The committee makes recommendations to NICE about the clinical effectiveness and value for money of technologies for use within the NHS. The committee will not recommend technologies if the benefits to patients are unproven, or if the technologies are not considered to be a good use of NHS resources. NICE is responsible for publishing the final guidance.
When forming its recommendations to NICE, the committee considers those factors it believes are most appropriate for each evaluation. In doing so, the committee takes into account the provisions and regulations of the Health and Social Care Act 2012 relating to NICE, and NICE's legal obligations on equality and human rights. The Act expects NICE, when doing its general duties, to be aware of:
the broad balance between the benefits and costs of providing health services or social care in England.
the degree of need of people in England for health services or social care.
the desirability of promoting innovation when providing health services or social care in England.
In reaching its decision, the committee bases its recommendations on the evidence presented, including statements from stakeholders and, when relevant, the views expressed by experts at the committee meeting. Formulating the committee discussion section of the guidance is an important component of the committee's work. These sections identify the key evidence considered by the committee and its views on this evidence. They highlight any areas of contention and uncertainty that have arisen during the committee's discussions of the evidence. They also present a general description of the committee's views on the written and oral inputs that have informed its decision.
The committee's provisional recommendations may be released for widespread consultation with stakeholders and the public. In reviewing responses to consultation, the committee is most interested in comments on its preliminary recommendations within the context of the evidence base reviewed at its first meeting and its consideration of that evidence. The comments received on the key issues identified at the first meeting are carefully reviewed.
The committee considers the effect of the consultation comments on:
the preliminary recommendations on the use of the technology
the other sections of the consultation document
recommendations for further research
issues for implementation, including:
resource availability to support implementation (for example, workforce planning and training, and new clinics)
the extent of any changes in current clinical practice
any implementation criteria agreed between NICE and the Department of Health and Social Care
the timing and potential impact of research in progress (for example, new randomised controlled trials [RCTs]).
The committee considers the comments and, if appropriate, amends its recommendations. The committee discussion section is modified to reflect any issues that have arisen from consultation.
The committee considers advice from NICE on the appropriate approach to making scientific and social value decisions. Advice on social value judgements is informed by the work of the NICE listens, NICE's advisory bodies, and the NICE Board, as well as legislation on human rights, discrimination and equality as reflected in NICE's equality scheme. Principles that describe the social value judgements that should, generally, be considered by the committee are in our principles on the NICE website.
The credibility of the guidance produced by NICE depends on the transparency of the committee's decision-making process. The committee's decisions must be explained clearly with reference to all the available evidence, the contributions of experts, and comments received during consultation. The reasoning for the committee's decision will be explained, with reference to the factors that have been considered, in the committee discussion section of the guidance.
The language and style used in the documents produced by the committee are governed by the following principles:
The need for clarity in explaining how the committee has come to its conclusions. The committee discussion section of the guidance document is particularly important. This summarises the key issues that have been debated and the rationale for the conclusions.
The understanding that the text of the documents does not need to reiterate all the factual information that can be found in the information published alongside the guidance. This needs careful consideration so that enough information and justification is given in the draft guidance document or final guidance to allow the reader to understand what evidence the committee considered and, if appropriate, who provided that evidence.
The committee is not empowered to alter any direction from the Secretary of State for Health and Social Care for guidance with a funding requirement that requires commissioners to make funds available for the implementation of relevant NICE guidance within 3 months of publication. However, the committee may consider circumstances in which this implementation period should be varied and advise NICE accordingly. When appropriate, the committee's consideration is limited to those circumstances in which it is apparent that either the technology cannot be acquired or the NHS will not be in a position to use it within the 3-month period, or both.
The committee does not normally make recommendations on using a technology outside the terms of its regulatory approval. Exceptionally, the Department of Health and Social Care may direct NICE to develop guidance on a technology outside of its regulatory approval.
Evidence relating to the technology being evaluated that is outside the terms of its regulatory approval may be considered during the assessment phase of the evaluation. This may inform the committee's discussions about the use of the technology within the scope.
The committee may consider factors that may provide benefits to the NHS or the population, such as patient convenience. It may also consider costs and other positive or negative impacts on the NHS that may not be captured in the cost analysis, such as improved processes.
