The UK’s National Institute for Health and Care Excellence (NICE) doesn’t stand still, having seen many changes during its 20-year existence.
With details emerging on the latest review of the methods NICE uses to make cost-effectiveness recommendations, more change could be on the way, but will it be evolution or revolution?
A new review
NICE has always kept its methods and processes under review. NICE has a usual cycle of three yearly reviews which typically include bringing in the great and the good (key are those working at the NICE Decision Support Unit who did a lot of work for the 2013 Methods Guide update) to share their views, as well as using public consultation.
This time around, the NICE review announced on 22 July 2019 is both business as usual, and also one of the commitments made as part of the wider deal struck between the industry and the UK government.
The 2019 Voluntary Scheme for Branded Medicines Pricing and Access (VPAS) included a commitment from NICE to scope and initiate a review of the methods for its Technology Appraisal Programme (TAP) in 2019/20, and indicated that industry and other relevant stakeholders will be active participants in the review.
What is not part of the review – because it’s a done deal as part of VPAS – is the basic cost- effectiveness threshold. After all, one option to get better value would be to lower the threshold, as some companies could drop prices in response to that.
Of course others may decide not to put their technologies through NICE. Its absence continues to put the threshold very much in the hands of government, despite its intrinsic importance to the recommendations that NICE will make.
Behind the scenes
NICE’s announcement of the details of the review includes a short-list of topics that will be considered. This illustrates that there has been a lot of behind the scenes work to set out what the review should include; there aren’t any details on what presumably was the initial longlist that led to the shortlist that NICE has shared.
It does look as though the industry, through the Association of the British Pharmaceutical Industry (ABPI), has got the edge on scoping – with the Working Committee tasked with setting the scope reportedly only inviting representatives from the ABPI.
There is, however, a Steering Group, although it’s unclear exactly who the members are. NICE has said it includes DHSC, NHS England and an independent academic adviser – it’s not clear why NICE hasn’t identified that adviser or stated why he or she was chosen.
It’s also not clear whether the review is picking up on the issues that many will have already identified when they contributed to the All Party Parliamentary Group (APPG) on Access to Medicines and Medical Devices evidence sessions on the NICE methods review inquiry.
Those who gave evidence to the APPG included staff from the Cystic Fibrosis Trust, Takeda, AstraZeneca, EMIG and FIECON, as well as from NICE. The influential academic, Prof Karl Claxton, also gave evidence. Expect to hear more from him on the NICE review.
A broad range of technologies
NICE has said that the review will go beyond Technology Appraisals (TAs) and Highly Specialised Technologies (HSTs) to also cover medical technologies and diagnostics assessment programmes.
This is a very broad scope; it also means that it brings in a diverse range of industry. Even within the pharmaceutical industry there are differences of opinion.
You can only imagine that this is magnified when you bring in such diverse technologies. We can only expect that not everyone will be happy.
It has already raised eyebrows that only the ABPI has been involved in scoping, with some pointing out that the ABPI doesn’t represent all of industry’s views. NICE’s response is that others from industry can be involved in other groups as the review progresses.
What’s in scope: hot topics including cell and gene therapies and HSTs
Andrew Dillon, NICE’s Chief Executive, has confirmed some areas that NICE will be looking at. These include personalised medicine, digitalisation of health, and cell and gene therapies.
The specific issues of technologies, such as treatments developed to target tumours based on genetics and not their position within the body, is also included.
Part of the review will focus on what is included in the HST programme. This is a concern for patients, as well as companies that have drugs that are due to be reviewed under the HST’s wider and more flexible approach, but have not reached that point yet, because although the drugs in question are for rare conditions, those conditions are not deemed to be rare enough.
Presumably cross-cutting, the issue of uncertainty is an area listed by Dillon. Part of exploring uncertainty and how to reduce it will include data analytics and real-world evidence. NICE already has a programme of work on data analytics to draw on, so stakeholders should use that to inform their inputs.
