What is the ‘proportionate approach’?
NICE are trialling a new approach to increase the efficiency with which technology appraisals are delivered and to ensure their capacity meets their commitment to appraising all new medicines. NICE have been going through a ‘test and learn’ process over the past 12 months and have conducted several pilots exploring different approaches to evaluation.
The ‘proportionate approach’ aims to differentiate the NICE appraisal process and provide tailored routes to access for certain new treatments. NICE’s goal is to allow for more rapid and efficient evaluations (particularly for appraisals considered low-risk), and to ensure more complex appraisals are associated with an appropriate level of engagement. Currently, five treatments have been recommended through the proportionate approach, with multiple additional pilots ongoing.
NICE published the 2022-23 final report as well as the interim methods and process guide for the proportionate approach to technology appraisals in April 2023. Interim implementation of these proportionate approaches followed by formal modular updates to the NICE methods manual are expected throughout 2023 and 2024.
What is being considered:
A simplified appraisal and decision-making process for cost comparison appraisals (somatrogon [TA863]) and appraisals considered low-risk (nintedanib [TA864]), with the aim of reducing the appraisal timeline.
The improvement of operational efficiencies though the proportionate use of technical engagement, streamlined confidential marking and the introduction of paired appraisals for topics in the same disease area with similar timelines (empagliflozin [ID3945] and dapagliflozin [ID1648]).
A single, ongoing appraisal spanning several medicines across an entire disease pathway with a centralised economic model providing a more dynamic approach to decision making (renal cell carcinoma pathways pilot [ID6186]).
Rapid entry to managed access
The development of a streamlined decision making framework for treatments highly likely to require a Managed Access Agreement, reducing the resource required to identify and appraise these submissions in the first instance.
Front-loading (no longer live)
The use of pre-specified assumptions based on committee preferences from previous appraisals to increase decision making efficiency. A retrospective pilot across three breast cancer appraisals in 2022 demonstrated it was not appropriate to pre-specify assumptions, as solutions to issues of uncertainty were not routinely generalisable. NICE have therefore stated they will not proceed with this workstream beyond the pilot.
Why is this important?
When correctly implemented, the proportionate approach may provide more rapid access to new health technologies. NICE’s streamlined approach to technology appraisals has been able to shorten the appraisal timeline by up to 45%. However, the risk is that if a treatment is incorrectly routed through the proportionate approach, a negative initial recommendation could risk a delay in patient access through the need for an appeal or re-evaluation.
Despite the introduction of paired appraisals offering the potential for streamlined decision making, this approach may also risk the introduction of bias. Decision makers may review data comparatively between the two new technologies, without formal inclusion within the individual decision problems and challenges identified in one appraisal may be carried into another in the absence of evidence to the contrary.
For the pathways appraisals approach, the development of a complex, centralised model built to assess multiple technologies across a whole disease pathway could result in a lack of transparency and flexibility, prolonging the appraisal process.
Ultimately, the approaches detailed above have the potential to improve decision making and accelerate access. For this to happen, manufacturers must have an advanced understanding of their value case and economic positioning earlier on in the scoping process. This will require a greater upfront investment of both time and resources in order to fully understand the benefits and risks associated with the proportionate approach.