Collaborative trial platforms position themselves as the best method to achieve a drug for non-alcoholic fatty liver disease

One in four adults suffers from this hepatic disease for which there is still no specific pharmacological treatment.


Non-alcoholic fatty liver disease (NASH) affects 25% of the adult population. Despite being so common, there is still no specific drug therapy to manage it. Currently, multiple molecules are being studied, but trials are proving slow and inefficient. A new article published in the Journal of Hepatology by the NASH working group of the European project EU- PEARL, co-led by Dr. Juan M. Pericàs, researcher of the Liver Diseases research group of the Vall d'Hebron Research Institute (VHIR) and member of the advanced chronic liver disease research group of CIBERehd, proposes a solution. The researchers argue that the current clinical trial design is not the optimal format for this disease and propose platform trials as an alternative.

Traditionally, fatty liver disease was related to excessive alcohol consumption. In recent years it has been shown that the liver can accumulate fat due to lifestyle factors and comorbidities such as diabetes or obesity. Patients with NASH have a diverse affectation ranging from an asymptomatic condition, to inflammatory episodes with varying frequency, or, in the most severe cases, cirrhosis and liver cancer. Its prevalence, the absence of symptoms in early stages of the disease, and the fact that it can have serious and even lethal consequences for patients, make it a priority pathology for health researchers.

A 2021 study identified more than 100 clinical trials with potential drugs to treat NASH, but so far none have been successful. The authors of the article postulate that one of the major obstacles for researchers is the difficulty in obtaining a critical mass of trial participants.  The recruitment capacity is limited by the fact that this is still a very underdiagnosed disease and that in order to participate it is necessary to undergo several liver biopsies. In order to optimize the available resources, they propose the use of collaborative platform trials. Collaborative platform trials subject patients to a unique selection process that makes them candidates for several treatment arms at the same time. The idea is to unify data analysis and standardize the different bureaucratic processes and decision making to optimize efforts and accelerate the search for new drugs.

One of the strengths of the platforms is that for the various drug groups tested there is only one control group. This increases the number of participants receiving an active treatment. Also, since different drugs participate simultaneously, if one drug is shown to be effective, it becomes the new control arm, and if a drug is not effective, patients who were receiving that treatment can be moved relatively easily to another treatment group. Another benefit is that by centralizing data analysis, it is easier to achieve standardized measurements across all units, increasing the reproducibility of experiments. In addition, the regulatory framework of the platforms, based on the Bayesian statistical method, encourages decisions to be made in strictly scientific terms.

The researchers recognize that the new process may have implementation problems among hospital centers due to its novelty, the initial bureaucracy required, and the questions it raises a priori regarding concepts such as data protection and intellectual property. Even so, they believe that with a clear and specific regulatory framework, these doubts can be overcome. This is the ultimate goal of EU-PEARL. EU-PEARL stands for 'European Union-*Patient-*CEntric clinicAl trial pLatforms'. It is a unique public-private strategic alliance focused on creating a framework to foster the deployment of the collaborative trial platform as the new paradigm for drug development. EU-PEARL is led by Janssen Pharmaceutica NV and coordinated by VHIR, specifically by Dr. Joan Genescà, Clinical Director of the Digestive Diseases Area at Vall de Hebron Hospital and head of the CIBERehd's breakthrough chronic liver disease research group, and funded by the Innovative Medicines Initiative (IMI2 JU).

A 2021 study identified more than 100 clinical trials with potential drugs to treat NASH, but so far none have been successful.

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