The scoping process aims to define what question the evaluation will answer and what will and will not be included. The scope provides the framework for the evaluation. It defines the issues for consideration (for example, population, comparators, care pathway, and outcome measures) and sets the boundaries for the work to be done by the external assessment group, and any evidence submissions for the evaluation.
The areas detailed in the scope include:
the disease or health condition and the population(s) for whom the technology is being evaluated
the technology being evaluated (and where it will be used, for example, in a hospital inpatient or outpatient setting, or in the community)
the care pathway
the relevant potential comparator technologies (and where they are used, if relevant)
the principal outcome measures appropriate for the analysis
the costs, including when the Department of Health and Social Care asks NICE to consider costs (or savings) to the public sector outside the NHS and personal social services
the costs of any companion diagnostic needed for a treatment, if not in routine use
the time horizon over which health effects and costs will be evaluated
consideration of patient subgroups for whom the technology might be particularly clinically effective or value for money
issues relating to advancing equality of opportunity, eliminating unlawful discrimination, and fostering good relations between people with particular protected characteristics and society as a whole
the remit of the evaluation for any technologies referred by the Department of Health and Social Care
other special considerations and issues that are likely to affect the evaluation, for example, existing relevant NICE guidance and the innovative nature of the technology.
For new technology appraisals and highly specialised technologies guidance, scoping normally takes place during (and is used in) topic selection. For new medical technologies and diagnostics guidance, scoping takes place after topic selection and the evaluation follows immediately after.
If the scoping process gathers additional information that suggests the topic should be evaluated by a different guidance programme, NICE may pause progression of the evaluation to request that the topic selection oversight panel reconsider the routing decision (see NICE health technology evaluation topic selection: the manual). Routing decisions are not subject to appeal.
For updates of published guidance, the process starts with the scoping stage and the evaluation follows immediately after.
The scope briefly describes the condition relevant to the technology being evaluated, with information on its prognosis, epidemiology and standard care or alternative technologies used in the NHS.
The scope includes information about the regulatory approval of the technology, and the stage of approval for technologies that have not yet received approval. The scope specifies how and in what circumstances technologies are used, particularly if this is different from that of alternative technologies or standard care for the same patient group, or when there are several other circumstances in which the technology may be used.
The technology may have multiple uses. For medicines, the relevant use will normally depend on the (expected) marketing authorisation or marketing authorisation extension. For other types of technology, the most relevant use (within the intended purpose or specified indications for use) is notified to NICE and included in the topic selection briefing. The scoping stage refines and clarifies the use of the technology in the clinical pathway after input from clinicians, patients and other stakeholders. The considerations include: the uses of the technology most likely to maximise benefit to the NHS and the population of England; areas of unmet need; and the degree of complexity of the assessment.
For diagnostic evaluations, alternative technologies that are not in common use or are newly available (or soon to be) may be included with the notified technology. The scoping process for these alternatives is similar to that for the technology. Alternative technologies are normally similar in action or intent to the notified technology. They are generally included when, for example, the technology might be used in very similar settings or circumstances and there is likely to be some benefit to the NHS in developing guidance on more than one technology.
A technology is only evaluated if it has or is expected to have regulatory approval (or appropriate regulatory signal) by the planned draft or final guidance publication date.
Unless the Department of Health and Social Care specifically indicates otherwise, NICE will not develop technology appraisal or highly specialised technology guidance on a technology for indications that have not been given regulatory approval in the UK. That is, for unlicensed or 'off-label' use outside the terms of the technology's regulatory approval.
For diagnostic technologies that are used in sequence, all technologies that make up the potential sequence should be included. The technology being evaluated needs to be precisely described because there may be many variants of a single technology that could be used (for example, different thresholds). These variants may need to be evaluated separately, or in different sequences.
All technologies that are to be included and evaluated as part of a technology evaluation must meet the eligibility criteria for selection in the topic selection manual.
The scope defines the population for whom the technology is being evaluated as precisely as possible. The scope may highlight potential subgroups of the population when the clinical effectiveness or value for money of the technology might differ from the overall population, or groups who need special consideration.
