PATT 2.0: what are NICE’s priorities and what does this mean for the pharmaceutical industry?

Introduction

In 2022, NICE began introducing the concept of a “proportionate approach to technology appraisals” (PATT). The idea was to simplify, remove or reconfigure aspects of the technology appraisal process to ultimately speed up patient access and increase NICE capacity.

Phase 1, which launched in 2023, included:

• Removal of technical engagement as part of a standard appraisal
• Launch of a new cost comparison approach
• Introduction of streamlined decision-making (i.e., decision-making without the need for a full committee meeting in areas of low risk and highly cost-effective products)
• Paired appraisals for products undergoing appraisals in the same indication and along parallel timelines.

A full explanation of Phase 1 can be found in Remap Consulting’s webinar here.

In Phase 2 of PATT, NICE is focusing on 4 main areas:

1. A pathways approach to technology appraisals (which began exploration in Phase 1)
2. High value steps
3. Products with many indications
4. Rapid entry to managed access (REMA).

Within this article, we will explore each of the aspects of PATT 2.0, what we know so far and what potential benefits and challenges they may bring.

PATT 2.0

Pathways Approach

The pathways approach to technology appraisals is a major part of PATT 2.0 that could result in a significant departure from current processes. Arising from NICE’s recognition that nearly half of technology appraisals take place only in 10 disease areas (including multiple myeloma, non-small cell lung cancer (NSCLC) and renal cell carcinoma (RCC)), the pathways approach is exploring the use of a single disease economic model for each of these respective areas.¹

NICE suggests that the approach will increase consistency, reduce repetition, and make guidance easier to understand. The first pathways approach pilot took place in RCC and involved the following key steps:

• An extensive scoping activity between NICE, external assessment groups (EAGs) and relevant stakeholders (companies, clinical and patient representatives) to outline the disease pathway
• Analysis and development of a single-disease model by an EAG using publicly available information
• A consultation process during which companies with new technologies being involved and comparator companies could submit evidence and comments
• Committee consideration of the evidence and model.²

It is important to recognise that the pathways approach is currently only in a pilot phase and there is no current requirement for companies to be involved or routed to this process. However, following pilots, if NICE chooses to adopt the pathways approach into its methods and processes, company’s undergoing this approach will have significantly less control and input into the appraisal process. The Association of British Pharmaceutical Industry (ABPI) has raised several concerns noting that:

• A single disease model developed by NICE “may not be feasible or desirable in practice”
• ABPI members have suggested that the ongoing pilots do not meet NICE’s justification of increasing efficiency
• There is potential to create inequity in the way medicines in different disease areas are evaluated.³

From Remap Consulting’s perspective, there are valid concerns around the pathways approach, and it will be highly challenging process to develop an approach that works efficiently and effectively meets the needs of all stakeholders involved. Given this complexity, we consider it unlikely that the pathways approach development work will be fully completed in 2024. Overall, this is an area where we expect several iterations, improvements, and stakeholder engagements before formal incorporation into NICE’s methods. 

High value steps

In this aspect, NICE is reviewing processes with the aim of identifying the most important (or “high value”) steps of evaluations. The aim is to ultimately introduce changes that will allow these steps to be reached more quickly, whilst maintaining NICE’s robustness. To achieve this, NICE has three workstreams:

1. Committee meeting management: where analysis of committee meeting discussions is being conducted to enable improvements that ensure appropriate consideration for each topic
2. Process improvements: focused on looking at NICE’s internal processes to assess where additional efficiencies can be introduced
3. Proportionate reviews: which is exploring how to increase efficiencies and be “more proportionate” in topic reviews, including reviews of biosimilars and generics.¹

The common theme on the three workstreams is increased efficiency, something that has been a recurring priority for NICE in recent years. Remap Consulting do not expect that companies will see an immediate noticeable impact through the work on process improvements, however tangible impacts could potentially be seen if changes are made to the way committee meetings are managed.

Products with many indications

Multi-indication products have become increasingly prevalent, particularly within the immuno-oncology space. In fact, a search of NICE guidance by Remap Consulting results in finding 25 and 31 appraisals involving nivolumab and pembrolizumab, respectively. Through PATT 2.0, NICE is exploring whether alternative methods and processes can be used to evaluate products such as these.¹ Whilst implementing alternative processes could increase efficiencies, we expect that implementing these changes will be challenging. New indications will always have their own technical issues and there is a question on whether introducing alternative processes for products with multiple indications could disadvantage newer innovative products which do not have existing indications.

REMA

In this final area, NICE is collaborating with NHS England to explore the potential for promising new technologies to be recommended for use with a managed entry agreement, without needing a full upfront assessment.¹

REMA has been identified by the ABPI as a “priority” given its potential to allow managed access without the current resource-intensive process of needing two-full evaluations (i.e., prior to and upon exit of a managed access period). From the ABPI’s perspective, REMA will need a move away from the requirement of demonstrating “plausible cost-effectiveness” prior to a recommendation for managed access.³ However, NICE and NHS England have clearly stated that eligibility for REMA will require the calculation of an incremental cost-effectiveness ratio (ICER).

From Remap Consulting’s perspective, it is likely that NICE will still require plausible cost-effectiveness prior to inclusion in REMA, however it is possible that the range considered “plausible” for cost-effectiveness could potentially have greater flexibility versus current processes. Regardless of a threshold requirement, the removal of the need of a full evaluation prior to managed entry has the potential to increase the attractiveness of the UK as an early launch market.

Conclusion

Overall, like Phase 1, PATT 2.0 is aiming to introduce efficiencies, increase NICE’s capacity and enable faster patient access. However, whilst Phase 1 arguably enabled launch of the “quick wins”, the focus of PATT 2.0 is in significantly more complex areas. This is particularly true of the work being conducted on REMA and the pathways approach. Both workstreams initially began development in Phase 1 of PATT and their continued exploration is a marker of the significant change both could represent.

REMA is being welcomed by the ABPI and represents an opportunity for early patient access in the UK without the need for a full evaluation. However, there are still several remaining questions on how this will be implemented, including how an ICER will be agreed on without an evaluation.

The pathways approach is, unsurprisingly, being considered with much greater caution by the pharmaceutical industry. If implemented, companies in relevant disease areas will be tasked with using models developed by EAGs. Whilst company resource requirements will be reduced, the significantly reduced level of input into the NICE evaluation and control over the model, will leave companies uncomfortable.

Overall, there is important work remaining on each of these areas and it is unlikely that processes will be finalised early in 2024. Nevertheless, whilst these may not reflect an immediate upcoming change, considering the significant impact they could have on future NICE evaluations, we encourage companies to take part in NICE consultations on changes to ensure that comments can help shape development prior to formal inclusion into methods and processes.

Have you watched our webinar on NICE’s Proportionate Approach to Technology Appraisals for Phase 1? Check it out here.

Sources:

1.  NICE (2024). Taking a proportionate approach to technology appraisals. Available at: https://www.nice.org.uk/about/what-we-do/proportionate-approach-to-technology-appraisals
2.  NICE (2023). Pathways approach. Process statement (renal cell carcinoma). Available at: https://www.nice.org.uk/guidance/gid-ta11186/documents/statement-2
3.  ABPI (2023). NICE proportionate approach to technology appraisals. Available at: https://www.abpi.org.uk/media/kktgeaov/nice-proportionate-approach-to-technology-appraisals-18102023.pdf