How are you taking part in this consultation?

You will not be able to change how you comment later.

You must be signed in to answer questions

    The content on this page is not current guidance and is only for the purposes of the consultation process.

    NICE integrated topic prioritisation and strategic principles

    Closed for comments This consultation ended on at   Request commenting lead permission

    Appendix 3: Highly specialised technologies

    NICE's highly specialised technologies programme identifies and evaluates technologies for very rare conditions that need the specific considerations and flexibilities permitted by this programme. Specifically, it evaluates technologies that:

    NICE's standard technology appraisals methods and processes are designed to be flexible and adaptable for all technologies (including health or medical technologies) and conditions. So, they are suitable for almost all technologies that treat rare conditions and small populations. For more information about the methods and processes used for standard technology appraisals, see NICE's health technology evaluations: the manual. NICE's highly specialised technologies programme is a deliberate departure from this. It recognises the challenges of generating evidence in very small populations with severe, very rare conditions. It also acknowledges that an appropriate value framework is needed to evaluate technologies that meet the programme's criteria.

    The programme has set criteria to identify technologies for very rare conditions for which there is substantial unmet need and people with those conditions would otherwise be disadvantaged. The highly specialised technologies methods and processes are only used in exceptional circumstances, that is, when all the following apply:

    • There is a need to provide fair and equitable access to technologies for people with very rare and serious conditions that significantly shorten life or severely impair its quality.

    • There are substantial challenges associated with the very rare condition, including difficulties in collecting enough good quality evidence.

    • It is likely that the technology would not be recommended using standard methods and processes, even if provided with substantial discounts or other economic mechanisms.

    • The developers are unable to recover the cost of bringing the technology to market under normal market conditions because of small patient numbers and other barriers to evidence generation, such as lack of natural history data for the condition.

    However, it can be difficult to identify those exceptional circumstances when the highly specialised technologies methods and processes should be used because of the difficulties in obtaining the information needed. Proxy information is often relied on and used to make subjective judgements. Routing criteria have been developed to 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; there is no limit on the number of technologies that can be routed. Also, the routing criteria are not a tool to manage affordability or budget impact in the NHS. This is managed by other measures such as the budget impact test and commercial arrangements.

    Highly specialised technologies routing criteria

    Technologies will be considered eligible for routing to highly specialised technologies guidance if they fulfil eligibility criteria (see sections 6.3.3 and 6.3.4) for selection (see section 7) and meet all 4 of the highly specialised technologies routing criteria.

    The condition is very rare

    'Very rare' is defined as a condition that has a prevalence in England lower than 1 in 50,000 people, or about 1,100 individuals in total.

    In exceptional circumstances, a technology may be routed to highly specialised technology guidance even if the condition 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 satisfy all of the remaining highly specialised technologies routing criteria to possibly benefit. Even if all the criteria are met, the decision to route a technology to the highly specialised technologies programme must still be judged to promote the purposes of that technology.

    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 highly specialised technologies programme if more than about 300 people are eligible for it. If more than 300 people are eligible, the severity of the disease, and whether there is lack of other effective treatments or a potential for significant benefits with the proposed technology are all considered.

    One-off treatments would normally be considered acceptable if they have an eligible prevalent population of up to about 50 people and an eligible incident population of no more than about 40 people a year. This is for the first indication under consideration for routing. This is capped at a maximum of 500 patients for a technology with multiple indications; this means 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 2024 Department of Health and Social Care 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.

    The very rare condition 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.

    There are no other satisfactory treatment options, or the technology is likely to be of significant additional benefit to 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.

    ISBN: To be added.