Outcomes can vary significantly depending on the population evaluated. For example, there may be differences in: the prior probabilities for various conditions identified by the technologies; test accuracy in different populations; the effect of treatment, and side effects or complication rates. For many technologies there may be multiple populations. For medicines, the relevant population will normally be informed by the (expected) marketing authorisation or marketing authorisation extension. In other cases, to keep the evaluation to a reasonable size, some people who could use the technology may not be included in the scope. Excluding people from the scope does not mean that the technology is not appropriate for these people. Because resources for the evaluation are limited, the patient populations may need to be selected carefully to maximise the benefit of the evaluation.
Defining the population also includes where, why and how the technology is used in the care pathway. This is described for each defined population.
The scope identifies all potentially relevant comparators that are established practice in the NHS. It considers issues likely to be discussed by the committee when selecting the most appropriate comparator. At this stage of the evaluation, identifying comparators should be inclusive.
Comparator technologies may include branded and non-proprietary (generic) medicines and biosimilars. They may also include technologies that do not have regulatory approval for the population defined in the scope if they are considered to be established clinical practice for the population in the NHS.
Sometimes both the technology and comparator or standard care are part of a sequence in the care pathway. In these cases, the evaluation may compare alternative sequences.
Technologies that NICE has recommended with managed access are not considered established practice in the NHS and are not considered suitable comparators.
The care pathway is an important consideration for evaluating the technologies' effectiveness and costs. It includes the entire sequence of tests and treatments relevant to the evaluation. It may also include technologies used to help with any adverse effects. The care pathway can vary depending on the patient's conditions, characteristics or comorbidities. It includes the stages after diagnosis or treatment. The treatment pathway or range of treatment pathways must be understood for the value of the technology to be assessed.
If appropriate, the scope describes the care pathway. For a diagnostic technology it includes any variations according to test results or the technologies used. It defines the time frame for the treatments covered, key steps leading to final outcomes, and the outcomes relevant to treatments that will be included in the evaluation. It covers the diagnostic sequences, treatments, monitoring, retreatment, treatment for adverse effects and complications that a person may have. In some cases, the care pathway includes tests and interventions that are not done because of the results of the test being evaluated. For example, if a test diagnoses a condition that would not have been diagnosed by the comparator, then the benefits of not having other treatments or tests are relevant. Even if a test diagnoses an untreatable condition, the costs and harms of treatment that can now be avoided are relevant.
As far as possible, the scope identifies the main measures of outcomes that are relevant to estimating clinical effectiveness. That is, they measure health benefits and adverse effects that are important to patients and their carers. For evaluations in which quality-adjusted life years (QALYs) are calculated, the clinical outcome measures may include quantification of survival or health-related quality of life that translates into QALYs to evaluate cost effectiveness.
Relevant outcomes include any health outcomes resulting directly or indirectly from any technologies being evaluated. They may also include informational outcomes of value to the patient for the relief, or imposition, of anxiety or for personal planning.
People with the condition should be consulted when selecting outcome measures in studies. A high-quality 'core outcome set', developed with people with the condition, may help with outcome selection. Core outcome sets should be used if suitable based on quality and validity; one source is the COMET database. The Core Outcome Set Standards for Development (core outcome sets-STAD) and Core Outcome Set Standards for Reporting (core outcome sets-STAR) should be used to assess the suitability of identified core outcome sets.
Patient-reported outcome measures can capture important aspects of conditions and interventions such as health-related quality of life, performance status, symptom and symptom burden, and health-related behaviours such as anxiety and depression. They can be either general or disease specific.
The potential effect on resource costs and savings that would be expected from introducing the technology should be considered from the perspective of the NHS and personal social services. In exceptional circumstances for medicines, when requested by the Department of Health and Social Care in the remit for the evaluation, the scope will list requirements for adopting a broader perspective on costs.
The scope defines the relevant cost areas for the evaluation, but it does not detail all the specific costs and other resource details to be incorporated in the evaluation.
The scope can include, when relevant, details of:
Related NICE guidance, such as other evaluations and clinical guidelines.
Related policy developments.
Service settings related to the technology being evaluated that are either of particular interest or will be excluded from consideration.
The potential innovative nature of the technology, in particular its potential to have a substantial effect on health-related benefits that are unlikely to be included in the economic evaluation.
