NICE process and methods
Appendix 1: highly specialised technologies
The vision
The Highly Specialised Technologies (HST) Programme evaluates technologies for very rare, and often very severe, diseases that need the specific considerations and flexibilities permitted by the programme. Specifically, it evaluates technologies that:
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meet the definition of a highly specialised technology, as described in legislation in Schedule 4 of the NHS Commissioning Board and Clinical Commissioning Groups (Responsibilities and Standing Rules) Regulations 2012, or may potentially need nationally coordinated delivery approaches, and
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need consideration using the methods and processes of the HST Programme, as identified through the highly specialised technologies routing criteria.
NICE's standard technology appraisals methods and processes are designed to be flexible and adaptable for all technologies and conditions. So, they are suitable for most technologies that treat rare conditions and small populations.
The HST Programme is designed to be used in exceptional circumstances. Its purpose is to evaluate technologies for very rare diseases that have:
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small numbers of patients
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limited or no treatment options
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challenges for research and difficulties with collecting evidence, because of the uniqueness of the disease.
The HST Programme aims to:
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encourage research on, and innovation for, very rare conditions when there are challenges in generating an evidence base that is robust enough to bring the product to market
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secure fairer and more equitable treatment access for very small populations with very rare diseases
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recognise that an approach that maximises health gain for the NHS may not always be acceptable: it could deliver results that are not equitable.
The HST Programme acknowledges that:
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It is important for NICE to apply appropriate limits on the very rare populations that can potentially be routed to the programme. This is because the HST Programme is a deliberate departure from the standard technology appraisal process (valuing the benefits from these technologies more highly by having a much higher incremental cost-effectiveness ratio [ICER] threshold) for the reasons outlined above.
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Each time NICE routes a topic to the HST Programme it is deciding that, if the technology is recommended, the NHS must commit to allocate resources that would have otherwise been used on activities that would be expected to generate greater health benefits.
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NICE has sought to strike a balance between the desirability of supporting access to treatments for very rare diseases against the inevitable reduction in overall health gain across the NHS that this will cause. Both considerations are valid and important, and neither can be given absolute priority over the other. Therefore, the HST Programme criteria and their anticipated application intentionally do not seek to capture every case when there are challenges in generating an evidence base or when there is a small population with a rare disease.
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This approach ensures that technologies routed to the HST Programme fulfil the vision of the programme and manages the displacement in the wider NHS.
However, it can be difficult to identify the exceptional circumstances when the highly specialised technologies methods and processes should be used because of the difficulty in getting the information needed. Proxy information is often relied on and used to make subjective judgements. The routing criteria identify which technologies should be routed for highly specialised technologies guidance. These criteria help make subjective judgements as informed, justifiable, consistent and predictable as possible. NICE's capacity to develop highly specialised technologies guidance can react to need and there is no limit on the number of technologies that can be routed.
Routing criteria
Technologies will be considered eligible for routing to highly specialised technologies guidance if they fulfil the selection considerations, are selected and meet all 4 of the routing criteria.
Routing criteria 1: The disease is very rare
'Very rare' is defined as a disease that has a prevalence in England lower than 1 in 50,000 people, or about 1,100 people.
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. Even if all the criteria are met the decision to route a technology to the HST Programme must still be judged to promote the purposes of that technology and align to the programme's vision.
Routing criteria 2: 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
The smaller the number of people eligible for the technology, the more likely this criterion will be met. A technology is unlikely to be considered suitable for the HST Programme if more than about 300 people are eligible for it.
If more than 300 people are eligible, the severity of the disease, unavailability of other effective treatments or a potential for significant benefits with the proposed technology are all considered. This is for the first indication under consideration for routing and it is capped at a maximum of 500 people for a technology with multiple indications. This includes all new active substances in their first indication and extensions to their marketing authorisation to add a significant new therapeutic indication, consistent with the definitions in the Department of Health and Social Care's 2024 voluntary scheme for branded medicines, pricing, access and growth. NICE has the discretion to apply some flexibility in these cases based on information and evidence gathered by the scoping exercise.
Routing criteria 3: The very rare disease for which the technology is indicated significantly shortens life or severely impairs quality of life
The terms 'significantly' and 'severely' are not defined because they require judgement.
Routing criteria 4: There are no other satisfactory treatment options, or the technology is likely to offer significant additional benefit over existing treatment options
Satisfactory treatments may include authorised medicinal products, medical devices, or other methods of treatment used in England. The term 'significant' is not defined because it requires judgement.