Vall d’Hebron Institute of Research (VHIR) and the Liver Unit of Vall d’Hebron University Hospital (VHUH) hosted a scientific meeting in Barcelona last May 11th & 12th to discuss the next steps in the development of platform trials for Non-Alcoholic Steatohepatitis (NASH) disease.
The meeting brought together academic and industry experts closely collaborating in EU-PEARL to create a generic framework and additional instruments for conducting collaborative platform trials. This novel approach in clinical research implies that different treatments for a single disease can be tested simultaneously, thus making clinical trials more efficient and potentially shortening timelines to bring new medicines to market. This innovative framework can become a game-changer in diseases with high unmet needs, such as NASH.
This innovative framework can become a game-changer in diseases with high unmet needs, such as NASH.
Non-alcoholic fatty liver disease (NAFLD) is characterized by fat buildup in the liver. Its more advanced form, NASH, is characterized by inflammation and damage to liver cells, leading to tissue scarring or fibrosis. NASH is a silent disease where there are no initial symptoms and thus, is often disregarded. Patients affected by this disease often develop other pathologies such as cardiovascular disease or liver cancer. This condition affects 25% of the population, and it is related to lifestyle and dietary habits. Indeed, obesity and diabetes are often comorbidities in NASH. Unfortunately, despite of the number of clinical trials going on, there is currently no approved pharmacological treatment for NASH.
“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” Juan M. Pericàs (VHIR)
In the meeting, held in a hybrid face-to-face and virtual format, around 30 participants from academia and industry discussed the framework to conduct collaborative platform trials for NASH. This meeting between different EU-PEARL stakeholders will contribute to developing 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 multi-stakeholder collaboration that puts the patient needs at the center” said Juan M. Pericàs, co-leader of EU-PEARL’s NASH team at VHIR and member of the Liver Unit at Vall d’Hebron University (VHUH) Hospital. “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 at Vall d’Hebron University Hospital , and principal investigator of the CIBERehd group on Advanced chronic liver disease.
“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” Joan Genescà (Vall d’Hebron & CIBEREHD)
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.
For additional information, please check these related articles and interview:
“Creating a platform trial for the specifics of NASH trials requires considerable planning but offers potential advantages” by Vlad Ratziu (Assistance Publique Hôpitaux de Paris) and Nick di Prospero (Janssen)
“EU-PEARL can serve as a reference in the diagnosis, prevention and treatment of NASH” interview with Juan M. Pericás and Jesús M. Rivera (VHIR)