In the UK, the National Institute for Health and Care Excellence (NICE) have different ways of assessing and deciding whether a new medicine will be made available by NHS England. One important route for assessment is called ‘Highly Specialised Technologies (HST). This is designed to make sure treatments for rare diseases are assessed fairly due to the fact that fewer people will need them than for more common conditions.

This means that some of the evidence is also less strong than for more common conditions, because medicines have been tested on smaller populations. NICE consult organisations like us to ask what we think, which is important for you, our community to understand. Have a read of our recent consultation response – it helps everyone to be better informed about why decisions are made and how.

If you have any questions, please get in touch via contact@metabolicsupport.org 

We are happy to announce the results of our latest research, “The Impact of Different Cysteamine Formulations on the Lives of People Living with Cystinosis.”

This research announces important findings from a survey conducted in collaboration with Cystinosis Foundation UK and Kidney Research UK, aiming to better understand the day-to-day experiences of those living with cystinosis, as well as their parents and caregivers.

Metabolic Support UK has responded to the National Institute for Health and Care Excellence (NICE)’s consultation on the proposed changes to its Highly Specialised Technologies (HST) routing criteria. The HST routing criteria are criteria that decide whether a new medicine will be assessed through the HST route, or not. When assessed through the HST route, different requirements are in place, for example, NICE is willing to consider more uncertainty that arises from clinical trials conducted in small populations, as well as a higher medicine price.  

The HST routing criteria are important for people with a rare disease in general, and inherited metabolic disorders (IMDs) specifically, because most new medicines for rare diseases and IMDs are assessed through the HST framework. When the criteria change, this could have an impact on whether these medicines will still be eligible for HST routing, and thus the different requirements (e.g. consideration of more uncertainty and higher medicine prices). 

The consultation by NICE closed on 30 January 2025. For further details about the consultation, please visit the consultation page on the NICE website. 

Summary of MSUK’s response and key recommendations:

• MSUK welcomes the opportunity to feedback on the newly proposed HST criteria which will have a direct impact on our IMD community. The current suggested amendments suggest a lack of understanding of the rare disease landscape and fail to acknowledge the numerous hurdles the rare disease community already face, thereby limiting the opportunities for treatment innovation for people living with a rare disease. 

• Specifically, greater clarity is needed around definitions included in the criteria, e.g. “exceptional negative impact” and “considered inadequate”, which by definition are subjective, to ensure these are consistently applied. 

• Repurposed medicines are a vital innovation option for people with IMDs, as well as the rare disease community in general. The blanket exclusion of repurposed medicines under the HST framework needs to be further expanded to ensure alternative incentive programmes are in place through either NICE or NHS England. 

• The requirement to be “the first”, stifles innovation, and suggests that if a treatment is available, that is good enough for people with a rare disease. This is inequitable in comparison to incentives created for non-rare disease, such as cancers, where incremental innovations are rewarded. 

To read MSUK’s full response, click the button below: