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  • Question on Consultation

    Do you have any comments related to routing criterion 1?

  • Question on Consultation

    Do you have any comments related to routing criterion 2?

  • Question on Consultation

    Do you have any comments related to routing criterion 3?

  • Question on Consultation

    Do you have any comments related to routing criterion 4?

  • Question on Consultation

    Do you have any comments on the overall proposal for the refinement of the existing HST routing decision criteria?
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National Institute for Health and Care Excellence

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Appendix A Comparisons of existing and refined HST routing criteria

The existing and proposed refined HST routing criteria

Existing HST routing criteria

Proposed refined HST routing criteria

Criterion 1

The disease is very rare, defined by 1:50,000 in England.

Criterion 3

The very rare disease for which the technology is indicated significantly shortens life or severely impairs its quality.

The disease is ultra-rare and debilitating

Routing criterion 1

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

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

  • is lifelong after diagnosis with current treatment, and

  • has an exceptional negative impact and burden on people with the disease.

Definitions

  • The first 2 bullet points of routing criterion 1 are about the 'disease', not about the symptoms associated with the disease (regardless of whether the symptom or set of symptoms are the dominating feature).

  • 'Disease' refers to a condition for which a diagnosis can be made based on the International Classification of Diseases (ICD-10 or ICD-11) developed by the World Health Organization (WHO). 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 when these are not clinically meaningful (that is, associated with a unique and clinically distinct phenotype, prognosis or treatment options).

  • '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 on a given index date compared with the total population of England at that time (NHS England).

  • 'Lifelong' indicates that the disease the people currently have:

    • needs ongoing clinical management, supportive care or both

    • is not relapsing–remitting, with periods when a person is free of symptoms and disease burden.

  • 'Exceptional negative impact' refers to shortened length of life or severely impaired quality of life. The precise assessment of what these are needs an element of subjective judgement.

Not applicable

We aim to encourage innovation and research

Routing criterion 2

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

Definitions

  • '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 disease (compared with the existing treatment or best supportive care).

  • 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

    • the technology should not currently be being explored in clinical trials for other indications.

  • A repurposed technology means new uses for medicines that are outside the scope of the existing licence for the medicine. This typically involves taking an existing medicine that already has a marketing authorisation or licence for human use for a particular condition, and then using it to treat another condition (Repurposing medicines in the NHS in England).

Criterion 2

Normally, no more than 300 people in England are eligible for the technology for its licensed indication and no more than 500 across all its possible indications

The technology should be limited to the population in its licensed indication

Routing 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.

Definitions

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

  • To promote innovation, 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' under consideration

    • should not be for an extension of an existing indication for another population or disease, or for a subgroup of people with the ultra-rare disease

    • 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, or other populations with other diseases

    • is not a repurposed technology.

  • '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.

Criterion 4

There are no other satisfactory treatment options, or the technology is likely to offer significant additional benefit over existing treatment options

There are no effective treatment options

Routing criterion 4

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

Definitions

  • 'Substantial additional benefit' means that the technology demonstrably extends the reduced length of life or demonstrably improves the severely impaired quality of life attributable to the disease, as exemplified by research data on relevant patient-reported outcome measures (PROMs).

  • 'The technology' means:

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

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