NICE: a terrible beauty

NICE (the National Institute for Health and Care Excellence) is one of the many arms-length bodies set up by the last Labour government – and one of the few to have survived. In the health sector it is almost the only one to have reached the present day with its original remit intact.

It has done so despite living much of its life in ferment. NICE has been accused of everything from being ‘a death panel’ – by denying people potentially life-saving treatment – to being one of Britain’s greatest cultural exports – countries around the world have sought to learn lessons from NICE as they seek to introduce health technology assessment and guidelines.

That has made an outline of NICE’s history – its origins, creation and controversies – worth recording; something that John Appleby, Sir Michael Rawlins (its chairman for 13 years) and I have done, under the title A Terrible Beauty.

Terrible because it has been, for those denied treatments that failed to cross NICE’s threshold for cost effectiveness. Terrible too, for those in the life sciences industry who spent time and effort producing new products only to find the price set by their employers was too high – for whatever reason – for NICE to approve.

But it has also been a beauty, in the way that, over the past 16 years, it has sought to balance an essentially unequal equation. Its elements include the interest of the taxpayer in the NHS being cost-effective. The interests of individual patients who, when not paying themselves, have little personal interest in cost-effectiveness – but who do nonetheless have a longer term interest (along with that of other taxpayers) in avoiding situations in which the cost ineffective drives out the cost effective. The interests of clinicians who generally welcome guidance but detest instruction. They are often – although not always – only too pleased to have someone else take on the difficult decisions about what should and should not be funded. And the interests of the life sciences industry – particularly the pharmaceutical industry – which needs a market for its products that will encourage not just innovation, but genuinely ground-breaking and worthwhile innovation, while providing the best possible return on its investment. And all the while, NICE has spared the politicians from having to make these difficult and sometimes heart-rending decisions.

NICE has done this not just by number crunching the cost-effectiveness issues, but also by applying a declared set of social values to the recommendations it eventually makes. And it has sought to operate by clinging to a set of principles: that it will be robust, inclusive, transparent, independent and contestable – allowing everyone, as Sir Michael Rawlins has put it ‘to have their say, although not necessarily their way’.

Some events have undermined NICE’s operations – most notably the Cancer Drugs Fund that Prime Minister David Cameron set up in 2010, which pays for cancer drugs that NICE has judged too costly to be cost-effective. This fund has become more mainstream in the funding of cancer treatments than was ever intended. It has repeatedly exceeded its budget. And it is a solution that pretty much everyone – including the pharmaceutical industry – has come to regard as broken.

NHS England has now produced its proposals for revamping it – retaining the name, but putting the decision about which cancer drugs will and will not be funded back into the hands of NICE. As ever, this is controversial, and there is a fair bit of detail still to be worked out before the new approach goes live in July. One thing this revised approach may succeed in doing, however, is providing better evidence for drugs’ effectiveness or otherwise – something the Cancer Drugs Fund has failed to do – in those future cases where NICE says that more data is needed in order to provide a definitive ‘yes’ or ‘no’ on funding.

Meanwhile, NICE – like all the other arms-length bodies in the health sector, including the Department of Health itself – faces a cut of 25 to 30 per cent in its running costs. So it is likely to have to shrink or surrender some of its myriad activities, which have grown appreciably over the years. It is used to surviving in challenging times, and has, over the years, rarely been far from controversy. However the next year or two, and how the revised Cancer Drugs Fund plays out in practice, could be defining. So its history – why it was established in the first place, what it has done and why – is worth revisiting.

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#545863 mike tremblay
policy advisor
cassis ltd

An interesting read, thank you for this. I would like to address something quite specific, viz the "rule of rescue". NICE has repudiated the applicability of this rule. And while it does refer to whether person at risk of imminent death deserves to be rescued; however, it has real-world consequences NICE's position neatly avoids.
1. Acts of supererogation are acts that are beyond the call of duty. In the absence of some other intervention, the 'state' is the last resort in this respect (think banking, refugees). That means that when all else fails (no one else has the duty), then the state has the duty; this is a moral argument about the purpose of the state itself, and in the UK, the state has the duty in respect of healthcare. This defines the contract with people for the state to provide a healthcare system; having assumed this duty, the state also becomes the last resort when all else fails. In respect of acts of supererogation, that means the state cannot avoid a duty to act as we have defined its moral obligations through the creation of the NHS itself. NICE has determined that there are circumstances that fail this test and in which the state has no duty to act. I don't recall this having been agreed to.
2. Now, the rule of rescue kicks in. In the absence of others acting and acknowledging that the state has a duty to act, the rule of rescue requires the state to be that last resort and to, in effect, rescue. That gaps in service are hard to fill within the state system of provision leads to rationing decisions and potential charges of misuse of public funds for administrative reasons (the European Court of Justice has ruled in this respect). The NICE system is fundamentally administrative in operation since the implementation of its decisions have an impact on the choices clinicians make that do not reflect clinical necessity.
3. The NICE logic model fails because there is a discontinuity between QALYs assessments that are above and below their decision threshold. It is an assumption that the decision logic that applies below the threshold also holds above the threshold; but these two areas are quite different. This is what is called a boundary problem. What NICE has done is defined moral duty to act out of their model. Where the boundary is set does not just separte two parts, it creates two zones in which different decision logic applies. That's why the QALY threshold, regardless of where it is set, while arbitrary (there is no evidence base that tells you where that boundary should be) has consequences. Below the threshold, the rule of rescue does not need to apply. Above the threshold, it defines the zone itself: it is where the withdrawal of the benefits of a medicine, for instance, are denied someone on grounds other than of duty. That people feel strongly that something is wrong with decisions above the boundary is evidenced by the need to create the Cancer Drug Fund. NICE is good for what it is good for, but it should be clear, as Wittgenstein wrote: "Whereof one cannot speak, thereof one must be silent."

#545865 Peter Button
Managing Partner
Home Strait LLP

Beautifully written and very accessible. Highly recommended reading for anybody interested in the challenges of cost effectiveness within the healthcare economy - everybody!

Terrific quotation from another publication at the start of first chapter: “The theory is tough, the science is hard, the economics difficult, and the statistics advanced. The unavoidable trade-offs are often agonizing, much is uncertain, reputations are at stake, and getting things wrong costs lives.” Anthony J. Culyer in A Star in the East:
A Short History of HITAP, 2016

#545879 Dr Freiberg

The comment deserves some thought but there is a misconception at its core. The "rule of rescue" concept seems to suggest a treatment that would lead to a situation of complete, long-lasting, prompt or sufficient "rescue" of a given case that is somehow denied to somebody in need. It suggest a swift recovery, with connotations of an emergency life-saving procedure (a sailor at sea). this is an oversimplification of chronic disease, and very uncommon in clinical practice. The odd events of a treatment so good that chances of recovery are indeed high are duly considered in all of NICE's assessments, even when it costs more.

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