Other cross-cutting issues include exactly how quality of life is incorporated into the calculations, as well as dealing with the position of technologies in the pathway.
NICE’s board papers add a few details to the headlines in the NICE press release. NICE will be looking at modifiers in decision-making, health-related quality of life, discounting, cost- minimisation methods, costs used and the general approach to decision-making.
What’s out of scope: hot topics including genomic techniques and digital health
NICE has ruled out some areas this time around. They include genomic techniques and testing strategies, medical devices and diagnostics used to tackle antimicrobial resistance, as well as reviewing digital health technologies. Limiting the review makes sense – NICE does not want to take on more than it can manage, especially as it already has a great deal on its plate.
A NICE style of review
In what is a hallmark of NICE’s approach to reviews, it will be commissioning work from others on different components of the review.
This is what has happened in previous reviews, such as exploring issues for NICE methods for regenerative and cell therapies or the adoption of changes to tools that measure quality of life.
This means it is likely that academics will decide on the parameters of the review, but there will also be a methods working group to include senior NICE staff, academics, patients and the life sciences industry, as well as NHS England and DHSC.
There’s also a working group to bring together feedback from patient organisations that will specifically look at improving patient involvement. Overall, there is clearly a lot of infrastructure for the review.
No silver bullet
There are other ongoing changes that should help the NHS to meet its goal to provide a high quality of care that offers good value for money to the NHS. They include the Accelerated Access Collaboration (AAC) – a fast-track route into the NHS for ‘breakthrough’ medicines and technologies – as well as the development of an NHS England Commercial Framework.
This framework was promised early in 2019, but has yet to be delivered (an indicator that there are probably a lot of notes in red pen on the working drafts). Getting the Commercial Framework right could go a long way to getting more drugs approved and getting value for money, depending, of course, on the advantages for companies.
Prospects for change: evolution
It’s wise to keep an open mind on just how much scope for change is realistic. There are already a couple of examples which might point to low expectations. NICE commissioned work on regenerative and cell therapy products in 2016 that found that NICE appraisal methods and the decision framework were applicable to these products.
It also said that there may be cases where innovative payment methods are needed to provide access. That feels like a more generally applicable finding, yet this is not within NICE’s parameters.
NICE has also been criticised for not adopting the EQ5D5L valuation set. NICE prefers to draw on, or map, submissions from companies, rather than the generic EQ5D3L health-related quality-of-life tool. That’s been the case since 2008.
There has been a great deal of work to look at improvements to the EQ5D, including the development of a more nuanced tool with five levels instead of three. The EQ5D3L allows people to select from three options: no problems, some problems and extreme problems.
The 5L version allows them to select from five options: no problems, slight problems, moderate problems, severe problems and extreme problems.
NICE has said, however, that it won’t adopt the valuation set for the 5L, and will instead stick to the valuation set for the 3L (this doesn’t mean the 5L questionnaire can’t be used, it’s just the calculations that build on it that should be based on 3L valuations, not 5L valuations). This is within scope, so there may be a change now.
It was also only in June that the results of a study, with £120,000 funding from Myeloma UK, reported on measuring patient preferences and explored how to determine how patient preferences data could be used in HTA.
The finding that there is no ‘one size fits all’ approach, and that more work is needed, is not exactly revolutionary, nor does it seem to make a case for change.
NICE also doesn’t expect to see big changes in methodologies for wider societal benefits since it has already looked at part of VBA, so it doesn’t expect to include these in future appraisals.
There’s also an important point in the July 2019 board papers discussing the review. The papers state that: ‘This is an incremental development of NICE’s existing world-class approach to evaluating new health technologies. The aim of this work is to review and amend the methods if changes are clearly needed and well justified.’ A message to all to keep their expectations in check.
NICE is planning to work on cases for change and then present proposals for a public consultation in the summer of 2020, with changes being implemented in 2021, but this may turn out to be nothing but a long wait for a small change.