The Missing Piece in Liver Disease Trials

Tim Jobson, MD PhD, Medical Director of Predictive Health Intelligence.

As liver cancer awareness month drawers to a close we reflect on the future treatment outlook for patients’ poor liver health. Liver disease can often be the unwanted third wheel of more discussed organs like the heart or brain. But it is one of the leading causes of morbidity and mortality worldwide, so I think it deserves attention.

Despite the extremely high prevalence of liver disease, 80% of trials fail to meet their enrolment targets. Which sets up any trials for difficulties. Add to that the hidden nature of many liver diseases; it's a uniquely challenging indication for finding the patients needed to develop treatments. 

Recent news about Akero failing to meet their primary endpoint for their NASH treatment follows Intercept's failure earlier this year. Highlighting that even when trials make it through the initial phases, developing therapies for liver disease seems uniquely positioned for failure. However, as many have noted, there is a big market here. 

A new hypothesis (based on experience in the clinical world) is that utilising existing data and a 'case-finding' approach could speed up recruitment, helping identify those with potential liver disease more efficiently and cost-effectively.

Why Does this Matter?

Liver disease is a leading cause of morbidity and mortality worldwide. Most cases of liver disease are preventable or treatable, which provides an opportunity for vast improvements in public health if the right resources are applied to research the condition. Amid overburdened healthcare systems, we need approaches to care that are proactive in preventing late-stage disease.

Despite significant investments in research and development, many promising drug candidates have faltered during clinical trials. Care has progressed over the last decade; however, many key types of liver disease are yet to have an effective treatment. This is partly due to drug trials in hepatology being impeded by costly recruitment, high screen failure rates, and difficulty in progressing past the initial phases.

With many trials globally failing to meet their recruitment timelines, the lifesaving research being conducted is delayed in its ability to help patients. In some cases, this research may never deliver real-world impact.

But what if the secret to participant recruitment lies in existing healthcare data? 

The problem is how to successfully implement a case-finding approach without overburdening the project management teams responsible for recruitment. This is where a simple yet effective solution leveraging existing healthcare data could be the answer.

People undergo blood tests for various reasons, and once conducted, those results often sit in a database, unused. These existing results could be vital in helping patients with liver disease. 

By unlocking the power of this data, those recruiting into clinical trials for almost any chronic liver condition, including viral, metabolic, inflammatory, and inherited diseases, could quickly identify which patients fit the eligibility requirements for a trial.

The case-finding approach isn't new; it has previously seen massive benefits within Hepatitis C. Tools like hepatoSIGHT provide an easy-to-use system to expedite the case-finding method and reduce the burden of recruitment on sites. This tool offers more than previous case-finding approaches, that focused on “lost-positive`’ cases, it allows the user to find a range of potential patients across many liver diseases - depending on specific parameters that they set. 

This new application of the case-finding approach could reduce both the time to market by speeding up recruitment and screen failure rates. Meaning treatments for liver disease are made available to those who really need it, thanks to existing data.