Five ways that NICE could help bring innovative cancer medicines into the NHS

Earlier this year, NICE released a complex and far-reaching set of proposals for redrawing how it evaluates drugs for use on the NHS. The process NICE uses has been the subject of intense debate for many years, and I certainly agree that reform is very much needed.

So the ambition for change was right, but unfortunately I and many others had serious concerns about NICE’s suggested package of reforms.

NICE put forward an extremely complicated formula for assessing drugs, attempting to take into account a host of different factors – from the burden of an illness on an individual to its broader impact on society.

I think NICE is right that there are a number of issues that need to be considered in drug appraisals. But at The Institute of Cancer Research, London, our concern has been that by attempting to include every aspect of a disease and its treatment, NICE could fail to emphasise properly those really important factors that should determine whether or not a drug should be provided on the NHS.

Most crucially, I argued in response to NICE’s proposals that it risked failing to take proper account of how truly innovative a treatment is, and because of this was in danger of closing the door on treatments that are very effective at the end of life – including many important cancer drugs.

NICE has obviously been listening to those concerns, because it has now performed an abrupt about-turn, and announced that it no longer plans to proceed with the changes it had proposed.

In a statement Sir Andrew Dillon, Chief Executive of NICE, said: “Following an extensive consultation, it’s clear that just changing NICE’s methods will not overcome concerns about how the NHS accesses new treatments.” Instead it plans a broader review of how new drugs reach the NHS.

Not every organisation finds it easy to listen and to change its mind as a result of feedback – and I congratulate NICE for doing so.

Nevertheless, NICE was right in its original diagnosis that change is needed.

We need a drug assessment system that is certainly led by evidence but that also properly prioritises and incentivises the discovery and development of exciting, innovative new medicines – including therapies for many different cancers and especially where there is unmet need.

I’ve spent some time thinking about what such a system might involve. Here are five suggestions which if implemented could help NICE bring innovative cancer medicines into the NHS.

1. We should incentivise pharma companies and academic institutions to take the risks in research that are needed to create genuinely new and innovative approaches to treatment.
   
   The pharmaceutical industry has become risk averse, with many companies working on the same drug targets. I certainly accept that competition can be helpful to quality and that different drugs working on the same target in pretty much the same way can offer some benefits – for example in side-effect profile. But the fact is that new versions of existing drugs are unlikely to achieve step-change improvements in outcomes.
   
   The really substantial long-term benefits will come from innovative drugs acting on unprecedented targets – or on established targets in very new ways. These involve a higher risk of failure than drugs working on established targets.
   
   At the ICR, we focus on discovering highly innovative drugs. We do this by attacking new drug targets – protein molecules in cancer cells that when hit give a strong anticancer effect. By carrying out high-risk drug discovery we can bridge the so-called ‘Valley of Death’ that commonly separates breakthrough discoveries in cancer biology from the development by pharmaceutical companies of new therapies based on those breakthroughs.
   
   This gap can all too often be a chasm – especially and frustratingly in cancer where there is a wealth of potential new targets emerging from genome research.
   
   To encourage the risk-taking research needed to develop game-changing medicines, it’s essential that any system for evaluating drugs gives strong weighting to their degree of innovation – and views the cost-effectiveness of a highly innovative drug more sympathetically than a ‘me-too’ drug, which acts on a target in a similar way to many others already on the market.
   
   That’s partly because NHS patients deserve access to innovative new treatments, and partly also because we have to reward those companies and academic institutions that take risks – because if innovating is not rewarded then no one will do it.
   
   NICE can currently raise the threshold for accepting a drug for use on the NHS from £20,000 to £30,000 per QALY if it is regarded as innovative – still much lower than the £50,000 per QALY for drugs that are effective at the end of life.
   
   I believe NICE should have the flexibility to pay more than £30,000 per QALY for drugs that are regarded as especially innovative. Equally, it could decide to pay less for ‘me too’ drugs that replicate the actions of others already available and are unlikely to represent a real step change.
2. We need a system that provides patients with stable access to cancer drugs over the long term.
   
   I have welcomed the efforts the Government has been making to expand access to new cancer medicines via the Cancer Drugs Fund.
   
   But the Cancer Drugs Fund isn’t the ideal solution. There is still no news on whether it will be extended beyond 2016, which means drugs that are currently made available could drop off the menu in two years’ time.
   
   Also the Cancer Drugs Fund is available only for patients in England, whereas what is needed is a process for bringing new drugs into the NHS that works for people with cancer right across the UK.
   
   More generally, the Cancer Drugs Fund always felt like a sticking plaster solution, which ameliorates the shortcomings of the current drug appraisal system but doesn’t actually address them.
   
