FDA backs first AI tool for use in trials, targeting MASH

 The FDA has qualified the first AI-based tool that can be used in clinical trials involving patients with metabolic dysfunction-associated steatohepatitis (MASH) to help measure disease activity.

Called AIM-NASH, it is a cloud-based tool designed to help pathologists score liver biopsy components on measures such as fat infiltration (steatosis), inflammation (hepatocellular ballooning and lobular inflammation), and scarring (fibrosis), which are all features of MASH.

FDA qualification means it can now be used in any drug development programme for a MASH treatment and support regulatory filings of new therapies. As it stands, there are two drugs approved for MASH – Madrigal Pharma's THR β-selective agonist Rezdiffra (resmetirom) and Novo Nordisk's GLP-1 agonist Wegovy (semaglutide) – and dozens more coming through the pipeline.

It has been billed as pharma's next big growth area, with some analysts predicting a market potentially worth tens of billions of dollars a year.

Metabolic dysfunction-associated steatotic liver disease (MASLD), including its more severe form MASH, is the most common chronic liver disease in the world, affecting roughly 38% of the world's adult population and claiming hundreds of thousands of lives every year. The condition is largely associated with obesity and an unhealthy diet and lifestyle, and is on the rise in industrialised nations.

In MASH, the disease has progressed to include advanced, life-threatening complications like cirrhosis, worsening of liver function that can require a transplant, and liver cancer.

AIM-NASH, developed by Boston-based AI specialist PathAI, is designed to reduce the burden of scoring biopsy samples, which is currently carried out by multiple independent experts and is time-consuming and can also be prone to variable results.

It uses AI algorithms to analyse images of liver biopsies and provides scores according to the standard NASH Clinical Research Network scoring system, according to the FDA. The EU drugs regulator, the EMA, endorsed use of the tool in MASH trials earlier this year.

The agency stressed that the process "keeps humans involved, as pathologists are fully responsible for final interpretation, reviewing the whole slide image and AIM-NASH outputs before accepting or rejecting the AI-generated scores."

There has been a major push to adopt digital and AI technologies by the FDA under the Trump administration, with other recent moves including the launch of a real-world review process to make it easier to bring digital health technologies (DHTs) to market, an agentic AI platform that can be used by staff to automate complex tasks, and a generative AI (GenAI) system called Elsa designed to speed up review processes.

https://pharmaphorum.com/news/fda-backs-first-ai-tool-use-trials-targeting-mash