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Highly specialised technologies: NICE prioritisation board routing criteria

Appendix A: Highly specialised technology (HST) routing assessment checklist

Introduction

The NICE HST routing assessment checklist highlights when a technology meets or does not meet the criteria for routing it to the HST Programme. All 4 criteria need to be met for a technology to be routed.

HST routing criteria checklist
Routing criteria Descriptions of how the criteria are met or not met through assessing the definitions

Criterion 1

The disease is ultra-rare and debilitating, that is:

  • 1A: It is defined as having a point prevalence of 1:50,000 or less in England (NICE strategic principles for rare disease).

  • 1B: It is lifelong after diagnosis with current treatment, and has an exceptional negative impact and burden on people with the ultra-rare disease, and their families and carers.

'Disease' refers to a condition for which a diagnosis can be made using the International Classification of Diseases (ICD-11) developed by the World Health Organization (WHO) as a guiding tool. Diagnosis is based on a unique set of signs and symptoms (characteristics) identified using:

  • clinical examination

  • patient history

  • imaging or laboratory tests that are, or can be made, available in the NHS in England.

'Disease' does not refer to subgroups based on age, sex, severity, or genetic subtype. These will only be considered if they are clinically meaningful.

Have these definitions been met or not met?

[Yes or No]

Notes and rationales:

'Point prevalence' refers to the point prevalence of the 'disease' in England. It counts the number of people with a diagnosis of the disease thought to be alive in England (numerator) on a given index date compared with the total population of England (denominator) at that time (NHS England).

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

'Lifelong' indicates that the disease needs ongoing clinical management, supportive care, or both.

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

'Exceptional negative impact' refers to shortened length of life or severely impaired quality of life. The precise assessment will require an element of subjective judgement.

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

Criterion 2

The technology is an innovation for the ultra-rare disease.

'Innovation' refers to a technology or medicine such as an advanced therapy medicinal product (ATMP), a new chemical or biological entity, or a novel drug device combination that brings additional health gains to people with the ultra-rare disease (compared with existing treatment or best supportive care).

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

To ensure the technology is an innovation for the ultra-rare disease:

  • the technology should not be a repurposed technology

  • the indication for the technology should not be a significant extension of an indication from another population or disease.

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

Criterion 3

No more than 300 people in England are eligible for the technology in its licensed indication, and the technology is not an individualised medicine.

'Eligible' refers to everyone who could have the technology under its marketing authorisation (obtained or in the process of obtaining) in England.

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

The 'technology' should only be developed for the ultra-rare disease, so the eligible population is small. The technology:

  • has to be the first treatment for the 'licensed indication' for the ultra-rare disease under consideration

  • should not be an extension of an indication from another:

    • related population or disease, or

    • subgroup of people with the same ultra-rare disease under consideration.

  • is unlikely to be suitable for other subgroups of the population with the ultra-rare disease in the future who are outside of its first indication.

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

'Individualised medicine' refers to a medicine that is developed based on a person's unique genetic profile (n of 1), or on the genetic profile of monozygotic twins or triplets.

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

Criterion 4

The technology is likely to offer substantial additional benefit for people with the ultra-rare disease over existing established clinical management, and the existing established clinical management is considered inadequate.

'Substantial additional benefit' means that, the technology is likely to:

  • significantly redress the reduced length of life, or

  • is likely to demonstrate substantial improvements in the severely impaired quality of life attributable to the ultra-rare disease, as exemplified by research data on clinically relevant measures, for example, patient-reported outcome measures (PROMs).

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

'The technology' means that:

  • if the technology is a disease-modifying treatment (including curative treatment), there is no other disease-modifying treatment available in the NHS in England for the same ultra-rare disease at the time of the routing decision, or

  • if the technology treats a symptom or set of symptoms unique to the ultra-rare disease, there is no other treatment available in the NHS in England for the same symptom for which the technology is indicated at the time of the routing decision.

Has this definition been met or not met?

[Yes or No]

Notes and rationales:

Overall routing decision:

[highly specialised technology or technology appraisal]

Other comments:

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