The evidence available, including any evidence gaps that may cause uncertainty during the evaluation, and whether the company believes the technology is a candidate for managed access.
Issues relating to advancing equality of opportunity, eliminating unlawful discrimination and fostering good relations between people with particular protected characteristics and society as a whole.
Potential issues relating to health inequalities, including whether the technology could address inequality or unfairness in the distribution of health across society.
Which type of evaluation is most appropriate for the technology.
Issues relating to the technology that are not particular to specific clinical situations. For example, a new imaging machine may have costs or radiation exposure that cover a broad range of clinical conditions. In this case the scope defines a wide category of patients, but the subsequent care pathway for those patients may not be included in the evaluation.
After identifying topics through the topic selection process, NICE develops a draft scope for each potential evaluation and seeks the views of stakeholders.
The first step in the scoping process is to identify information about the technology or technologies. This is done using literature searches, checking the availability of relevant evidence, and requesting information from the company. NICE uses this information, along with the technology briefing, to prepare a draft scope.
Identifying stakeholders is an important stage of the process. NICE identifies stakeholders before it consults on the draft scope or holds a scoping workshop.
A patient and carer organisation or professional organisation can be a stakeholder if it works at a national level (covering the UK or England, or a UK branch of an international body) and represents patients, carers or healthcare professionals either broadly or directly related to the technology being considered. Other stakeholders include the company, NHS commissioning groups and specialist centres that manage care in conditions with small patient populations.
When there is no patient and carer organisation working at a national level for the technology being considered, as defined above, NICE may request and approve an international organisation becoming a stakeholder in the evaluation at its discretion.
Stakeholders also include research organisations with an interest in the technology, developers or distributors of a relevant technology, a provider of NHS services in England, organisations that cover the NHS as a whole such as the NHS Confederation, patient and professional organisations covering Northern Ireland or Scotland or Wales only, and relevant comparator and companion diagnostic test companies. Other organisations may be included as stakeholders when appropriate.
Any specialist committee member applicants for diagnostic evaluations are automatically registered as stakeholders.
During the scoping phase, NICE aims to identify the widest possible range of relevant stakeholders who have an interest in the technology or disease area being considered. This includes, but is not restricted to, national organisations representing relevant specific ethnic groups, disabled people, and people with mental health problems or a learning disability.
It is important that enough expertise goes into developing the scope. NICE welcomes and values all input from stakeholders at consultation and during workshops.
If an organisation wants to be a stakeholder, it needs to contact the project manager (see the NICE website for details). Organisations can ask to take part as a stakeholder at any point up to the issue of final draft guidance.
For guidance being updated, including those which are recommended with managed access, NICE will update the original stakeholder list ahead of the guidance update commencing.
The aim of the consultation is to make sure the evaluation covers all the relevant areas and issues.
NICE sends the draft scope and stakeholder list to stakeholders for comment and asks them if there are other organisations that need to be included in the consultation. The draft scope and list of stakeholders is then published on the NICE website.
Consultations are either 28 days (long), 14 days (medium), or 7 days (short). Long consultations will be used if there is a reasonable degree of uncertainty about elements of the draft scope or whether the technology should be evaluated. If the draft scope contains only a small degree of uncertainty, or a scope has previously been well defined in other related NICE outputs in the last 12 months, a medium or short consultation may be used. Please see the consultation lengths table below for distinctions between the consultation lengths.
|
Length of time |
When consultation length is used |
|---|---|
|
7 calendar day consultation (short) |
For medical technologies – information for the scope has already been gathered during the development of the medtech innovation briefing. Technology appraisals and highly specialised technologies will not use this approach. |
|
14 calendar day consultation (medium) |
If there is a small degree of uncertainty or a scope has previously been well defined in other related NICE outputs within the last 12 months. |
|
28 calendar day consultation (long) |
If there is a reasonable degree of uncertainty about elements of the draft scope, or whether the technology should be evaluated. Diagnostics, technology appraisals and highly specialised technologies will normally use this approach. |
NICE asks the company to confirm the expected timing and details of regulatory approval in the UK. Companies should also highlight any evidence gaps and if they intend to make a managed access proposal to generate more evidence, as part of their response to the draft scope consultation.
