Unpacking NICE’s review of the HST routing criteria: implications for manufacturers

The National Institute of Health and Care Excellence (NICE) hosted a webinar on September 23rd detailing its planned review and refinement of routing criteria for the highly specialized technology (HST) evaluation program.  

What are the current HST routing criteria? 

• Criterion 1 (prevalence): The disease is very rare (prevalence in England lower than 1 in 50,000 people or about 1,100 people)  
• Criterion 2 (eligibility): Normally, no more than 300 people in England are eligible for the technology in its licensed indication, and no more than 500 across all its indications 
• Criterion 3 (innovation): The very rare disease for which the technology is indicated significantly shortens life or severely impairs quality of life  
• Criterion 4 (unmet need): There are no other satisfactory treatment options, or the technology is likely to offer significant additional benefit over existing treatments 

Why is NICE undertaking this review?  

NICE emphasized that the vision behind the HST criteria will remain the same: encouraging innovation and securing fair and equitable access for patients suffering from rare diseases. While the essence and intent of the four existing criteria will remain, NICE acknowledged that further clarifications would be beneficial in supporting sponsors to assess whether their technologies will meet the requirements of the HST routing criteria. These changes are intended to support NICE in implementing a more predictable and transparent routing process thus maintaining its reputation as an objective and fair public body. Most importantly, these changes could ultimately result in faster patient access to new technologies.  

“(…) we also recognized that more could be done. And in particular, what we want to try and do is to ensure that the criteria that we use are as clear, transparent and consistent as possible, so that the colleagues in the life sciences industry understand those criteria and understand how they are applied and that we are confident that we’re doing that in a consistent way that fully reflects the underlying vision of HST” – Professor Jonathan Benger CBE, Chief medical offices and deputy CEO, NICE 

Is there ambiguity in the current criteria? 

In its current wording and format, manufacturers cannot always predict whether their technologies will enter the STA or HST program. This can create challenges from an evidence-generation and a commercial planning perspective. NICE can only maintain its reputation as a fair and transparent public body if it can ensure that its guidance and processes result in predictable, transparent and consistent decisions. Parexel conducted a review to highlight examples of ambiguity within the current criteria: 

• Criterion 1 (prevalence): NICE outlines that in 'exceptional circumstances, a technology may be routed to highly specialised technologies guidance even if the disease it treats has a prevalence above 1 in 50,000. This is a discretionary departure from normal policy, so it is not possible to fully define when this discretion may be used. A technology would need to clearly and strongly meet all the remaining routing criteria to possibly benefit". Here, the term "exceptional circumstances" is not defined, nor is it clear how a technology could 'strongly meet' the remaining criteria.   
• Criterion 2 (eligibility): NICE states, 'Normally, no more than 300 people in England are eligible… A technology is unlikely to be considered suitable for HST if more than 300 people are eligible'. Here, using the words 'normally' and 'unlikely' creates ambiguity as the explicit criteria of when exceeding 300 people would be appropriate is unclear and could result in inconsistency in how NICE applies the criterion. Additionally, greater guidance would be welcomed on navigating this criterion in parallel with criterion 1, which limits the size of the prevalent population to 1,100.  
• Criterion 3 (innovation): In this criterion, NICE explain that the disease in which the technology is indicated 'significantly shortens life or severely impacts the quality of life'. The lack of guidance around the words 'significantly' or 'severely' creates ambiguity in the process. 
• Criterion 4 (unmet need): The final criterion requires there to be 'no other satisfactory treatment options, or the technology is likely to offer significant additional benefit over existing treatment options… The term 'significant' is not defined because it requires judgement.' While NICE lists examples of satisfactory treatments (e.g., 'authorised medicinal products, medical devices, or other methods of treatment used in England'), the point at which these are considered 'satisfactory' is unclear. Similar to criterion 3, the lack of guidance around the use of 'significant' also adds to the ambiguity.  

What will be the cost implication to the NHS? 

It was emphasized that this is not a review of NICE's HTA appraisal methods (i.e., “the goalposts remain the same”). This means that once a technology is routed using the new criteria, the methods used to appraise the submission remain unchanged. NICE also intends this review to be cost-neutral; this means that there will be no explicit (i.e., direct increase to the £100K HST threshold) or implicit (i.e., through single technology appraisals [STAs] entering the HST process and taking on the higher threshold offered by the program) change to the opportunity cost for the NHS.    