Patient access schemes, commercial access agreements, managed access proposals and any related process and documents apply to technology appraisals and highly specialised technologies only, unless specifically stated otherwise. For diagnostics guidance please refer to the interim addendum on access proposals.
The committee is not able to make recommendations on the pricing of technologies to the NHS, but can consider a commercial arrangement or managed access proposal.
The committee must make decisions on the appropriateness and relevance of comparator technologies because this is crucial to considering the clinical and economic evidence.
When selecting the most appropriate comparators, the committee will consider:
established NHS practice in England
the natural history of the condition without suitable treatment
existing NICE guidance
cost effectiveness of the comparator
the licensing or regulatory status of the comparator.
The committee will normally be guided by established practice in the NHS when identifying the appropriate comparators. When the evaluation suggests that an established practice may not be considered a good use of NHS resources relative to another available treatment, the committee will decide whether to include it as an appropriate comparator in the evaluation, after reviewing an incremental economic analysis. The committee's overall decision on whether a cost-ineffective practice is a valid comparator will be guided by whether it is recommended in other NICE guidance, or whether its use is so embedded in clinical practice that this will continue unless it is replaced by a new technology.
The committee can consider as comparators technologies that do not have regulatory approval for the population defined in the scope when they are considered to be part of established clinical practice for the population in the NHS. Long-standing treatments often do not have a company to support the regulatory process. Specifically, when considering an 'off-label', 'unlicensed' or 'unregulated' comparator technology, the committee will take into account the extent and quality of evidence, particularly for safety and efficacy, for the unregulated use.
The committee can consider the full range of clinical studies that have been done and is not expected to restrict itself to considering only certain categories of evidence. This means the committee considers all of the evidence presented to it. This includes RCTs, non-randomised studies and test accuracy studies. It also includes any qualitative evidence related to the experiences of patients, carers and experts who have used the technology being evaluated or are familiar with the relevant condition.
The importance given to these various kinds of evidence depends on both the overall balance and quality of the evidence from different sources, the suitability of a particular type of evidence to address issues under consideration, and the particular evaluation context (including, for example, the type of technology, evaluation or population). In general, greater importance is given to evidence from studies of higher quality with methods designed to minimise bias. NICE expects high-quality evidence to be presented, and will assess it proportionately according to each circumstance, context, and decision problem.
The committee's decisions on clinical effectiveness take account of the following factors:
The nature and quality of the evidence derived from:
the written evidence submissions
the analysis of the external assessment group
the views expressed by the clinical experts and, if relevant, specialist committee members, particularly their experience of the condition and the technology in clinical practice
the experience of the patient experts, carers and specialist lay committee members of living with the condition and using the technology being considered.
Uncertainty generated by the evidence and differences between the evidence submitted for regulatory approval and that relating to effectiveness in clinical practice.
The possible differential benefits or adverse outcomes in different groups of patients.
The impact of benefits and adverse outcomes associated with the technology as seen from the patient's perspective.
The position of the technology in the overall care pathway and the alternatives to the technology that are established in clinical practice.
For highly specialised technologies, the committee will consider the following additional factors in its deliberations around clinical effectiveness:
The overall size of health benefits to patients and, when relevant, carers.
Robustness of the current evidence and the contribution the guidance might make to strengthen it.
Extent of disease morbidity and patient clinical disability with current standard care.
The extent to which these factors are taken into account in making decisions about the clinical-effectiveness evidence is at the committee's discretion.
For technologies evaluated using cost-comparison analysis, conclusions on the similarity of health benefits will be based on a pragmatic view of all available evidence for the technology compared with the relevant comparators. Clinical, technological, biological, or pharmacokinetic evidence can be used to support such a conclusion. Ideally, a non-inferiority or equivalence study with appropriate non-inferiority margins should be presented. Alternative methods, such as meta-analysis and indirect comparisons (including, for example, observational studies with a comparator drawn from the population through a matching-adjusted indirect comparison) may be considered when an RCT was not possible. The methods used to do the analysis must be rigorous and transparent.