   I’d like to see a long-term, sustainable solution to the issue of drug access, one that doesn’t necessarily separate out cancer drugs as a special case, but which does ensure that the system as a whole gives innovative cancer medicines the priority they deserve.
3. Our systems for drug approval need to treat all cancer patients equally.
   
   A basic principle for a sustainable solution, capable of attracting widespread support in the long term, is that it is seen to be fair as possible to all patient groups.
   
   I am strongly opposed to any moves to pitch one group of cancer patients against another – I think all cancer patients are equally important.
   
   I agree that the current drug approval system doesn’t provide enough incentives to develop treatments for children, which is one reason so few new childhood cancer drugs reach the market.
   
   I certainly support efforts to rebalance the system to help develop drugs for children’s cancers – as long as the utmost care is taken not to disadvantage other groups, such as older cancer patients.
   
   At the ICR, we’ve suggested changes to ensure pharma companies investigate cancer drugs in children whenever their mechanism of action suggests they might work.
   
   But I feel NICE’s previously proposed formula had risked going too far, by heavily favouring drugs for paediatric medicines ahead of those for older people, while also removing the end-of-life criteria that have been so vital for gaining access to drugs for cancer patients.
4. We must try hard to be fair to all cancer patients.
   
   
   NICE needs to address the illogical discrepancies in the way its end-of-life criteria are applied.
    
   Cancer drugs tend to be tested first in patients who are near the end of their lives, since it is generally considered appropriate to initially offer patients standard-of-care treatment. So use of end-of-life criteria, which favour drugs that benefit patients in their last two years of life, is essential to get new treatments approved for NHS use in the first instance.
   
   Some critics argue that the costs of new drugs are not justified by their benefits, but in my experience the extension of life – commonly involving an extra several months – is highly valued by patients, their families and friends. There are also two important additional benefits that are not always so well appreciated.
   
   Firstly, the end-of-life criteria have been a vital gateway into the NHS for drugs that are subsequently shown to be even more effective when given earlier in treatment. A case in point is the prostate cancer drug abiraterone – discovered at the ICR – which was initially approved for use after chemotherapy and then found to be active before chemotherapy as well, sparing patients the toxic side-effects of chemotherapy.
   
   Secondly, once approved drugs can go on to show major benefits as part of combination treatment – and drug combinations will be vital to overcome evolution of cancer within a patient and the resulting scourge of drug resistance.
   
   Without NICE’s end-of-life criteria, many cancer drugs – including abiraterone, which has transformed care of prostate cancer – would never have been made available on the NHS.
   
   But in fact abiraterone has also been the victim of a bizarre discrepancy in the current system.
   
   Its use after chemotherapy was approved under the end-of-life criteria, but its use earlier in treatment was turned down, on the grounds that patients treated at this stage lived too long for end-of-life criteria to be applied.
   
   That can’t be logical. Surely if a drug is approved under end-of-life criteria in advanced cancer, NICE should have the flexibility to apply the same criteria when deciding whether a treatment should be made available earlier in the course of disease.
5. NICE should continue its open dialogue with a broad range of stakeholders.
   
   Broad involvement of stakeholders is essential to produce a drug assessment system that commands extensive support. So I’m very pleased that NICE says its methods need to be looked at “as part of a wider review of the innovation, evaluation and adoption of new treatments (including those for cancers)”.
   
   I applaud the fact that NICE will carry out its broader review in consultation with key groups such as “patients, people working in or with the NHS, the life sciences industries and health researchers”.
   
   Although we await the details, I welcome the proposal to establish an “office for innovation inside NICE” aimed at providing pharmaceutical companies with a “flight path” through the various stages of developing, evaluating and adopting their products for the NHS.
   
   I hope this office will adopt the positive approach to innovation that I’ve outlined. If so, this should encourage high-risk, high-gain research by providing industry with reassurance that there will be joined-up thinking, planning and action.
   
   I welcome NICE’s proposal to reach agreement with NHS England and the Department of Health on the NHS's willingness to pay for new treatments, taking into account special cases, among which cancer is named.
   
   Likewise I support the proposal to explore sharing of risk between companies and the NHS, with the aim of progressively assessing and reflecting the value of new treatments as evidence builds on their value.

Final thoughts

NICE has a very difficult job in trying to come up with a system for accepting drugs into the NHS. It must try to satisfy numerous different and often passionately conflicting interests.

But the general principles I have outlined could help to produce a system that could deliver what everyone ultimately wants – really innovative, effective new drugs, for cancer and many other diseases besides.

We need innovative, affordable drugs that make a real difference in defeating human disease. Industry must be incentivised to be more innovative and at the same time must be realistic about pricing in a cost-constrained environment. We need to mend what is a failing drug development ecosystem which is not delivering as much clinical benefit as it could from the breakthroughs in our understanding of human cancer.

All stakeholders have a role to play in fixing the model, and NICE has moved one step in the right direction.