NICE publishes the draft scope and list of stakeholders on its website, for information, within 7 days of sending these documents to stakeholders.
For diagnostics evaluations, NICE normally holds a scoping workshop and does not have a consultation on the draft scope.
At scoping consultation, questions will be asked relating to the population, treatment pathway, benefit and clinical similarity to help establish whether cost comparison is appropriate. The aim is to establish whether the intervention is clinically similar, such that it can be compared with another intervention that NICE has previously recommended in technology appraisal guidance for the same indication, using cost-comparison methods. The chosen comparator must be established in practice and have substantial use in the NHS in England for the same indication.
The draft scope sent out at consultation will indicate whether NICE considers a cost-comparison evaluation would be a potentially suitable process to follow. NICE welcomes early engagement from the company on cost-comparison suitability.
During scope consultation, NICE's medicines optimisation team will engage with medicines and prescribing associates to create a briefing report on the appropriateness of cost comparison. This report will be published alongside topic information on the NICE website.
The scoping consultation will enable NICE to decide on the suitability of the cost-comparison process, taking into account input from stakeholders. If it is established that cost comparison is appropriate, NICE will invite stakeholders to make a cost-comparison submission. If cost comparison is not appropriate, stakeholders will be invited to submit to a single or multiple technology appraisal. This decision will consider relevant risks associated with the appraisal and the decision to use cost comparison. Decisions on which process a topic will follow are not subject to appeal.
NICE decides whether to hold a scoping workshop to discuss the draft scope with stakeholders. This may happen if the topic covers a new disease area or care pathway that NICE has not evaluated before or recently, or there are uncertainties about the evaluation that a workshop could address. The scoping workshop will usually be held virtually. NICE invites stakeholders to send representatives to this workshop.
The aims of the workshop are to:
make sure the scope is appropriately defined
discuss the issues raised by stakeholders during any previous consultation
discuss the appropriateness of completing an evaluation and the appropriate evaluation process
discuss any other issues relevant to the potential evaluation.
At the scoping workshop, the company can provide preliminary details of the evidence it will submit in the evaluation. This may include details of trials in progress, for example the inclusion and exclusion criteria used, and any evidence gaps that may cause uncertainty during the evaluation.
At the end of the workshop, if needed, the company can confidentially discuss commercially sensitive information and technical issues about the proposed evaluation with NICE.
For diagnostic technologies, after the scoping workshop, NICE meets with the assessment subgroup (committee chair, specialist committee members, committee lead and the external assessment group) to agree the final scope and protocol for the evaluation.
For technologies that were recommended with managed access, NICE will update the original scope. This is to make sure that the guidance update considers the care pathway and use of the technology in England at the time the guidance update starts. NICE can review any element in the scope, including changes that happened during the managed access period to the:
eligible patient population
treatment pathway
relevant health outcomes measures.
NICE may also consider whether to expand the scope of the guidance update for technologies that had recommendations (see section 6.4) with managed access.
When changes to the original scope are identified after a period of managed access, NICE will consult on a draft scope as described above.
When no or limited changes to the original scope are identified after a period of managed access, the original scope and stakeholder list will have a consultation period of 14 days.
NICE may hold a scoping workshop as part of scoping activities for technologies after a period of managed access when there are issues relevant to the guidance update or uncertainties in the draft scope that a workshop could address. At the scoping workshop, the company will be asked to provide preliminary details of the evidence it will submit in the evaluation. This may include details of trials in progress, for example the inclusion and exclusion criteria used, and any evidence gaps which may cause uncertainty during the evaluation.
NICE updates the scope, considering comments received during any consultation, and the discussions at any scoping workshop.
It may become clear during scoping that a topic is not suitable for evaluation and NICE may decide not to proceed. The decision is made by the centre director or topic selection oversight panel. Stakeholders are told about the decision and the reason why.
If the scope for a diagnostic evaluation is too large for the available resources, NICE may revise it in collaboration with the assessment subgroup and the external assessment group.
If there is a significant length of time between scoping and the evaluation, NICE may need to update the scope. Depending on the extent of the update, NICE may engage further with stakeholders. NICE does not routinely hold another scoping workshop.
The final scope is signed off internally and published on NICE's website once NICE is ready to start the evaluation.