In the reference case, the committee will regard all quality-adjusted life years (QALYs) as being of equal weight. However, when considering the overall health benefits, the committee can consider other factors and decision-making modifiers. Also, when relevant and in exceptional circumstances, it can accept analysis that explores a QALY weighting that is different from that of the reference case. Deviating from the reference case and applying modifiers should be morally and ethically supported by reason, coherence, and available evidence.
Decision-making modifiers are factors that have not been included in the estimated QALY because they cannot be (that is, they are factors that go beyond QALYs), and value judgements. Modifiers can be taken into account qualitatively through committee discussion or quantitatively through QALY weighting.
The committee will consider the severity of the condition, defined as the future health lost by people living with the condition with standard care in the NHS (including use of other available treatments, diagnostics, or best supportive care). The extent of unmet health need is reflected within the severity definition.
When assessing the severity of the condition in technology appraisals, the committee will consider the associated absolute and proportional QALY shortfall.
Absolute QALY shortfall is the future health, including quality and length of life, that is lost by people living with a condition, compared with the expected future health without the condition over the remaining lifetime of the patients. Absolute QALY shortfall is calculated as the expected total QALYs that people living with a condition would be expected to have with current treatment over their remaining lifetime subtracted from the total QALYs that the general population with the same age and sex distribution would be expected to have. The expected QALYs for the condition with current treatment is equivalent to the total QALYs gained with established practice in the NHS.
Proportional QALY shortfall represents the proportion of future health, including quality and length of life, that is lost by people living with the condition. Proportional QALY shortfall is calculated by taking the absolute QALY shortfall and dividing it by the remaining QALYs that the general population with the same age and sex distribution would be expected to have over their remaining lifetime.
The committee may apply a greater weight to QALYs if technologies are indicated for conditions with a high degree of severity. The data used to estimate both absolute and proportional QALY shortfall should focus on the specific population for which the new technology will be used and be based on established clinical practice in the NHS.
Absolute and proportional shortfall calculations include an estimate of the total QALYs for the general population with the same age and sex distribution as those with the condition. The population EQ‑5D data and survival data used for the estimates should be based on a recent and robust source. Absolute and proportional shortfall calculations should include discounting at the reference-case rate.
The QALY weightings for severity are applied based on absolute and proportional shortfall, whichever implies the greater severity level. If either the proportional or absolute QALY shortfall calculated falls on the cut-off between severity levels, the higher severity level will apply.
| QALY weight | Proportional QALY shortfall | Absolute QALY shortfall |
|---|---|---|
|
1 |
Less than 0.85 |
Less than 12 |
|
x1.2 |
0.85 to 0.95 |
12 to 18 |
|
x1.7 |
At least 0.95 |
At least 18 |
For medical technologies evaluated through the medical technologies evaluation programme, the concept of a quantitative QALY weight is not applicable. The severity of the condition should be considered deliberatively within decision making.
For diagnostics, a QALY weight for severity based on absolute and proportional QALY shortfall is unlikely to reflect the societal value and severity of disease in a way that is relevant to the diagnostics context. Therefore, the severity modifier will not normally be applicable in diagnostic evaluations.
For highly specialised technologies, the severity of the condition is already implicitly captured in the selection of technologies for evaluations. No additional QALY weighting for the severity of disease is applied.
Technologies recommended after applying the severity modifier will be considered as relevant comparators for future evaluations of new technologies introduced for the same condition. They must have been recommended for routine use and represent established practice in the NHS at the time of evaluating the new technology. Second and subsequent extensions to the regulatory approval for the same technology will be considered on their individual merits.
For highly specialised technologies, the committee will consider the size of the incremental QALY gain in relation to the additional weight that would need to be assigned to the QALY benefits for the cost effectiveness of the technology to fall within the highly specialised technologies £100,000 cost per QALY level.
For this weight to be applied, there will need to be compelling evidence that the treatment offers significant QALY gains. Depending on the number of QALYs gained over the lifetime of patients, when comparing the new technology with its relevant comparator(s), the committee will apply a weight between 1 and 3, using equal increments, for a range between 10 and 30 QALYs gained.
