Preparing for the next breakthroughs in liver disease treatments
With no licensed treatments for MASH, a serious liver condition affecting 3 million in the UK, NICE has co-developed a framework with patients, clinicians, and industry to accelerate access to promising new medicines.
More than 3 million people across the UK live with metabolic dysfunction-associated steatohepatitis (MASH) - a serious form of non-alcoholic fatty liver disease. The condition causes inflammation and damage to the liver, even in people who rarely, or never, drink alcohol.
Currently, there are no licensed treatments for MASH. But that could soon change, with promising new medicines in development that could transform how we treat this complex condition.
The challenge for NICE is how we make sure that the treatments reach patients quickly, when they go through our typical evaluation processes.
A common but overlooked condition
MASH affects around 5% of the UK population yet remains significantly underdiagnosed. Left untreated, it increases the risk of serious complications including cardiovascular disease and liver failure.
The pipeline of potential treatments is encouraging. Several medicines are in development. Others including GLP-1 agonists like semaglutide (currently used for obesity treatment) are being repurposed for MASH. These treatments could present a step-change in patient care if introduced to the NHS.
However, there's a problem. MASH is complex, often progresses slowly, and lacks long-term treatment data. As a result, manufacturers may use different approaches when demonstrating their treatments' effectiveness and value. This inconsistency could create uncertainty for NICE's independent committees, potentially delaying patient access to beneficial treatments.
Getting ahead of the challenge
NICE took a proactive approach to addressing this challenge. We set out to identify the evaluation challenges this new therapeutic area would present and to develop solutions before they became barriers to patient access.
Our approach involved extensive collaboration internally and with external stakeholders, we:
conducted a comprehensive review of current NHS clinical practice and compared it with ongoing research
systematically examined existing economic models for MASH treatments
held 3 workshops. Inviting clinicians, patient organisations, system partners, health economists and industry representatives to identify key issues and co-create solutions.
Guidance for generating and evaluating evidence
The result is a new economic model concept that offers a common framework for evaluating MASH treatments. By using the same model concept to develop and assess evidence submissions, manufacturers and evaluation committees can work together to make consistent and efficient decisions.
This means that when promising MASH treatments reach NICE for evaluation, both manufacturers and our committees will be working from the same playbook. This reduces uncertainty and speeds up the process of getting effective treatments to patients who need them. The full report, including the economic model concept, shows the collaborative work of NICE's HTA Lab (Health Technology Assessment Innovation Laboratory).
HTA Lab – creative solutions for complex HTA problems
The HTA Lab enables NICE to develop creative solutions to complex problems in health technology assessment. It offers a ‘safe space’ for creating solutions in collaboration with system partners and stakeholders.
If you're working in an area where you think NICE's HTA Lab could help address evaluation challenges, we'd welcome your suggestions.
Find out more about the HTA Lab including upcoming and past projects and how to get in touch the team.
Pharmaceutical and healthtech companies seeking to enter the NHS market can contact our NICE Advice service for support and expertise.