Why this question matters now
In April 2025, NICE revised its HST routing criteria with the aim of improving the predictability and transparency of routing decisions by introducing clearer definitional statements for the four qualifying criteria.
What we set out to explore
In this white paper, we looked at each of the new HST routing criteria, the number of appraisals being routed using the new criteria, and established the key factors contributing to these decisions. We then used these learnings to provide recommendations to manufacturers with orphan medicines around how they might optimise their chances of achieving HST routing, including key supportive evidence.
Key findings
At the time of our analysis, only one of the nine appraisals considered using the new routing criteria had successfully been routed to HST. While most companies successfully demonstrated that their medicine was innovative, most failed to provide sufficient evidence that the disease was ultra-rare and debilitating, and that there are no effective treatment options already available for patients.
What this means in practice
To increase likelihood of HST routing, companies developing therapies for conditions at the boundary of rare and ultra-rare disease will need to invest in compiling an accurate description of the disease burden and realistic population estimates well ahead of NICE engagement. We have identified several key activities to support preparation for the HST routing process, including conducting targeted literature reviews, engaging with patient and clinical experts, and sourcing UK registry data to validate disease prevalence.
From the authors
While uncertainty is generally a central theme in health technology assessments in ultra-rare diseases, we always look for ways to minimise that wherever possible. We’re looking forward to taking the learnings from this research and applying it when guiding our clients through their upcoming HTA appraisals in rare diseases so they can make early and informed decisions around their health technology.
Read the full white paper
The full paper explores these themes in more detail and is available to download here.