Detail set out in this section supersedes NICE's early value assessment interim statement and covers early-use HealthTech guidance (previously called early value assessment [EVA]).
Early-use assessments are an evidence-based approach designed to improve the care of people and effective use of NHS resources through quicker access to promising health technologies that address high unmet need for patients or the NHS. It champions stronger partnership working between regulatory, healthcare and research organisations to benefit people and better support innovators while ensuring value for money for the NHS.
There are 4 key aims of the early-use assessment approach:
To focus on promising innovations that meet the needs and priorities of people, and the health and social care system.
To enable earlier access to useful innovations through faster assessments and timely guidance production, while additional data is collected.
To better support use of technologies and evidence generation by embedding early-use assessments in cross-partnership working.
To realise the benefits of promising innovations and ensure value for money for the health and social care systems.
The aims will be achieved for selected technologies by:
identifying available evidence
exploring if the technologies have the potential to address the identified unmet need and offer value for money
helping inform further evidence generation for future evaluations
determining if any clinical, economic and system risk posed by early use can be managed and, consequently, if the technologies should be used while further evidence is generated.
Section 2.2 describes the approaches for using evidence for HealthTech guidance assessments. Early-use assessments happen earlier in the lifecycle of a technology, so the evidence assessment has been adapted to reflect this.
The aim of the evidence review is to identify the most relevant evidence relating to the decision problem defined in the scope. It is expected that the available evidence will vary substantially between topics and technologies. If no evidence is identified that is directly relevant to the decision problem, a broader evidence base should be considered. For example, evidence from the technology's use in a different population or setting.
Data on final outcomes may be limited so surrogate and intermediate outcomes should be considered. Supplemental searching and grey literature searching may also be helpful for technologies with limited published evidence. Broad evidence-mapping searches may need to be done to identify evidence on the technologies because articles may be published in less well-known journals, and studies may not be well indexed or may only be presented as conference abstracts. Companies and other stakeholders will be given the opportunity to provide evidence to NICE in response to an evidence request (see section 1.3.4). Published and unpublished studies provided by companies and other stakeholders should be considered.
Searches should also identify existing economic evaluations and resource and cost-impact analysis that addresses similar or related decision problems that may provide relevant information for the economic evaluation.
Searches for ongoing studies should also be done.
Pragmatic or rapid-review methodology and principles can be used in the literature review, with specific components of the systematic review process being either restricted or omitted. For example, the Cochrane Rapid Reviews Methods Group provides guidance on doing rapid reviews of the effectiveness of health interventions. Justification and rationale for this should be described in the assessment protocol, along with clear explanation of the components of the review process that have been restricted or omitted.
A full critical appraisal of all studies and outcomes is not expected. But the review should discuss the potential biases in key studies, how the risk of bias could affect key outcomes, and the generalisability of the results to clinical practice in the NHS.
The review should describe evidence gaps and suggest outcomes to focus on in future evidence generation, including those relating to patient safety. The report should describe any identified ongoing studies that may address the evidence gaps. It would also be beneficial to describe any data collections or real-world data sources that may address evidence gaps identified during the evidence review.
The economic evaluation that will be most beneficial for committee decision making is likely to vary by topic.
The key objectives of the economic evaluation are to:
assess how well the technologies are likely to resolve the specified unmet need
assess how likely the technologies are to offer value for money
identify uncertainties that are likely to be key drivers of decision uncertainty.
The economic evaluation should ideally generate estimates of clinical and cost effectiveness, or cost comparison, from an economic model. The model should follow, as closely as possible, the modelling methods and exploration of uncertainty as described in sections 4.6 and 4.7 in NICE technology appraisal and highly specialised technologies guidance: the manual. Advice on approaches to follow can be found in the NICE Decision Support Unit's report on economic evaluation in NICE early value assessments. For example, making greater use of existing models or model outputs, or if these are not available then producing simplified models or reporting intermediate outcomes with threshold analyses.
There is likely to be less evidence and limited time to develop full new models for early-use assessments. So, it should be pragmatically decided how to provide analyses that inform considerations of how likely the technologies are to offer value for money. Analyses can be provided that may be considered more exploratory or based on larger assumptions than would usually be considered to support guidance for routine use of technologies. The economic evaluation should clearly describe the limitations of these analyses and the assumptions made for them. The committee can then decide to what extent it uses such analyses in its decision making.
Using expert elicitation or expert opinion should be considered to provide evidence to support economic evaluation work (see sections 3.3.21 to 3.3.23 of NICE technology appraisal and highly specialised technologies guidance: the manual).