“This is all about clarity. What it is not about (…) is changing the vision, it’s not about changing the underlying principles, and neither is it about moving the goalposts or changing the bar. So overall this review is designed to neither increase or decrease the number of technologies that are routed into the HST program.” – Professor Jonathan Benger CBE, Chief medical offices and deputy CEO, NICE 

Do the existing criteria restrict innovation and competition? 

During the webinar, a key issue that surfaced was the potential for the existing criteria to be restrictive to innovation and competition. Two main concerns were raised by the webinar attendees: 

1. The routing criteria favors manufacturers that are first to market. As per the wording of criterion 4, the second or third technology will likely be routed through the STA process because the first technology would be considered a 'satisfactory treatment' option available to people with the disease.  
   
   The underlying concern is that the manufacturers that are second or later to market may be deterred by the lower willingness to pay (WTP) threshold in the STA process (£20K-£30K), especially where the first manufacturer had the opportunity to engage with the higher HST threshold (£100K). However, this concern may not be entirely valid, as the second or third technology may still be able to command a higher price than the first-to-market technology, regardless of an STA or HST routing, if the new technology offers innovation through greater health benefits (QALYs) when compared with the first-to-market technology. Furthermore, NICE’s introduction of the severity modifier should further mitigate this concern by offering manufacturers routed through the STA process an opportunity to operate at the £50K WTP threshold if the relevant criteria are met. Nevertheless, a revision to the criteria that provides greater clarity on the magnitude of the innovation required over the first-to-market to be routed into the HST program would be helpful.  
    
2. A single technology may have multiple rare indications, but criterion 2 limits the total number of people using the technology across indications to 500 for the HST routing to apply. 

In this case, the objections from industry stakeholders are more relevant. We present two examples to highlight this issue further:  

• Example 1: If a technology received reimbursement for stroke, then, following additional research, it was found that the technology could also be used to treat a very rare condition; this technology would be routed through the STA process for the second indication because stroke is a non-rare condition with a high prevalence that exceeds 500 people.  
• Example 2: If a technology has been priced and achieved routine commissioning for a rare disease using the higher HST threshold, expanding to another rare disease indication could exceed a population size of 500, resulting in an STA routing for the 2nd indication. If the technology receives a positive recommendation in the second indication, without indication-based pricing, the new (potentially lower) price will be used in both the first and second indications.  

Both examples are connected to the broader policy issue of indication-based pricing; nonetheless, clearer routing guidance will ensure that the HST values of encouraging innovation are retained. The current routing framework may restrict R&D activities because manufacturers may not be sufficiently incentivised to expand into new rare disease indications, hindering patient access to new, potentially life-saving technologies. 

Timelines for review and implementation 

The current timelines outlined during the webinar aim for public consultations to be held in December 2024, with the expectation of publishing the revised HST criteria by March 31st, 2025, and operationalising the process starting in the new financial year, on April 1st, 2025. 

Our final thoughts  

Pipeline scanning: These changes will certainly impact manufacturers with rare disease technologies in their pipeline. Manufacturers who have technologies that will undergo the routing process between December 2024 (publication of draft criteria and public consultation) and March 2025 (final criteria) should engage with NICE to seek clarity on the consequences of the existing versus new criteria and whether it would be strategically advantageous to wait for the new criteria to be operationalised.  

Evidence generation planning: Alongside engagement with NICE, we recommend integrating the HST routing criteria into early value strategies to understand the pricing implication of the technology in England. Parexel can support manufacturers with early landscape assessments to understand the disease burden, unmet needs and current treatment options for a given rare disease. Additionally, with the use of early economic models, manufacturers will be able to quantify the total number of people that will use the drug across all indications and the incremental quality of life and survival benefits that technology offers over the existing comparator(s). With all these data points in hand, the early conversations with NICE are likely to be more informed and hopefully provide manufacturers with greater clarity on their likely routing method. 

“We will be consulting, and we need your help to ensure that the work that we do is a genuine step forward and makes it easier to understand, easier to prepare an application, easier to submit and achieve the result.” – Professor Jonathan Benger CBE, Chief medical offices and deputy CEO, NICE 

Engagement: More broadly, manufacturers and patient groups should engage in the consultation and, importantly, ensure that these changes are an improvement in clarity rather than a restriction that prevents technologies that would have otherwise entered the programme (though it should be noted that NICE have said this is not the intention of these upcoming revisions). Parexel will continue monitoring NICE’s activities on this topic and actively engage in the consultation process to share thoughts and insights gained from our clients and project work.  

Interested to discuss the implications of the HST routing criteria review? Please reach out to our authors at Parexel Access Consulting. We’re always available for a conversation.  

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