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.