The weighting is applied as described in table 6.2 below.
| Incremental QALYs gained (per patient using lifetime horizon) | Weight |
|---|---|
|
Less than or equal to 10 |
1 |
|
11 to 29 |
Between 1 and 3 (using equal increments) |
|
Greater than or equal to 30 |
3 |
NICE considers the overall resources available to the NHS when determining value for money. Therefore, decisions about a new technology must consider implications for healthcare programmes for other patient groups that may be displaced by the adoption of the new technology; the opportunity cost, including those programmes or technologies not evaluated by NICE.
As far as possible, the committee will make sure that its decisions about what constitutes good value for money are consistently applied between evaluations.
The committee's decisions on cost effectiveness or cost savings are influenced by the following factors:
The strength of the supporting clinical-effectiveness evidence.
The robustness and appropriateness of the structure of the economic models. In particular, the committee considers carefully whether the model reflects the decision problem at hand and the uncertainties around the assumptions on which the model structure is based.
The position in the care pathway.
The plausibility of the inputs, and the assumptions made, in the economic models.
The committee's preferred modelling approach, taking into account all of the economic evidence available.
The range and plausibility of the incremental cost-effectiveness ratios (ICERs), net health benefits (if appropriate) or cost savings generated by the models reviewed.
The likelihood of decision error and its consequences.
The committee will consider carefully which individuals benefit most from the technology and whether there are subgroups of individuals for whom the effectiveness evidence suggests differential cost effectiveness or cost savings. The committee may recommend a technology for subgroups of the population only if there is clear evidence that the characteristics defining the subgroup influence the effectiveness or value for money of the technology. It can only do this based on an appropriate consideration of subgroups, to make sure that the decision is clinically justifiable, methodologically robust, ethical, and lawful under equalities legislation. The committee should be particularly aware of the benefits and harms (to individuals and to the NHS as a whole) of including or excluding a given subgroup. If considering excluding a subgroup, the committee must be convinced the harm to the NHS of including it is great enough to justify this decision. If appropriate, the committee may decide to not recommend a technology in a particular subgroup (that is, to exclude a subgroup from the recommendation), even if the technology is clinically and cost effective in the whole population, if they consider it appropriate. When considering subgroups, the committee pays particular attention to its legal obligations with respect to legislation on human rights, discrimination and equality when considering subgroups.
When the evidence on key parameters used to estimate cost effectiveness or cost savings has serious limitations, or when a variety of assumptions have been necessary in the economic modelling, the additional uncertainty this creates is a key factor in the committee's decisions.
The committee should consider the reliability and generalisability of the evidence presented when considering cost-effectiveness estimates. In its consideration, the committee will decide whether to recommend or not recommend a technology based on both the evidence presented and the impact of the evidence on key decision uncertainties. When the evidence is highly uncertain and leads to a high degree of decision uncertainty, the committee may consider making recommendations that include managed access, data collection or research (see section 6.4).
The committee considers how its advice may allow more efficient use of available healthcare resources. In general, it will want to be increasingly certain of the cost effectiveness or cost savings of a technology as the impact of the adoption of the technology on NHS resources increases. Therefore, the committee may need more robust evidence on the effectiveness and cost effectiveness or cost savings of technologies that are expected to have a large impact on NHS resources.
When considering uncertainty, the committee should take into account the likelihood of decision error and its consequences for patients and the NHS. There should be an explicit reference to the potential benefits and risks to patients based on the level of decision uncertainty and whether this can or cannot be mitigated. The committee should also consider the risks to the NHS of using the technology, based on the most plausible ICER and the impact of adopting the technology on NHS resources.
Decisions about the acceptability of the technology as an effective use of NHS resources will specifically take account of the degree of certainty around the value for money. In particular, the committee will normally be more cautious about recommending a technology if they are less certain about the evidence presented. However, the committee will be mindful that there are certain technologies or populations for which evidence generation is particularly difficult because they are:
rare diseases
for use in a population that is predominantly children (under 18 years old)
innovative and complex technologies.
In these specific circumstances, the committee may be able to make recommendations accepting a higher degree of uncertainty. The committee will consider how the nature of the condition or technology(s) affects the ability to generate high-quality evidence before applying greater flexibility.
