EU-PEARL is set to transform the way clinical trials are conducted by developing tools and methodologies to create Integrated Research Platforms (IRPs). This novel approach has the potential to make drug development processes more efficient and faster. A pivotal element is the master protocol, which is still a relatively novel concept. Its implications in critical areas such statistical methodology and regulatory aspects continue to evolve. In this sense, EU-PEARL provides a trusted and inclusive environment to conduct this necessary debate amongst key stakeholders. With this objective in mind, the Consortium organised four online Stakeholder Workshop sessions last October which brought together around 600 experts. All in all, this initiative has proved a great opportunity to interact with a variety of external stakeholders early on in the IRP design. A second Stakeholder Workshop is planned for 2023, once the IRP framework is further advanced.
Four sessions to discuss four critical topics
Four interactive panel sessions were organised, each with a different focus. The workshop started off with an introductory session on October 8th, where the EU-PEARL project and its objectives in relation to the current challenges in designing platform trials were presented. After outlining the difficulties involved in four different disease areas –Major Depressive Disorder (MDD), Tuberculosis (TB), Non-Alcoholic Steatohepatitis (NASH) and Neurofibromatosis (NF)-, project representatives underlined how the IRP concept might address these challenges. Furthermore, participants also had the opportunity to listen from other existing platform studies experiences, such as I-SPY2 and INTUITT-NF2.
The following sessions covered statistical aspects of IRPs with a focus on multiplicity framework on October 22nd, followed by an interactive workshop on regulatory and ethics aspects on October 26th. The event closed on 29th October focussed on patient engagement with patient representatives explaining the topics that concern them most in relation to master protocols and IRPs communication and design.
IRPs, an ongoing learning process
Specific interest was drawn by the discussions between the experts from academia, industry, and Health Authorities (HA) on the importance of various statistical challenges for the development of the multiplicity framework, specifically focussing on critical issues regarding multiple treatments, subgroups, and adaptions. The platform trial paradigm was contrasted to more traditional drug development programs. There was general agreement on the idea that sharing a common control group when investigating the efficacy of multiple drugs does not necessarily imply a multiplicity adjustment per-se. Furthermore, it was highlighted that sharing controls could also be very attractive for patients as it increases the probability of being randomised to one of the new experimental treatments.
Experts from ethic committees and health authorities referred to the importance of explaining the trial design in a clear manner in the regulatory submission regardless of their complexity, to facilitate their review and execution. At the same time, they acknowledged that innovative frameworks entail a learning process for all the stakeholders and stressed their interest in open communication and shared standard mechanisms to discuss IRPs in the future.
Patient centricity: drawing from past experiences
Since EU-PEARL has a specific focus on patient-centricity, the last session, devoted to patient engagement, was co-created with the EU-PEARL Patient Advisory Group (PAG). EU-PEARL’s researchers and different patient representatives shared experiences on past clinical trials from patients’ perspective and the difficulties that some diseases, such as MDD, encounter.
The experiences shared on patient engagement from the STAMPEDE project, showed that timely communication to patients in IRPs is of essence and patient’s voices need to be heard early in the project when key decisions are being made. Critical concerns from patients were mentioned specifically on communication aspects, such as the language and the complexity of available information.
Member of the scientific coordination team and co-organiser of the Stakeholder Workshop, Juan Manuel Pericàs from Vall d’Hebron, was pleased with the outcome of the workshop as it was “moving the needle in three aspects that are crucial to our project endeavour: master protocol design and analysis, regulatory and ethical framework, and patient-centricity.” Likewise, Andrew Thomson, statistician at the European Medicines Agency, commented after attending the event that EU PEARL “is clearly focussed on ensuring the clinical trials of the future are going to be fit for purpose for all stakeholders to draw robust conclusions from them”.
After this successful first Stakeholder Workshop, EU-PEARLs aims to continue interacting with key external stakeholders to receive their feedback during the process of designing the platform trials. A follow-up workshop is planned to further promote the IRP concept and disseminate EU-PEARL main results at the end of the project in 2023.
Find out more about EU-PEARL’s 1st Stakeholder Workshop and access all presentations.