HST routing criteria: encouraging innovation in the treatment of ultra-rare diseases

Ultra-rare diseases have a significant impact on the lives of patients and those that care for them. That they affect very small populations presents specific challenges in the evaluation of suitable treatment options.  

This is why our Highly Specialised Technology (HST) programme is crucial in helping to encourage research and innovation in areas where gathering robust evidence can be difficult. It recognises that a different approach is needed to evaluate technologies for ultra-rare diseases so that we can provide fair access to treatments for very small patient populations.  

While NICE puts no limits on the number of treatments that are routed via the HST programme, it's important that the programme’s focus remains on ultra-rare conditions. Our role is to help practitioners and commissioners get the best care to patients, fast, while ensuring value for the taxpayer. When we recommend a treatment, it inevitably displaces health gains for others. So, in deciding whether to recommend a treatment we must consider the care that would be displaced elsewhere in the NHS and ensure this is not disproportionate. It’s the HST programme that helps us balance this with the need to provide access to treatments for debilitating diseases that severely impact small patient populations and that require additional support to drive research and equitable treatment access.  

 Our Single Technology Appraisals (STA) process, which is the route that most medicines take, is designed to be flexible and adaptable for almost all technologies and conditions. Therefore, it continues to be the most suitable route for most potential treatments, including those for rare conditions and small populations. An example of this is our evaluation of sapropterin (Kuvan) for the treatment of hyperphenylalaninaemia in people with the rare disease phenylketonuria (PKU), which was recommended by NICE via the STA route.  

It's the HST routing criteria that describe the exceptional circumstances in which new technologies should be routed for assessment by the HST programme. While improvements were made to the existing criteria in 2021/22, we recognised that we could do more to provide greater clarity about the decisions we make.  This is why we have further refined the criteria to help ensure fairer and more predictable decisions.  

I would like to say a huge thank you to everyone, including patient groups and those from industry, who took the time to share their views during the consultation. I want to assure you that your feedback made a difference and has really helped us shape the final criteria.   

As a result of the consultation, we have now introduced a clearer set of four criteria to ensure fairer and more predictable decisions.  We’ve also included additional definitions to deliver greater consistency and transparency. These are not additional criteria; instead, they help add detail to make decision-making more objective and consistent. Something we hope will aid predictability, transparency and efficiency for all parties including patients, manufacturers and NICE.  

I’m often asked if the aim of the revised criteria is to change the number of medicines routed to the HST programme. The answer is no; it's to more clearly define the circumstances when it appropriate to do so. It’s also not about cost-saving. However, we do hope that by introducing greater clarity we can help speed-up and streamline the decision-making process.  

Full transparency about the use of the criteria will be provided by the NICE Prioritisation Board which will make the final routing decision and publish details on the NICE website.     

I really hope that the efforts we have made to ensure greater clarity in the routing criteria for the HST programme demonstrate our commitment under the Rare Diseases Action Plan to encourage innovation and support the rapid adoption of effective new treatments for NHS patients with rare diseases.