NICE Draft Programme Manual – what’s missing?

NICE’s 187-page draft programme manual, published today for consultation, has more than enough by way of reform to HTA methods and processes for any market access aficionado to get their teeth into. However, perhaps these proposed developments (heralded by NICE as evolution rather than revolution in an attempt to manage expectations) will live and die by the conspicuous absence of reform in a number of key areas. Here, I take a look at some of the gaps in the draft programme manual, and the potential impact that these omissions might have for commercialisation of medicines in the UK in future.

First, in terms of changes to methodology, few would be surprised that the reference case discount rate (which was off the table, then on the table, then potentially back off again) is firmly out of scope, despite the fact that NICE maintains its ‘view that there is a case for changing the reference-case discount rate to 1.5% for costs and health effects’. Disappointingly, the suggestion that any change to the discount rate would impact the Voluntary Scheme for Branded Medicines Pricing and Access that runs to the end of 2023, suggests that revision of the rate may fall into scope of the renegotiation of VPAS, with any change effective from 2024. Of course, the non-reference discount rate is still an option for some, but the challenges around its implementation have been made clear in recent months, including for potentially curative ‘one-time’ treatments.

A second notable absence from the methods section of the consultation is around a modifier for health inequalities. Given the recent focus on this across the health system, prompted by the Covid pandemic and the Black Lives Matter movement, some may be disappointed that NICE has failed to deliver on this issue. However, there is a recognition of the importance of reducing health inequalities, and a commitment to introduce a modifier, likely in a qualitative way, to the future methods guide. This will be one to watch over the coming months.

Turning to processes, many of the big-ticket items which would have most significantly changed NICE’s standard TA processes are mentioned, but no substantive changes on these are set out for consultation. This applies to the ‘straight-to-managed-access’ option that garnered significant support from stakeholders during the last round of consultation. Despite the absence of detail on this particular option, there are provisions in the draft programme manual to allow routine recommendations for topics with managed access agreements. This may support access in circumstances where there is resolvable clinical uncertainty.

Other novel process options, which NICE has omitted, include an expedited appraisal route for technologies which gain rapid regulatory approval and an alternative process for technologies with multiple indications. On the former, this is potentially one of the greatest challenges facing NICE, as it is being asked to speed up appraisal for therapies which may have a less mature evidence base (and therefore greater inherent uncertainty). One wonders if there comes a point at which NICE will simply be unable to make a recommendation on the basis that the uncertainty is too great. Perhaps the answer would be a straight-to-managed-access option while additional data is gathered.

On the question about a light-touch appraisal process for technologies for multiple indications, this is an area with the potential to significantly reduce NICE’s work programme and alleviate capacity challenges, if got right.  It may be in NICE’s interest to work up and consult on a draft process in the short term, as we are likely to see a large number of therapies with over a dozen indications coming down the line in future.

NICE has committed to exploring all of these gaps in the current consultation in future modular updates to the programme manual (with the exception of discounting). Therefore, one hopes we will see additional consultations in the next few months to enable the realisation of the aims of the methods and process review (which, lest we forget, includes ‘promoting innovation and supporting the life sciences industry’). The proof of the pudding will be in the eating.