Skip to main content
European Commission logo
English English
CORDIS - EU research results
CORDIS
CORDIS Web 30th anniversary CORDIS Web 30th anniversary

EU Patient- cEntric clinicAl tRial pLatform

Periodic Reporting for period 3 - EU-PEARL (EU Patient- cEntric clinicAl tRial pLatform)

Reporting period: 2022-05-01 to 2023-04-30

EU-PEARL aimed to transform the single-treatment clinical trial approach to a platform trial approach, which can include multiple treatments sourced from different companies and organizations. To achieve this highly ambitious goal, the public and private sectors formed a strategic alliance to develop an Integrated Research Platform (IRP) paradigm to conduct these platform trials. This IRP paradigm is centred on a master trial protocol which can accommodate multi-sourced treatments, while using existing hospital infrastructure and federated patient data in the trial design, planning and execution. Moreover, the IRP paradigm aims to assure an optimized regulatory pathway for these novel treatments. Altogether, EU-PEARL developed frameworks and tools that will pave the way for more efficient development of new treatments, while also improving patient centricity in clinical trials.

Background: why EU-PEARL?
For a variety of diseases, there is a clinical need for faster development of new treatments. Currently, the standard clinical trial model evaluates one drug at a time, which often results in sequential cycles of drug development and might thus not be the most efficient way of getting treatments to patients. Moreover, from a patient-centric perspective, the current trial environment often creates challenges for recruitment and patients struggle to navigate the complex trial landscape to find the optimal study.

Platform trial designs offer opportunities for more efficient drug development that could subsequently speed up the delivery of new treatments to patients. Even though such multi-treatment platform trials are already being conducted, the absence of a general framework for this new clinical trial model created hesitancy to initiate this collaborative approaches more routinely. The reality of this became again apparent during the Covid-19 response, as hundreds of individual trials were set but many struggled to achieve meaningful outcomes quickly.
To achieve this goal EU-PEARL had 4 objectives: 1) Create an open, dynamic and patient inclusive IRP governance structure to manage regulatory, ethical, legal, statistical and data requirements; 2) Disseminate and exploit the EU-PEARL IRP paradigm by providing the necessary common tools, procedures, expertise and operational skills meeting the highest scientific, regulatory and ethical standards and best practices. These were developed jointly by public and industry partners in a consensus-based approach; 3) Create site and data networks ready to execute EU-PEARL’s platform master protocols, and so EU-PEARL helped in the development of 4 ready-to-go IRPs based on 4 disease areas: Major Depressive Disorder (MDD), Tuberculosis (TB), Non-Alcoholic Steatohepatitis (NASH) and Neurofibromatosis (NF). Each of these areas has its own specific challenges, which were used as examples for other teams to build on when they are interested to initiate an IRP; and 4) Conceptualize a way to sustain project outputs in order to maintain an IRP framework that can be used for any disease area with unmet need.
In conclusion, EU-PEARL developed a framework to allow for novel treatments to be tested and developed in an IRP setting that bridges clinical care and clinical research, while involving multiple companies and organizations. EU-PEARL has provided tools and a framework for executing platform trials in different diseases, and specifically by providing tools to do platform trials in the specific diseases comprised in the project.
During the 42 months of project implementation (01/11/2019 – 30/04/2023), EU-PEARL developed both disease-agnostic and disease-specific IRP materials and tools, while generating awareness on the project and the advantages of adaptive platform trials. All public materials and publications are available on EU-PEARL website: https://eu-pearl.eu/

From a disease-agnostic perspective, EU-PEARL developed 1) A glossary with key terminology and scenarios for platform trials; 2) A rplatform trial best practices tool; 3) A suite of templates including master protocol template, Intervention Specific Appendix and guidance materials. Also, several publications have been issued, mainly covering methodological and regulatory challenges, and linking EU-PEARL to Covid-19 platform trials set to tackle the pandemic situation.

In parallel, teams focussing on disease-specific IRPs (MDD, TB, NASH, NF) designed protocols for the platform trials (and Longitudinal Natural History Study (LNHS) in MDD and NF) to be implemented in each disease-specific IRP, based on an initial assessment of clinical challenges. Feasibility assessment and cohort identification exercises leveraging Electronic Health Record (EHR) data and OMOP/standard data model tools were also performed in the four diseases.

Patient engagement structures - i.e. patient advisory group (PAG), expert advisory group (EAG) - and events - patient and community engagement days - were set in place to enable close collaboration with patient representatives and to ensure that the patient’s voice was heard throughout the design of the IRP frameworks.

EU-PEARL devoted a lot of efforts to make the project become widely understood: besides a communication and dissemination strategy, EU-PEARL also developed an alliance strategy which maps how EU-PEARL will promote knowledge and understanding on platform trials and introduce the concept of IRPs, launch dialogue and collaborations with relevant external experts and related on-going initiatives, and foster the sustainability and endorsement for future adoption of this innovative framework. With this aim, EU-PEARL organized several workshops to encourage knowledge sharing and open discussion amongst all stakeholders. All the information and materials from past events can be found at https://eu-pearl.eu/workshops/
EU-PEARL collaboration and knowledge sharing will continue, finding its new roots in the EU-PEARL Community of Practice. Anyone interested in the platform trial field can sign up to it here and help being a driving force for innovative clinical research.
With the completion of the EU-PEARL project, frameworks for the 4 disease-specific IRPs (MDD, TB, NASH, NF) including the 4 master protocols, are available on the website of EU-PEARL. Additionally, more generalizable frameworks on how to set up and conduct an IRP, together with a plan on how to sustain and maintain these frameworks have also been created. By using these frameworks, future platform trials will be set up to generate high quality evidence that is based on strong and consistent data networks and statistical methods. These frameworks will also emphasize and provide guidance on how to embed the patient voice as a partner in the development of IRPs.

Altogether, the EU-PEARL IRP framework lays the foundation for a clinical trial environment that enables more collaborative platform trials and creates opportunity for faster response to patient needs. The project has produced 26 scientific papers addressing various components of Integrated Research Platforms.
The project is wrapped up in: Current state of the art and gaps in platform trails for drug development: ten things you should know inside from EU-PEARL (https://doi.org/10.1016/j.eclinm.2023.102384)
EU-PEARL Logo horizontal