In all cases, the committees must consider the nature, scale and consequences of the decision uncertainty and the risks to patients and the NHS. It should be cautious in accepting a higher degree of uncertainty in circumstances when the highest standard of evidence generation that should be expected in the circumstances has not been achieved. Uncertainty will be considered proportionately for the evaluation context (including, for example, the type of technology, evaluation, or population).
In general, the committee uses the most plausible ICER or most plausible level of cost savings as the primary consideration when making decisions about the acceptability of technologies as a cost-effective use of NHS resources. However, its overall conclusions are also affected by the following additional considerations:
If its decisions have a bearing on broader social considerations and the extent that these are covered by principles on social value judgements in our principles on the NICE website.
If there are strong reasons to suggest that the health benefits of the technology have been inadequately captured and may therefore misrepresent the health utility gained.
If a substantial proportion of the costs (savings) or benefits are incurred outside of the NHS and personal and social services, or are associated with significant benefits other than health, only when requested specifically by the Department of Health and Social Care as part of the remit.
The committee does not use a precise maximum acceptable ICER above which a technology would automatically be defined as not cost effective or below which it would. Given the fixed budget of the NHS, the appropriate maximum acceptable ICER to be considered is that of the opportunity cost of programmes displaced by new, more costly technologies. NICE does not have complete information about the costs and QALYs from all competing healthcare programmes to define a precise maximum acceptable ICER. However, NICE considers that it is most appropriate to use a range as described in sections 6.3.4 to 6.3.8. Also, consideration of the cost effectiveness of a technology is necessary but is not the only basis for decision making. Consequently, NICE considers technologies in relation to this range of maximum acceptable ICERs, so that the influence of other factors on the decision to recommend a technology is greater when the ICER is closer to the top of the range.
To be transparent in decision making, when applying decision-making modifiers, net health benefits should be routinely presented to show the effect on opportunity costs of recommending a technology that meets specific decision-making modifiers. Net health benefits should be presented using values placed on a QALY gain of £20,000 and £30,000, both with and without the QALY weighting applied. Positive net health benefits mean that overall population health is increased because of the new technology. Negative net health benefits mean that the health benefits associated with the new technology are not large enough to prevent overall health loss because of healthcare not being funded elsewhere in the system. Technologies associated with negative unweighted net health benefits may still be recommended when decision-making modifiers have been applied. This is because there is an ethical and moral rationale to value the health benefits gained with these technologies more than those gained by technologies not meeting decision-making modifiers.
When multiple technologies are being compared, cost-effectiveness rankings may be used to present the results of probabilistic model analyses. This should show the probability that each technology is ranked highest (produces the highest net benefit). It may also help to know the probability that each technology is ranked second, last, and all positions in between. Ranking-based histograms ('rankograms') may be used to present this information in a simple way, alongside the expected net benefit of each technology.
Below a most plausible ICER of £20,000 per QALY gained, or £100,000 per QALY gained for highly specialised technologies, the decision to recommend a technology is normally based on the cost-effectiveness estimate and the acceptability of a technology as an effective use of NHS resources. When the estimated ICERs are less than £20,000 per QALY gained, or £100,000 per QALY gained for highly specialised technologies, and the committee decides that the technology should not be recommended, the committee will make specific reference to its view on the plausibility of the inputs to the economic modelling, or the certainty around the estimated ICER, or both. This might be affected, for example, by sensitivity analysis or limitations to the generalisability of findings about effectiveness.
Above a most plausible ICER of £20,000 per QALY gained, or £100,000 per QALY gained for highly specialised technologies, decisions about the acceptability of the technology as an effective use of NHS resources will specifically consider the following factors:
The degree of certainty and uncertainty around the ICER.
Aspects that relate to uncaptured benefits and non-health factors.
For highly specialised technologies the committee may give particular consideration to:
The impact of the technology on the overall delivery of the specialised service
Additional staffing and infrastructure requirements, including training and planning for expertise.
As the ICER for a technology increases in the range of £20,000 to £30,000 per QALY gained, the committee's decisions about the acceptability of the technology as an effective use of NHS resources will make explicit reference to the relevant factors listed in section 6.3.5.
