Claudia Rubin, Director at Decideum, explores a new publication which will inform the National Institute for Health and Care Excellence (NICE) methods review.
So many extraordinary things are taking place in British politics, that it is easy to overlook the regular activity happening out of the spotlight.
Even putting aside the egregious neglect of urgent crises – from social care to the global climate – due to Brexit, the Prime Minister’s outrageous lengthy suspension of Parliament hampers progress with much of its day-to-day business.
“There are opportunities for stakeholders to contribute to the work taking place between now and next summer, not least to insist that the recommendations from the APPG, which reflect the views of a wide and deep range of stakeholders, are given proper consideration”
Pricing, funding and access
It was pleasing therefore, that an important piece of work has been delivered by the cross-party group of MPs who came together a year ago to examine pricing, funding and wider access issues relating to the availability of medicines and medical technologies on the NHS in England. The first publication by the All-Party Parliamentary Group on Access to Medicines and Medical Devices (APPG) led by Anne Marie Morris MP aims to inform the NICE methods review, the topics for which were approved by the NICE Board in mid-July.
The fact that the review is taking place at all is linked to commitments made in the 2019 Voluntary Scheme for Branded Medicines Pricing and Access, and it is these commitments that have strongly influenced the priorities of the review.
NICE, which of course marked its 20-year anniversary this year, has a formidable reputation for its robust appraisals. It can also justifiably claim to respond well to changing times, evolving its programmes and having its remit expanded often. Its core work remains its four health technology evaluation programmes and all of these will be considered within the review, with the expressed intention of ensuring that their methods remain robust and up-to-date and to examine what more NICE can do to ‘better support the uptake of new innovative products in a timely and efficient manner’.
Agreed topics for review
NICE has grouped the agreed topics for the review into three categories, in order of priority:
- Factors that will address the expectations outlined in the 2019 Voluntary Scheme.
- Scientific and methodological innovations affecting the types of products and types of evidence available for those products.
- Other general improvements to methods, including systematisation of committee’s value judgements to support consistent decision-making.
The two main elements of the first category reflect themes that will be very familiar to many within industry, who remain frustrated by the lack of flexibility, particularly in the single technology appraisal process, for demonstrating a product’s benefit in terms outside the standard quality-adjusted life year framework.
Firstly, NICE has, as a priority, undertaken to look at other modifiers that can be considered in decision-making. Many, such as unmet need, burden of illness and disease severity, have been familiar to this narrative for many years. The potential to explore additional factors such as how qualitative judgments can be made in a consistent manner by the committee on NHS policy priorities, wellbeing, experience of care, organisational efficiency, and curative potential, is also included, but unlikely to produce anything robust enough to satisfy NICE’s standards.
The APPG does find a case for the limited use of a modifier, in the case of a therapy meeting an unmet need in a severe condition.
A careful look at the second topic within the top category is also clearly needed, that of how to explore and quantify clinical uncertainty in a consistent manner. An area that has always caused problems for NICE, it is only going to become more of an issue given the medicines landscape, with smaller patient populations and earlier regulatory approval. The recommendation from the APPG is for NICE to replicate a solution that seems to be working well for oncology medicines; introducing an interim conditional approval mechanism, which would work in a similar way to the Cancer Drugs Fund.
Having said that, the APPG is keen to point out that companies must also do their bit to mitigate clinical uncertainty. Given that NICE makes its data expectations clear, some companies could do more to generate data that is fit for a NICE submission.
The second category specifically reflects the need for NICE to keep up to date with advancing science. Reviewing the guidance on genomic technologies, products that address the growing threat from anti-microbial resistance and methods to assess digital technologies are all within the agreed scope. The digital health landscape, expanding daily as artificial intelligence software takes off, is in urgent need of a consistent framework upon which value can be assessed.
The review must also grapple with how to include real world evidence, in particular patient registries, and as it does so, those involved in building those registries have a job to make them as credible – and their data as usable – as possible. Linked to this is the perceived weakness of NICE being unable sufficiently to take account of the patient experience. This comes through clearly from the APPG’s research, which again thoughtfully turns to precedent for a solution; recommending that NICE introduces Patient and Clinician Engagement (PACE) style meetings used by the Scottish Medicines Consortium.
With the scope now agreed, and the governance arrangements finally, if still rather controversially, in place, work is underway to deliver a draft manual for public consultation in summer 2020. There are opportunities for stakeholders to contribute to the work taking place between now and next summer, not least to insist that the recommendations from the APPG, which reflect the views of a wide and deep range of stakeholders, are given proper consideration.
In order to ensure that this happens, and that the review is conducted appropriately, we will turn as ever to our elected representatives. Now, if only we can find them…
Go to www.decideum.com