On occasion of International Clinical Trial Day (ICTD) we have asked Dr. Burç Aydin, Project Manager at ECRIN and EU-PEARL member, to share his views on the challenges and new opportunities platform trials entail for the future of clinical research.
- What is the purpose of the ECRIN International Clinical Trials Day?
ECRIN launched International Clinical Trials Day (ICTD) in 2005 to commemorate the start of James Lind’s first clinical trial on scurvy, 20 May 1747, which laid the foundation for modern clinical research. The event aims to acknowledge the achievements that result from clinical research and unite the different stakeholders in clinical research: research organisations, clinical research professionals, policy makers, patient groups and citizens from Europe and beyond. Each year a theme is selected that is relevant to all the stakeholder groups. This year the theme nominated by the ICTD scientific advisory board is platform trials, an innovative trial methodology with implications for all the stakeholders.
ICTD has been replicated by some of ECRIN’s Member/Observer countries, and by several national or international organisations, who have now introduced their own national/international clinical trials day celebrations.
- What are the main opportunities and challenges in this field?
Clinical trials are evolving and platform trials, that, to date, have been used principally in the field of oncology, are one way to accelerate the identification of promising therapies.
Platform trial is a generic term that encompasses several methodologies. Platform trials can have an adaptive design (with a pre-planned interim analysis allowing them to stop interventions with no or little evidence of benefit and to add new arms when new knowledge is available), can allow a multitude of treatment arms to be compared with a single control arm.
“Platforms trials have the potential to answer key research question in a timely manner and as a result shape public health and policy.”
The COVID-19 pandemic has demonstrated the need for platform trials to rapidly advance knowledge for clinical and public health responses and to provide evidence of the proposed treatment strategies. It has also highlighted some of the challenges associated with this trial methodology. Among the difficulties to overcome is the design of a new platform trial, the development of its master protocol, the national regulatory approvals that need to be updated as the intervention arms evolve, prioritizing the objective of simplicity or a more sophisticated trial, the development of an appropriate statistical and data analysis plan, the identification of appropriate sponsors, to name a few. How best to address these challenges are among the questions that ECRIN hopes will be broached at ICTD 2021 . Given their perpetual nature, existing platform trials that are operational and ready to be activated to develop new or improved diagnostic and treatment strategies could be a good investment to answer any future outbreak.
- How is EU-PEARL contributing to move forward?
EU-PEARL is playing an important role as an overarching European project and as a scientific forum. It brings together the diverse expertise from all corners of the clinical research world, which is needed to prepare the infrastructure to be utilized in the not-so-distant future for platform trials and Integrated Research Platforms (IRPs). Key elements of this infrastructure are anticipated to be tailored regulatory guidance, early and continuous patient contribution, governing models and structures, study protocols and documents, as well as data access and management. While many deliverables are still in development, the idea is already being tested within the internal clinical research groups as well as external European projects. We believe that EU-PEARL will strongly support the needs of clinical researchers and patients and facilitate communication with health authorities and policymakers for the advancement of collaborative complex clinical trials.
- What will this new scenario imply for patients and society as a whole?
Patients and patient representatives are deeply involved in the design of the platform trials and their contribution is welcome at all steps. Compared to “conventional” clinical trials the platform trials, by sharing the control arm and allowing the ineffective treatment arm to be stopped, could help increase the enrolment of participants since they will have an increased likelihood to be allocated to an effective treatment strategy.
Platform trials are the easiest solution to test multiple treatment options for a given disease condition, either for registration, for repurposing, or for comparative effectiveness/treatment combination or optimisation. Platforms trials have the potential to answer key research question in a timely manner and as a result shape public health and policy.
More information and registration to ICTD event: https://ecrin.org/events/ictd2021