This past November marked the first 12 months of EU-PEARL. A fruitful time in which the project Consortium has largely focused on laying the foundations to start developing the innovative IRP framework. Once in place, this new paradigm which deepens into the adaptive platform trials approach, will bring more efficacy and speed to the drug development process. An advancement that COVID-19 has revealed essential to address global health challenges.
The initial steps in this inspiring journey to create state of the art clinical trials have centred in raising awareness and build knowledge about the IRP concept and its potential impact on a wide range of stakeholders: patient representatives, researchers, health care providers or the pharmaceutical industry. To set the scene for this new framework and its shared tools, tasks touching on governance, common terminology and best practices have taken center stage: generic guidelines on data management and governance have been issued, together with a glossary expounding the main IRP and platform trial terms and a report on best practices in clinical operations.
Multi-stakeholder collaboration and dialogue to build the clinical trials of the future
At the same time, methodology aspects -statistics- have already been considered early on in the project. In fact, EU-PEARL is actively contributing to this debate through scientific publications or by actively engaging in dialogue with scientists and regulators. Indeed, EU-PEARL has fostered dialogue, knowledge sharing and collaboration with relevant actors in the clinical trials field and related on-going initiatives. A stepping stone in this direction has been the stakeholder workshop held last October which gathered around 600 participants. The sessions focused on regulatory and ethical issues, methodological challenges, and patient engagement priorities for the development of IRPs.
Along the same lines, disease specific teams -focused on Major Depressive Disorder, Tuberculosis, Non-Alcoholic Steatohepatitis (NASH) and Neurofibromatosis–, have already assessed the current scenarios in clinical trials. During this year, they have interacted with experts in their fields to present and validate their preliminary results on IRP challenges definition and how to overcome them during the next steps of the project. Furthermore, the assessment has led to preliminary drafts on the trial designs and, in the case of Neurofibromatosis also, to a specific document defining which manifestations of NF should be considered for IRPs.
Through the year, several publications have been developed, mainly covering methodological challenges – statistics – and linking EU-PEARL’s goal of creating a framework of IRPs with several attempts to conduct clinical research studies to fight COVID-19 using the adaptive trials approach. Additionally, a Clinical Network Working Group has been created to guide the set-up of clinical networks for each IRP whilst several documents have been issued –with inputs from disease-specific teams– establishing the general data requirements to build the federated data network.
Patient centricity is already taking shape
As an initiative that places the patient at the center of the clinical trial design, this last year has also seen progress in this area with the setup of the structure to promote Patient Engagement. The two bodies integrated by patient representatives (Patient Advisory Board) and WPs (Expert Advisory Board), constitute a major opportunity to explore patient engagement opportunities in the design of clinical trials. At the same time, an interactive workshop to foster interaction between researchers, clinicians and patients was organised last September. During this session, participants could actively learn from each other.