Value & Access

Public Consultation on NICE Integrated Topic Prioritisation and Strategic Principles: What’s Set To Change?

On the 5^th March 2024, the National Institute for Health and Care Excellence (NICE) shared their draft methods and process manual for integrated topic prioritisation, and we were delighted to participate in the public consultation. We have provided a top-line summary of the proposed changes and our key takeaways.

NICE’s transformation

Whilst NICE remain committed to their fundamental principles of independence, transparency and rigour, NICE also acknowledge the need for evolution due to the rapidly changing health and care system and an ever-increasing need for NICE guidance. As such, NICE are introducing changes to focus on providing the most relevant guidance to the UK health and care system in a timely manner.

NICE are introducing a new prioritisation and topic selection process, guided by a single prioritisation board, to improve the coordination and development of guidance. Prioritisation decisions will be based on NICE’s strategic principles for public health, social care and rare diseases.

What is NICE’s integrated topic prioritisation manual?

The draft integrated topic prioritisation manual has been designed to replace the topic selection process and methods document.^1,2 The new draft manual details the proposed processes for identifying, prioritising and subsequently routing new topics and updates to existing NICE guidance.¹

Within the manual, NICE proposed a novel prioritisation framework in which eligible topics (as defined by “pre-Stage 1” eligibility criteria) are then assessed against “Stage 1” and “Stage 2” criteria. Stage 1 and Stage 2 assessment will be performed by the newly-introduced NICE prioritisation board – membership of which comprises a number of roles from within NICE, plus two lay members, alongside clinical fellows who hold the role of observers. If topics are not considered to meet the Stage 1 and Stage 2 criteria, they will be deprioritised for routing to one of NICE’s guidance products.

Which types of NICE guidance are covered by the integrated topic prioritisation manual?

The proposed prioritisation processes would apply across topics including guidelines (clinical, social care, public health), health technologies (devices, diagnostics, digital health technologies, interventional procedures), medicines (please see further context below), combined health products (e.g. combination regimens or device-aided medicines) and other topics that are regulated as a medicine or medical device (such as human tissue products).

In some exceptional circumstances, topics that are not usually eligible may be considered on a case-by-case basis.

Are all topics eligible for integrated topic prioritisation?

No – some topics are not routinely eligible for consideration, such as medicines or health technologies that will not receive regulatory approval for use in the UK within 24 and 12 months, respectively. Additionally, established interventional procedures with a well-known efficacy and safety profile, unlicensed or off-label medicines or health technologies and new generic or biosimilar medicines where the branded version is already recommended in NICE guidance will not be eligible for integrated topic prioritisation.

Topics that are outside of NICE’s remit are also not routinely eligible, such as those considered by the Joint Committee on Vaccination and Immunisation or the National Screening Committee.

The filtering out of such topics is described as “pre-Stage 1” in the proposed prioritisation process.

Key Insight

How will medicines be considered in the prioritisation process?

The manual describes that certain topics will be omitted from the prioritisation process. Most notably, all new medicines or significant licence extensions anticipated to receive UK regulatory approval within 24 months of topic selection would be omitted and would go straight to routing, except where there is a clear rationale to apply the prioritisation process (the manual implies this will be an exceptional circumstance). This is because the 2024 Department of Health and Social Care voluntary scheme for branded medicines, pricing, access and growth (VPAG) states that all such topics will be evaluated by NICE. There is some lack of clarity in the manual as to whether both stages of the prioritisation process would be omitted, or Stage 2 only (see description of stages below).

The manual also describes that medicines will be subject to Stage 2 of the prioritisation process where a routing decision is required for technology appraisal or the highly specialised technology (HST) programme. It is not clear from the manual how NICE will identify where such a decision is required. The HST eligibility criteria in Appendix 3 of the consultation for the prioritisation process are presumably designed to be used by the prioritisation board as part of their Stage 2 review to make the final decision. However, it is not clear whether the HST eligibility criteria will also be applied by other at NICE in pre-Stage 1 in order to determine that a new medicine has the potential to be considered for HST and hence needs to be reviewed by the prioritisation board. In general, we think the manual could stand to improve clarity over exactly how new medicines will be handled, though the take-home message is that most new medicines and significant licence extensions will not require review by the prioritisation board and will be routed straight to the technology appraisal programme.

What are the key stages of the integrated topic prioritisation process?

Topics that are considered eligible are assessed using the prioritisation framework Stage 1 criteria, determining if a new topic is appropriate for NICE to address. Only if these criteria are satisfied do topics then proceed to Stage 2, which comprises a more detailed set of criteria used to support NICE’s decision making process.

Stage 1

NICE’s role: what value would be added to the UK health and care system by providing or updating this guidance?
Health and care need: would the guidance address avoidable illness or care burden? Could the guidance avoid premature mortality and morbidity, or reductions in quality of life?
Evidence availability: is evidence available or expected to become available to support the development of the guidance?
Availability and access: is it feasible to implement the intervention or technology in the UK health and care system?

