The National Institute for Health and Care Excellence is making progress in improving access to new cancer treatments but it needs to take proper account of true innovation and be more flexible on price for genuine game-changers.

That is the view of Paul Workman, one of the world’s leading oncology experts and interim chief executive of the Institute of Cancer Research in London, who has laid out a series of suggestions for the cost watchdog. He was concerned about NICE’s suggested package of reforms, “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”.

Prof Workman was therefore delighted to see that NICE has now performed “an abrupt about-turn”, having announced in September that it no longer plans to proceed with the changes it had proposed. Chief executive Sir Andrew Dillon said last month that “following an extensive consultation, it’s clear that just changing NICE’s methods will not overcome concerns about how the NHS accesses new treatments”.

The ICR chief said that “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”. He then went on to outline his five-point plan.

Incentivise genuine innovation

First up, he believes “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”. Prof Workman argues that while they may offer some side effect benefits, “the fact is that new versions of existing drugs are unlikely to achieve step-change improvements in outcomes”.

He argues that “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 so he believes NICE  should “view the cost-effectiveness of a highly innovative drug more sympathetically than a ‘me-too’ drug”.

Prof Workman notes that NICE can currently raise the threshold for accepting a drug for use on the NHS from £20,000 to £30,000 per QALY  (quality-adjusted life year) 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”. He believes the Institute  should have the flexibility to pay more than £30,000 for drugs that are regarded as “especially innovative” and pay less for me-toos.

Cancer Drugs Fund 'sticking plaster'

Secondly Prof Workman calls for a system that provides patients with stable access, noting that the Cancer Drugs Fund “isn’t the ideal solution”. He adds that the latter “always felt like a sticking plaster solution, which ameliorates the shortcomings of the current drug appraisal system but doesn’t actually address them”.

Next, he argues that drug approval systems need to treat all cancer patients equally, feeling that “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”.

His fourth point is that NICE needs to address “the illogical discrepancies in the way its end-of-life criteria are applied.” The latter has been “a vital gateway into the NHS for drugs that are subsequently shown to be even more effective when given earlier in treatment”, he says, giving the example of Janssen’s prostate cancer drug Zytiga (abiraterone).

'Bizarre 'Zytiga case

The drug, discovered at the ICR, was initially approved for use after chemotherapy and then found to be active before chemotherapy as well, sparing patients the toxic side-effects of the latter.However, Zytiga “has also been the victim of a bizarre discrepancy in the current system”, as its use after chemotherapy was approved under the end-of-life criteria, but 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,” Prof Workman says. “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”.

High-risk, high-gain research

His fifth and final suggestion is that NICE “should continue its open dialogue with a broad range of stakeholders” and is particularly enthusiastic  about proposals for 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”. This should encourage “high-risk, high-gain research”.
Prof Workman acknowledges that “NICE has a very difficult job [and] it must try to satisfy numerous different and often passionately conflicting interests”. However, “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”.

He concludes by saying that “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”.