Above a most plausible ICER of £30,000 per QALY gained or £100,000 per QALY gained for highly specialised technologies, the committee will need to identify an increasingly stronger case for supporting the technology as an effective use of NHS resources, considering the factors listed in sections 6.3.5 and 6.3.6.
For technologies that provide less health benefit at a lower cost compared with the relevant comparator(s) (that is, that fall in the south-west quadrant of a cost-effectiveness plane), cost-effectiveness considerations should consider the usual cost-effectiveness levels of £20,000 to £30,000 per QALY. Any relevant additional factors and modifiers should also be taken into account.
Recommendations for using a diagnostic test may also be limited to specific circumstances such as: the patient's characteristics, aetiology of the disease, the training and skills of those providing the test, availability of equipment, and the availability of other portions of the care pathway.
A technology may have multiple uses and not all of these may be explored in the evaluation. The committee forms recommendations only for the use of the technology described in the scope.
When a technology has already been purchased, the committee may take into account that its recommendations are made in the context of additional use of existing equipment.
The committee's main considerations when making its decisions are:
Benefit to patients – if the technology has measurable benefit to patients over currently available health and social care system technologies, measured by relevant outcome indicators.
Benefit to the health and social care system – if the technology is likely to reduce costs or resource use (for example staff or facilities) compared with current management.
The committee makes its recommendations based on the clinical and economic evidence, informed by contributions from experts and stakeholders. The committee needs to be confident that the evidence is of sufficient quality, quantity and consistency to make robust recommendations. If there are any uncertainties, the committee makes informed judgements and describes its uncertainties in the guidance. The committee should also consider the degree of severity of the condition when evaluating cost-saving technologies and may take that into account when assessing the level of uncertainty of the evidence presented.
The committee produces recommendations based on the extent to which the potential patient and health and social care system benefits are supported by evidence.
| Case for adoption and potential benefits | Type of recommendations that are normally made | For details see section |
|---|---|---|
|
Case is fully supported. |
Recommended (as an option) |
6.4.2 to 6.4.5 |
|
Case is partially supported – for example, it is supported for specific circumstances or populations. |
Recommended (as an option) in specific circumstances ('optimised recommendation') |
|
|
The case is not currently supported but the technology has the plausible potential to be cost effective and has potential to provide significant patient or healthcare system benefits if the uncertainties in the clinical evidence are addressed. |
Recommended with managed access |
6.4.6 to 6.4.11 |
|
Case is currently not fully supported but the technology has potential to provide significant patient or healthcare system benefits if the uncertainties in the evidence are addressed. |
Recommended with data collection |
6.4.12 to 6.4.15 |
|
Case is not currently supported because the clinical effectiveness or evidence on the impact on other health outcomes is absent or uncertain, but the technology has potential to provide significant patient or healthcare system benefits and a recommendation in a research context is considered appropriate. |
Recommended only in a research context |
6.4.16 to 6.4.17 |
|
Case is not supported. |
Not recommended |
The committee will recommend a technology (as an option) when it considers that there is enough evidence that it provides appropriate benefits and value for money and so should be made available in the NHS.
For technologies evaluated using cost-comparison analysis, the committee usually recommends a technology when it considers that:
there is enough certainty that the technology has at least equivalent clinical or health and social care system benefits compared with current management, and overall uses less resources or
there is enough certainty that the technology has significantly greater clinical or health and social care system benefits compared with established practice in the NHS, and overall uses similar resources.
The committee may recommend the technology only under specific circumstances (sometimes referred to as an 'optimised recommendation'). For example, only for patients with a particular condition who meet specific clinical eligibility criteria, only for a specific subgroup of people, or that the treatment must be given by staff with certain training or in a particular care setting.
When recommending technologies that are one of several similar options, committees may specify what should be taken into account when choosing between them, if it considers this appropriate. These considerations may include cost, if appropriate.
When a committee is unable to recommend a medicine because there is still significant resolvable uncertainty, it can make a recommendation for further evidence to be gathered subject to managed access. The committee can consider a recommendation with managed access when:
the medicine has not been recommended, it has the plausible potential to be cost effective at the currently agreed price, but the evidence is currently too uncertain, and
new evidence that could sufficiently support the case for recommendation is expected from ongoing or planned clinical trials, or could be collected from patients having the medicine in clinical practice, and
these data could feasibly be collected within a reasonable timeframe (up to a maximum of 5 years) without undue burden.
