Vall d’Hebron hosted IMI- EU-PEARL work package meeting to develop a platform for clinical trials for NASH

Scientifics of the Vall d’Hebron University Hospital Hepatology Department and the research group of Liver diseases of Vall d'Hebron Research Institute (VHIR) and industry experts, all partners in the IMI-2 EU-PEARLConsortium (grant agreement nº 853966) met in Barcelona on May 11th and 12th. The main aim of the meeting was to advance the development of platform trials for Non-Alcoholic Steatohepatitis (NASH) disease. This meeting took place in the context of the European Union Patients Centric Clinical Trial Platforms (EU-PEARL). EU-PEARL is a strategic partnership funded by the Innovation Medicines Initiative encompassing relevant actors from the public and private sectors at the global level, to develop a generic framework and additional instruments to conduct collaborative platform trials where different treatments for a single disease can be tested simultaneously. 

Tools developed under the concept of Integrated Research Platforms, include templates and guidance documents related to platform trials and other resources, such as an extensive network of sites to ensure adequate numbers of participants and tools to better support patients and research communities. The generic framework for collaborative platform trials being developed by EU-PEARL will contribute to making clinical trials more efficient, potentially shortening the timeline to develop and bring to market new medicines for patients. 

In the meeting, held in a hybrid format, around 30 participants from academic and industry discussed on the framework to conduct collaborative platform trials for NASH. Despite being a prevalent condition, there is no approved pharmacologic treatment for NASH at the moment. This meeting between different EU-PEARL stakeholders will contribute to the development of a platform trial for this disease. “It is time to think out of the box. Trials for NASH have been running for many years and there are still no drugs approved for treating NASH. Platform trials provide a unique opportunity to advance in the field through a multistakeholder collaboration that puts the patient needs at the center” said Juan M. Pericàs, co-leader for WP6 at VHIR member of the Hepatology Department at Vall d’Hebron. “This is important because of the confluence of two important topics, a new way of performing clinical trials and an extremely prevalent disease as NASH” mentioned Joan Genescà, scientific coordinator of EU-PEARL, clinical director of Digestive Diseases, and principal investigator of the CIBERehd group on Advanced chronic liver disease.

Among the topics discussed these two days were patient engagement, operational requirements to implement platform trials for NASH, interactions with regulatory agencies for the approval of the master protocol, and sustainability of the project.