D investments in Europe and, most importantly, 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.

Photo: Janet Lambert, CEO of Alliance for Regenerative Medicine

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