Highly specialised technologies: NICE prioritisation board routing criteria
2 HST routing criteria
Technologies will be considered eligible for routing to the HST Programme if the NICE prioritisation board agrees that all 4 routing criteria have been met (see section 3). Each criterion has a set of definitions that help to explain how it will be assessed. The criteria should not be assessed in isolation. The NICE prioritisation board will always consider all the definitions when assessing each routing criterion.
2.1 Criterion 1: The disease is ultra-rare and debilitating
Description of the HST Programme's vision
The rarer a disease is, the more challenging it is to do research and generate an evidence base that is robust enough to bring an effective technology to market. The HST Programme's vision aims to encourage research when it is most challenging.
Not all ultra-rare diseases are debilitating. The vision focuses on ultra-rare diseases that cause ongoing debilitating symptoms and have an exceptional burden on the people with them, and on their carers and families. This is to justify prioritising access to HST technologies over overall population health.
Routing criterion 1
The disease is ultra-rare and debilitating, that is,
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1A: it is defined as having a point prevalence of 1:50,000 or less in England (NICE strategic principles for rare disease).
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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 carers and families.
Definitions
These definitions have been developed to help define what an ultra-rare disease is, and the debilitating nature of the disease. Relevant information should be collected during scoping by NICE (from the company, and other research or academic sources) to explain how each definition is considered by the NICE prioritisation board.
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1A of routing criterion 1 is about defining the ultra-rare 'disease', not about the symptoms associated with the ultra-rare disease (regardless of whether the symptom or set of symptoms are the dominating feature). 1B of routing criterion 1 is about the characteristics of the ultra-rare disease.
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'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:
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clinical examination
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patient history
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imaging or laboratory tests that are, or can be made, available in the NHS in England.
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'Disease' does not refer to subgroups based on age, sex, severity, or genetic subtype. These will only be considered if they are clinically meaningful.
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'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).
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'Lifelong' indicates that the disease needs ongoing clinical management, supportive care, or both.
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'Exceptional negative impact' refers to shortened length of life or severely impaired quality of life. The precise assessment of this will require an element of subjective judgement.
2.2 Criterion 2: NICE aims to encourage innovation and research
Description of the HST Programme's vision
This criterion is designed to uphold the HST Programme's vision to encourage innovation and research into ultra-rare and debilitating diseases for which there is poor service provision within the NHS (for example, delay in diagnosis, no treatment options beyond supportive care). Without these incentives from the HST Programme, the technology may not be available either after launch, or during development or testing of the technology in England. The availability of the innovation can also reshape NHS services and advance awareness.
Routing criterion 2
The technology is an innovation for the ultra-rare disease.
Definitions
These definitions have been developed to help define an innovative technology. Information about the technology should be collected by NICE from relevant sources (for example, the Medicines and Healthcare products Regulatory Agency [MHRA], ongoing trials, registries) to explain how each definition is considered.
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'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).
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To ensure the technology is an innovation for the ultra-rare disease:
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the technology should not be a repurposed technology
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the indication for the technology should not be a significant extension of an indication from another population or disease.
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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. This can also include generic treatments or treatments that have had marketing authorisation withdrawn and the developer is seeking a new indication.
2.3 Criterion 3: The technology should be limited to the population in its licensed indication
Description of the HST Programme's vision
This criterion is designed to establish the acceptability of the technology as an effective use of NHS resources, considering the significantly higher ICER threshold. So, the eligible population needs to be small. This is to strike a balance between the desirability of supporting access to treatments for ultra-rare diseases and the inevitable reduction in overall health gain across the NHS because of a higher ICER threshold. A small subpopulation within a population with a common disease would not be suitable for the HST Programme.
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
These definitions have been developed to help define what kind of licensed indication is suitable for a technology to be considered for routing to the HST Programme, and to help explain what an individualised medicine is. Relevant information about the licensed indication of the technology should be collected by NICE to explain how each definition is considered.
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'Eligible' refers to everyone who could have the technology under its marketing authorisation (obtained or in the process of being obtained) in England.
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The 'technology' should only be developed for the ultra-rare disease, so the eligible population is small. The technology:
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has to be the first licensed treatment indicated for the ultra-rare disease under consideration
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should not be an extension of an indication from another:
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related population or disease, or
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subgroup of people with the same ultra-rare disease under consideration
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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.
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'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.
2.4 Criterion 4: There are no effective treatment options
Description of the HST Programme's vision
This criterion is designed to address the lack of effective treatment and access to NHS services for some ultra-rare diseases. To justify prioritising treatment access for ultra-rare diseases over overall population health, the technology under consideration should be anticipated to provide substantial health benefits to people with the disease over existing clinical management and supportive care.
Routing 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.
Definitions
These definitions have been developed to help define what is substantial additional benefit, and to help to explain the meaning of no other treatment options. Relevant information should be collected by NICE to explain how each definition is considered.
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'Substantial additional benefit' means that the technology is likely to:
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significantly redress the reduced length of life, or
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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).
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'The technology' means that:
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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
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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.
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