The committee may also make recommendations with managed access for a medicine in specific circumstances only. For example, only for patients with a particular condition who meet specific clinical eligibility criteria, or that the medicine must be given by staff with certain training or in a particular care setting.
A recommendation with managed access is intended to reduce uncertainty about specific evidential issues identified by the committee.
Medicines recommended with managed access are not commissioned routinely by the NHS but are made available to patients for a time-limited period. Patient access is determined by the terms of the managed access agreement between NHS England and NHS Improvement and the company.
A recommendation with managed access is not considered established practice in the NHS because:
the committee has found that a recommendation cannot be supported, until further evidence is available
the committee has made a recommendation with managed access using a temporary price to mitigate the uncertainty
the funding during the evidence generation period for these medicines is made available from dedicated managed access funds, rather than routine NHS funding
if, once further evidence is available, the committee does not recommend the medicine, the medicine will then not be available in the NHS for people who have not yet had treatment
although there is plausible potential to satisfy the criteria for a recommendation, the uncertainty in the clinical data (and consequently the cost-effectiveness estimates) was too great to make such a recommendation at the time of the evaluation.
A recommendation with managed access is distinct from a recommendation only in a research context because managed access is designed to allow further evidence generation in the NHS, in addition to current or ongoing clinical research. A recommendation only in a research context, however, comes without a requirement for NHS funding.
For devices and diagnostics, when a committee is unable to recommend a technology because further research or data collection is needed, the committee may make recommendations with data collection if:
the technology could provide substantial benefits to patients or may release significant resources but
there is substantial uncertainty about whether the potential clinical and system benefits are realisable in clinical settings in the NHS; uncertainties may relate to whether clinical outcomes will be achieved, or to service impact (for example, the likelihood of the technology being introduced in a way that leads to the claimed benefit of released resources).
The committee considers the following factors when deciding whether to make a recommendation with data collection:
the most important clinical, economic, technical or other evidence gaps relating to use of the technology in the health and social care system
the research questions that future studies could address
the likely net benefits for all NHS patients of only using the technology in research during the time that the recommended research is being done
irrecoverable costs incurred from introducing the technology
the likely costs and benefits of the research (to ensure that a research recommendation does not become a barrier to innovation).
Research recommendations may focus on the care pathway after the use of a diagnostic test if uncertainties about the pathway affect the value of testing.
A recommendation with future evidence generation and data collection is not intended to prevent the use of the technology in the health and social care system but to identify further evidence which, after evaluation, could support a wider recommendation. Evaluations that include a recommendation with data collection will normally be reviewed at an appropriate time, to reconsider the evidence and value of the technology.
When the evidence of clinical effectiveness or impact of a technology on other health outcomes is either absent, weak or uncertain, the committee may recommend that the technology is used only in the context of research. Before issuing such recommendations, the committee will consider the following points:
The need for and potential value to the NHS of additional evidence that can inform the development of NICE guidance and clinical practice on the use of the technology.
The uncertainty in the analysis and what could be gained by reconsidering the decision in the light of research findings.
The impact of recommendations on the feasibility of doing the research.
Information about ongoing or planned relevant research, or the likelihood that the research needed will be commissioned and successfully reported.
The time it is likely to take for research findings to be available to inform subsequent NICE guidance and clinical practice.
Ethical or practical aspects of doing further research.
In considering these factors the committee may seek advice from research commissioners, the wider research and clinical communities and stakeholders. The committee will consider these factors to balance the potential net benefits to current patients in the NHS of a recommendation not restricted to research with the potential net benefits to both current and future patients in the NHS of producing guidance and basing clinical practice on a more secure evidence base.
A recommendation only in a research context is not considered established practice in the NHS.
If the benefits and value for money delivered by a technology are not supported by the evidence and are not likely to be realised in practice even if further evidence was generated, the technology is not recommended. The committee's rationale is described in the committee discussion section of the guidance.