Co-authored with Berkeley Greenwood, Senior Partner, Newmarket Strategy

NICE is implementing changes to its technology appraisal processes in what is described as a bid to increase its capacity and output, while also enabling faster patient access, and simplifying the process for industry. Several process changes are being grouped under a so-called ‘proportionate approach’ umbrella.

Following a webinar in March, NICE published its ‘Interim methods and process guide for the proportionate approach to technology appraisals’ at the end of April. These provide us with insight into the implications of these changes but for manufacturers of pharmaceuticals in England, several questions remain unanswered.

Cost comparison

The cost comparison route has been an option to manufacturers since 2017 (initially as part of the fast-track appraisal process, now removed) in cases where the new intervention offers similar or greater health benefits compared to existing treatments, while costing the same or less. To date, the choice to submit a cost comparison analysis, rather than a cost-utility analysis has largely been that of the manufacturer, although NICE have in some cases disagreed that the evidence supports comparable efficacy and have requested a cost-utility analysis.

Going forward, stakeholders will be asked questions at scoping consultation to establish whether there is a case for cost comparison, including questions intended to establish the intervention’s clinical similarity to established and approved comparators. A briefing report will be published alongside topic information on the NICE website commenting on the appropriateness of cost comparison. Following stakeholder engagement, a final decision will be made by NICE, and the manufacturer will be invited to either make a cost comparison submission or to submit to a single technology appraisal. Critically, submissions made using the standard single technology appraisal template after cost comparison has been referred will be rejected and the topic may be delayed.

It is currently unclear how NICE will determine whether a new intervention is ‘clinically similar’ to the chosen comparator and, indeed, how the comparator will be selected. Insisting on statistical superiority, for example, could result in an undervaluation of new interventions, especially in the case of indirect treatment comparisons where credible intervals will inherently be larger. While we anticipate that the submitting company’s ability to sway the decision may be limited, manufacturers would be well-advised to make their opposition known.

Streamlined decision making

For single technology appraisals, the review and decision-making process will only become apparent post-submission, upon receipt of the external assessment report. At this point it will be determined whether or not there is a need for technical engagement, and whether or not a full committee is required. As such, there is no change in the process of preparing for an STA submission, but treatments could potentially be approved quicker post-submission.

Notably absent

The interim methods make no reference to the previously proposed ‘pathway appraisal’ approach, piloted in renal cell carcinoma and lung cancer. Further, no additional insight is currently given into the previously hinted-at changes to managed access. The front-loading approach, piloted in breast cancer, is known to have been dropped considering limited realised efficiency gains.

Implications for manufacturers

The interim methods appear to indicate an increased imperative for compelling evidence of treatment benefit for the English market to mitigate the risk of being benchmarked against low-cost comparators. In many cases, manufacturers will need to be prepared to submit via either the cost comparison or the STA route, with implications in terms of planning and evidence generation. At LCP and Newmarket Strategy, we will be keeping a close eye on developments in NICE’s methods and processes to enable us to support our clients in navigating the changing HTA landscape in the UK.