The reference case is the same as described in section 4.2 of NICE technology appraisal and highly specialised technologies guidance: the manual. Additional analyses can be presented when 1 or more aspects of methods differ from the reference case. But these must be justified and clearly distinguished from the reference case. Intention to provide such analyses, for example, a non-reference case type of economic evaluation, should be discussed with NICE as early as possible.
The economic evaluation should highlight any potential impacts of technology use that are not captured in model results. This could, for example, relate to impacts on the health and social care workforce or system efficiencies.
The economic evaluation should present any model outputs that show how well the technologies are likely to resolve the specified unmet need.
Key goals for decision making in early-use assessments are to decide if technologies should be used as an option in the NHS while further evidence is generated and to prioritise uncertainties that need to be reduced by collection of further data to support future decision making. This evidence is for future NICE guidance to decide whether to recommend a technology for routine use.
Recommendations will only be for the use, or uses, of the technologies as specified in the scope.
When making decisions the committee will consider if a technology has plausible potential to address the specified unmet need and offer value for money. It will also consider how much any clinical, system or economic risks of using the technology could be managed in practice (further description is provided in sections 2.1.24 to 2.1.26). The flow chart in figure 1 describes how these considerations link to available recommendations.
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Is there plausible potential that the technology will address the specified unmet need? Considerations include the extent that this is supported by available evidence and other relevant information (including the views and experiences of people who will use the technology).
Is there plausible potential that the technology offers value for money? This consideration is based on assessing if the technology is expected to be cost effective, including providing similar or greater health benefits at similar or lower cost than the relevant comparator. Technologies considered unlikely to offer value for money typically would not meet this criteria. Considerations include:
The likely size of any impacts of technology use (positive, including addressing the unmet need, and negative) on patients, and, when relevant, carers, the NHS and personal social services (including impacts on system efficiencies), and the extent that available evidence or other information supports this.
Analyses done as part of the economic evaluation work to assess how likely the technology is to offer value for money.
How much can any clinical, system or economic risks posed by using the technology be mitigated or managed in practice? For example, by specifying how the technology should be used or whether provision could be made for special safety monitoring measures. Or, if there could be reductions in technology cost or alternative ways in which the technology is charged for, particularly if there are large irreversible costs associated with using it.
Should the technology be used in the NHS as an option while further evidence is generated? Important considerations include:
the potential of the technology to address the specified unmet need and offer value for money
the extent that any risks of using the technology in practice can be managed.
If there are greater risks associated with early use (for example, substantial irreversible costs that cannot be recovered if a recommendation for use is later changed), there should be greater confidence that the technology is expected to offer value for money. The extent to which the identified uncertainties (see section 2.4.28) will be resolved by ongoing evidence generation activities (that is, that are occurring or will occur regardless of a NICE recommendation), the timescale for this and any possible impact of a recommendation on these activities should also be considered.
When multiple technologies are considered, each should be assessed independently, unless the committee believes it is appropriate for available data that has been generated using a technology to be used for others. The committee may need to consider any difference between technologies in terms of whether they may solve the specified unmet need and any differences in further evidence needs. Different recommendations can be made for different technologies included in the guidance.
Use while further evidence is generated
If there is plausible potential that the technology will address the unmet need and offer value for money, and that any economic, system or clinical risks posed by uncertainty in evidence can be managed, then the technology can be used in the NHS while further evidence is generated (during the evidence generation period) and be paid for using core NHS funding. Any identified measures for mitigating risks of using the technology are presented with the recommendation.
This recommendation needs to be accompanied by defined uncertainties that the committee considers are worthwhile and feasible to address by collecting further evidence during the evidence generation period (see section 2.4.28).
Technologies should only be used in the NHS during the evidence generation period if the evidence outlined in the evidence generation plan is being generated.
More research is needed
More research is needed before the technology can be used routinely or funded by the NHS. Access to the technology (for the use or uses assessed in the guidance) should only be through company, research or non-core NHS funding. This can be because it is too uncertain that the technology will address the unmet need or offer value for money.
Should not be used
If the technology will not address the unmet need or offer value for money. For example, because of how it functions, potential safety issues or based on available evidence on performance, particularly if it has a high cost.
For recommendations for use while further evidence is generated during the evidence generation period the uncertainties that the committee needs further data on to support future decision making should be listed and include a focus on those that:
are essential to future decision making, and
can be resolved in 3 years from the point of guidance publication (longer periods than this will only be allowed in exceptional circumstances).
Technologies recommended for use while further evidence is generated and that complete the evidence generation process (see section 1.7) can be re-evaluated by NICE (see section 1.8).