NICE’s ‘Proportionate Approach to Technology Appraisals (PATT)’: The Story So Far

Cost-Comparison Appraisals

What is the cost-comparison appraisal route?

• To improve efficiency in the route to reimbursement for next-in-class or similar medicines, NICE have further streamlined the cost-comparison route (introduced in January 2022 to replace the fast-track appraisal [FTA] process). The cost-comparison route is suitable for technologies associated with similar or greater health benefits, at similar or lower costs, than technologies already recommended by NICE for the same indication.
• Time savings highlighted in the interim methods and process guide include:
  • A proportionate review of the evidence package by an external assessment group (EAG)
  • A reimbursement decision made outside of a full committee meeting (sub-committee sign-off)
    • If the subset of the committee cannot make a recommendation, a full committee meeting will be required²
• The interim methods and process guide states that a technology will be selected for the cost-comparison route at the scoping meeting stage (following discussion on the population, treatment pathway, benefit and clinical similarity to existing treatments in UK clinical practice)

What impact have we seen?

So far, our experience of supporting submissions that have undergone the cost-comparison route has been broadly positive, resulting in significant time savings in the route to reimbursement compared to both the prior FTA and STA processes. These findings were corroborated by the results of NICE’s pilot learnings on the proportionate approach, presented at their March 2023 webinar, which found that the cost-comparison route reduced the appraisal process to 23 weeks (compared to 34 weeks and 44 weeks for the former FTA process and the existing STA process, respectively).³

What challenges and opportunities have we faced?

Whilst these findings are positive, technologies suitable for a cost comparison could be further differentiated on the basis of risk at the scoping stage. Further streamlining this process could result in faster reimbursement for novel medicines, free up NICE’s capacity to focus on more complex appraisals and reduce costs to manufacturers:

• Lower-risk cost-comparison cases could be further streamlined: It may be reasonable to accept greater uncertainty around cases of comparable efficacy when a decision is associated with low risk, for example, when the opportunity cost of marginal differences in treatment efficacy is likely to be very low, and an intervention is associated with considerable cost savings. Accordingly, the appraisal process for lower-risk appraisals could be aligned more closely to the corresponding ‘abbreviated submissions’ process at the Scottish Medicines Consortium (SMC).⁴ The SMC abbreviated submission process is stated to take 18 weeks from scheduling to publication. However, in our experience of submitting over 10 abbreviated submissions, the process often takes less than 12 weeks. Our research presented at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 2023, revealed that 71% of appraisals proceeding via the FTA route at NICE also utilised the SMC abbreviated submission process, all of which received positive recommendations from both HTA bodies.⁵
• Processes could be optimised for borderline, higher-risk cases, to ensure correct routing at the outset: The appraisal process could be further optimised for borderline, higher-risk cases, where there is uncertainty around whether an intervention satisfies the criteria for a cost comparison, e.g. where comparable efficacy versus recommended treatments is informed by an indirect treatment comparison (ITC). If the available evidence is associated with too much uncertainty to accept the case for comparable efficacy, the cost-comparison case may be rejected. The interim methods and process guide provides no clear guidance on the process and timelines associated with rejection, and our experience has not been consistent. In particular, the guidelines do not specify whether a new STA submission would be required, and if so, whether this appraisal would be re-scoped, which could result in substantial delays to reimbursement timelines. Further guidance on the evidence required to fulfil the eligibility criteria for cost-comparison appraisals, and additional clarity on the processes and timelines involved in cases of rejection, would encourage earlier routing of appraisals to the STA process where evidence is too uncertain to accept the criteria for a cost comparison.
• Guidance and transparency surrounding cost comparison scoping could be improved: There is a lack of clarity around the definition of the decision problem for a cost comparison appraisal. The user guide for the FTA cost comparison company evidence submission published in February 2018 notes that it “is acceptable to make a cost-comparison case with only 1 of the comparators in the scope, provided that the selected comparator satisfies all of the following criteria: 1) it adequately represents the NICE-recommended treatments as a whole, both in terms of its cost and effects, 2) it has a significant market share and 3) it is recommended in published NICE technology appraisal guidance for the same indication.”⁶ No such wording is included in the user guide for STA cost comparison company evidence submission template published in January 2022. Our experience of recent cost-comparison appraisals is that selection of a single or subset of comparators from those listed within the NICE scope can still be accepted in certain circumstances, but this appears to be inconsistent across appraisals without clear rationale. For example, we have seen EAGs conclude that all treatments listed in the NICE scope should be included in the cost comparison if they represent relevant options in clinical practice, but in other cases EAGs have accepted restriction to a single comparator despite many treatments being available and used in practice.

Other Proportionate Approaches

What other ‘streamlined approaches’ are available?

• NICE have expanded the streamlined approaches already used for cost-comparison appraisals to include a wider group of medicines. Potential streamlining approaches for appraisals not satisfying the cost-comparison criteria include:
  • Streamlined decision-making: Reimbursement decisions made outside of a full committee meeting (sub-committee sign-off)
  • Optional technical engagement: Removal of the 4-week technical engagement step
  • Paired appraisals: Joint committee meetings for appraisals in the same disease area following similar regulatory timelines
• Unlike the cost-comparison route (where a routing decision is made at scoping), the interim methods and process guide specifies that appraisals may only be re-routed down the streamlined route following full review of the evidence package by the EAG.²

What impact have we seen?

Current timelines for appraisals proceeding via the standard STA route are lengthy, taking a minimum of 44 weeks. We therefore welcome that NICE are investigating ways to streamline the decision-making process for all medicines, not just those satisfying the cost-comparison criteria. Overall, our experience of proportionate approaches outside of the cost-comparison route has been positive, with a reduction in the time to reimbursement versus the standard STA process. In particular, removal of the 4-week technical engagement step has the potential to make the post-submission process less resource-intensive for both manufacturers and NICE, and reduce the time to reimbursement accordingly.

What challenges and opportunities have we faced?

• Criteria for other streamlined approaches are not well defined: The criteria appraisals must satisfy to be eligible for the changes to topic progression under the proportionate approach remain unclear. In particular, as part of the consultation on the interim methods and process guide, we requested that further clarity be provided on which appraisals may satisfy the requirements for ‘streamlined’ decision-making. Our experience of the cost-comparison process suggests that providing clear requirements with an accompanying incentive of faster routes to access may encourage manufacturers to offer price flexibility to mitigate uncertainty. Manufacturers may opt to present a cost comparison even in cases where available evidence suggests the intervention has additional benefits over existing treatments, but where such benefits are uncertain or difficult to quantify and thus where a price premium would likely require comprehensive evaluation. Similarly, manufacturers may be willing to accept more conservative assumptions around intervention benefit to gain faster access through ‘streamlined’ decision-making.
• Paired appraisals may be at risk of key issue ‘creep’: Consultees raised concerns around the impact of the paired appraisal process, highlighting that scheduling of two appraisals to align may cause delays to individual appraisal timelines. To this extent, in response to public consultation, NICE have updated the manual to be clear that no topic would ever be delayed, or timelines shifted, to allow alignment to take place. However, beyond timelines, there remains a risk of bias, in which key issues identified may not be common across appraisals, and which may hinder efficient and productive discussion about individual technologies.