1Liver Unit, Internal Medicine Department, Vall d'Hebron University Hospital, Vall d'Hebron Institute for Research (VHIR), Universitat Autònoma de Barcelona, Centros de Investigación Biomédica en Red en Enfermedades Hepáticas y Digestivas (CIBERehd), Barcelona, Spain. Electronic address: firstname.lastname@example.org.
2Department of Hepatology and Gastroenterology, Charité - Universitätsmedizin Berlin, Berlin, Germany.
3Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle NIHR Biomedical Research Centre, Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle Upon Tyne, UK.
4Janssen Research and Development, Raritan, New Jersey, USA.
5Novo Nordisk A/S, Bagsværd, Denmark.
6Analytics Department, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
7Center for Medical Statistics, Informatics and Intelligent Systems, Medical University of Vienna, Vienna, Austria.
8Liver Unit, Internal Medicine Department, Vall d'Hebron University Hospital, Vall d'Hebron Institute for Research (VHIR), Universitat Autònoma de Barcelona, Centros de Investigación Biomédica en Red en Enfermedades Hepáticas y Digestivas (CIBERehd), Barcelona, Spain.
9Department of Hepatology, Pitié-Salpêtrière Hospital, University Paris 6, France.
Non-alcoholic fatty liver disease is a condition that affects 25% of the population. Non-alcoholic steatohepatitis (NASH) is a progressive form of the disease that can lead to severe complications such as cirrhosis and hepatocellular carcinoma. Despite its high prevalence, no drugs are currently approved for the treatment of NASH. The drug development pipeline in NASH is very active, yet most assets do not progress to phase III trials and those that do reach phase III often fail to achieve the endpoints necessary for approval by regulatory agencies. Amongst other reasons, the methodological and operational features of traditional clinical trials in NASH might impede optimal drug development. In this regard, platform trials might be an attractive complement or alternative to conventional clinical trials. Platform trials use a master protocol which enables evaluation of multiple investigational medicinal products concurrently or sequentially with a single, shared control arm. Through Bayesian interim analyses, these trials allow for early exit of drugs from the trial based on success or futility, while providing participants better chances of receiving active compounds through adaptive randomisation. Overall, platform trials represent an alternative for patients, pharmaceutical companies, and clinicians in the quest to accelerate the approval of pharmacologic treatments for NASH.