Stage 2

Budget impact: what is the expected cost impact from implementing the guidance?
System impact: what is the potential impact on health and care infrastructure and workforce capacity?
Population impact: what is the size of the relevant population, and would this guidance improve health outcomes for these individuals?
Evidence quality: does the available evidence quality meet NICE’s requirements?
Health inequalities: what is the potential impact on health inequalities across the UK?
Environmental sustainability: will the guidance reduce the use of healthcare services, supporting disinvestment and reinvestment plans?

What happens if a topic is deprioritised?

Deprioritised topics may be revisited at a time when more evidence becomes available, an alternative NICE product may be produced in place of NICE guidance, or cross-references may be made to recommendations produced by other organisations. No further action was also highlighted as a possible outcome of de-prioritisation.

Stakeholders have the opportunity to query NICE prioritisation decisions by submitting questions within 10 working days of the publication of the prioritisation board’s decision; this process will not usually offer an opportunity to revisit or overturn the decision.

What are the potential implications of the new approach?

As part of our participation in the public consultation, we have appraised the integrated topic prioritisation manual. We have a number of comments and questions regarding potential implications of the new framework, including:

Consideration of new criteria in decision making: Formal consideration of health inequalities and environmental impacts represents a positive evolution in NICE’s decision-making process. Our consultation response called for more information on how these factors will be appraised (in particular, whether any quantitative analysis will be used or whether this will be based on subjective judgement of members of the prioritisation board). It will be interesting to monitor the outcomes of the pilot phase of the prioritisation framework and determine the impact of these criteria on overall decision-making.
Exclusion of generic or biosimilar products: The manual excludes generic or biosimilar products from the topic prioritisation process. In some cases, the availability of a generic or biosimilar product may present a case for expansion of a recommendation made for the original branded medicine into the full product licensed indication, for example where the recommendation for the branded medicine was restricted by NICE on the basis of cost-effectiveness. The currently proposed approach does not appear to account for situations like this and hence risks missing opportunities to maximise patient access to effective and cost-effective products where availability of generics or biosimilars can support this.
Potential for deprioritisation of topics with limited evidence bases: NICE’s new framework could be taken to imply that guideline topics or health technologies with limited evidence bases may be deprioritised in place of topics with more robust evidence. We understand the intention – if there is simply insufficient evidence to conduct an evaluation and reach a decision then NICE resources may be best prioritised to topics that can reasonably be evaluated. However, we are concerned that the consultation documents did not sufficiently recognise that expectations regarding availability and quality of evidence cannot be the same across all disease areas. Otherwise, the prioritisation framework risks the deprioritisation of rare diseases and other conditions that are associated with well-known challenges in the collection of robust data, and for which a different context needs to be applied to judgements of available evidence. Such deprioritisation could restrict access to health technologies in populations facing high unmet need.
- For example, the criteria for “sufficient evidence” in the context of the Stage 1 criteria are currently unclear. In our comments on the manual, we have requested that NICE provide further guidance on this, and we have highlighted the importance of inviting input from stakeholders, including manufacturers, on this aspect of the decision-making framework to ensure that natural limitations on evidence availability and likelihood of further evidence generation are taken into account.
Representation of stakeholders in the decision-making process: The NICE prioritisation board includes a high ratio of NICE employees relative to external individuals; there is a risk that decision-making may be lacking in diversity of perspectives and may exclude relevant stakeholders. Furthermore, it is crucial to engage relevant external stakeholders in Stage 1 and 2 of the prioritisation process, for example to provide input on the evidence that is/will be available on a given topic or product. Currently, the draft manual indicates very limited opportunity for stakeholder involvement, which we think runs a real risk of prioritisation decisions being made on incomplete or inaccurate evidence.
De-incentivising infrastructure change: Depending on how they are applied, Stage 2 criteria may risk deprioritisation of topics that will be challenging to implement within the current UK health and care infrastructure. This runs the risk of setting back evolutions of the health system that offer wider system benefits beyond the individual topic; for example, where a new diagnostic technology for a specific disease requires system infrastructure investment to get off the ground but may then have spillover benefits for timely diagnosis of other conditions. Furthermore, rare conditions may also have no established infrastructure or services in place currently, and thus these criteria may limit or delay access to much-needed interventions and services. It is important that these considerations are considered by the NICE prioritisation board in their decision-making.

References

NICE integrated topic prioritisation and strategic principles (In development [GID-PMG10003]). Available here. Last accessed: April 2024.
NICE health technology evaluation topic selection: the manual. Available here. Last accessed: April 2024.

If you would like any further information on the themes presented above, please do not hesitate to contact Alex Porteous, Senior Consultant – HTA and Health Economics (LinkedIn). Alex Porteous is a member of the HTA division and an employee at Costello Medical. The views/opinions expressed are their own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners. For more information on our services, please explore What We Do. For more information about HTA at Costello Medical, please contact Rose Wickstead (LinkedIn).