Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and Cons.

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, there are no drugs currently approved to treat NASH. The drug development pipeline in NASH is very active, yet most assets do not progress to Phase 3 trials and those that do reach Phase 3 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 by using a master protocol which allows for evaluating multiple investigational medicinal products concurrently or sequentially with a single, shared control arm. Through Bayesian interim analyses, these trials allow early exit of drugs from the trial based on success or futility, while providing participants better chances of receiving active compounds through adaptive randomization. Overall, platform trials represent an alternative for patients, pharmaceutical companies and clinicians in the quest for accelerating pharmacologic treatment for NASH.