NICE health technology evaluation topic selection: the manual

The Highly Specialised Technologies 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:

• 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

• need consideration using the methods and processes of the Highly Specialised Technologies 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 Highly Specialised Technologies Programme is designed to be used in exceptional circumstances. Its purpose is to evaluate technologies for very rare diseases that have:

• small numbers of patients

• limited or no treatment options

• challenges for research and difficulties with collecting evidence, because of the uniqueness of the disease.

The Highly Specialised Technologies Programme aims to:

• 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

• secure fairer and more equitable treatment access for very small populations with very rare diseases

• 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 Highly Specialised Technologies Programme acknowledges that:

• 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 Highly Specialised Technologies 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.

• Each time NICE routes a topic to the Highly Specialised Technologies 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.

• 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 Highly Specialised Technologies 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.

• This approach ensures that technologies routed to the Highly Specialised Technologies 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.

Technologies will be considered eligible for routing to highly specialised technologies guidance if they fulfil the selection considerations, are selected (see section 6) and meet all 4 of the routing criteria.