EU-PEARL stands for ‘European Union-Patient-cEntric clinicAl tRial pLatforms’ project. It is a unique public-private strategic partnership focused on creating a framework to foster the deployment of collaborative platform trials as the new paradigm for drug development. EU-PEARL is led by Janssen Pharmaceutica NV and coordinated by VHIR, and funded by the Innovative Medicines Initiative (IMI2 JU).

EU-PEARL hopes to transform standard clinical trials into collaborative multi-compound research platforms focused on patients

The standard current trial model evaluating one drug at a time implies often a lengthy and sequential development cycle, requiring substantial investment. For patients, recruitment and timely access to more effective treatments may be also challenging.

“It is not that the current model does not work. It offers a safe and reliable way to test drugs at the development phase. But there are more efficient models that could be used, with the proper collaborations and tools in place” states Dr Sarai Rodríguez Navarro, VHIR’s Scientific Project Manager of EU-PEARL. “Adaptive platform trials allow multiple treatments to be evaluated simultaneously – in a safe and reliable way too –, but potentially reducing the time for patients to access new treatments”. The EU-PEARL framework shall contribute to make clinical trials more efficient, potentially shortening the timeline to develop and bring to market new medicines for patients.

EU-PEARL focus its efforts on four disease areas: major depressive disorder, tuberculosis, non-alcoholic steatohepatitis (NASH), and neurofibromatosis

Since November 2019, the EU-PEARL Consortium works to develop general methodologies and tools to enable collaborative platform trials in any disease area; but also focussing on the platform design in four conditions with high unmet needs: major depressive disorder (MDD), tuberculosis, non-alcoholic steatohepatitis (NASH) and neurofibromatosis.

Within Vall d’Hebron Research Institute several groups contribute to EU-PEARL activities. The Mental Health and Liver Diseases groups co-lead the working teams in their respective disease area. For Dr Antoni Ramos-Quiroga, Head of the Psychiatry, Mental Health and Addictions Department, “EU-PEARL initiative constitutes the first and only Integrated Research Platform in MDD focused on Phase II/Proof of concept clinical trials on patients who do not respond adequately to first line treatment and continue to have impairment, which will allow for faster and more agile development of new treatments for MDD”

In the field of NASH, “with a high prevalence and many compounds in the pipeline for drug approval, a platform trial in NASH setting will be an advance and efficient way to accelerate testing and approval for pharmacological treatments; thus a way to fulfil a gap in a global public health issue” highlighted Dr Juan M. Pericàs, co-leader of the EU-PEARL NASH working group and member of the Liver Unit.

As a strategic partnership between public and private sectors, EU-PEARL aims to promote collaboration amongst pharmaceutical companies, clinicians, regulators, patients and researchers, to shape the future of clinical trials

With a multi-stakeholder approach, EU-PEARL aims to enrich the scientific discussion on key questions concerning platform trials methodology and disease-specific platform requirements. With this mindset, EU-PEARL shares main tools and results publicly through scientific papers and project deliverables. To name a few, EU-PEARL has built a common terminology around platform trials to facilitate understanding and stakeholder collaboration, an assessment of Clinical Operations best practices, reviewed software and simulation tools for trial design, and reports on the main challenges to design collaborative platform trials for each the disease-area.

While facing its last year of execution, the project aims to increase multi-stakeholder interaction through more events and workshops and will work to define the sustainability strategy for project outcomes and disease-specific platforms. Once the project is completed and funding is secured, a reusable infrastructure for screening, enrolling, treating and assessing participants in adaptive platform trials in each of the four diseases could be a reality. “A permanent stable structure hosting any platform trial initiative in Europe, that might be led by VHIR, is the envisioned outcome of this project” states Dr Joan Genescà, coordinator of the project and Clinical Director of Digestive Diseases.

*This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement No 853966. The JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA and CHILDREN'S TUMOR FOUNDATION, GLOBAL ALLIANCE FOR TB DRUG DEVELOPMENT NON PROFIT ORGANISATION, SPRINGWORKS THERAPEUTICS INC.