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A consultation on integrating the Cancer Drugs Fund into the appraisal process of the National Institute for Health and Care Excellence has been announced.
All our previous output on this subject can be seen here.
Dr Charlotte Chamberlain, Clinical Research Fellow, University of Bristol, said:
“This is good news compared with the existing system – NICE will now evaluate all drugs, instead of the two-tiered system that was in place with the Cancer Drugs Fund; value for money is now acknowledged as important to assess for cancer drugs and most importantly, there is now an emphasis on collecting meaningful data so drugs that don’t deliver for patients can be removed from the NHS.
“But – and it is a big but – there are some persistent concerns.
“1. This approach prioritises cancer drugs over innovative treatments for other conditions (why not a new medicines fund as in Scotland?).
“2. The new approach prioritises treatment for those in the last two years of life over prevention of disease or potentially increasing quality length of life at other times – so far the public have not consistently shown a preference for the ‘end of life’.
“3. While real world evidence is useful it should not be a replacement for gold-standard research – a managed access fund would be better managed if it delivered the drugs using sound randomised controlled trial principles – and then made conclusions on their value in patient care.
“4. While pharmaceutical companies are charged with collecting sufficient ‘real-world evidence’ there is no mention of how quality of life of patients will be adequately captured – the devil may be in the detail of whether patient benefit can truly be demonstrated without bias.
“And finally, 5. Why is it okay for the NHS to fund treatments that are less evidence-based in cancer than in other diseases – clinical trials are the best way to discover true patient benefit. Isn’t there a danger the system may be manipulated so that cancer drugs, unlike treatment for other diseases, are presented to NICE with less and less evidence so that NHS funding for ‘real-world evidence’ research becomes essential?”
Prof. Karl Claxton, Professor of Economics, University of York, said:
“The Cancer Drugs Fund has done considerable net harm to NHS patients. The current proposals might reduce the scale of that harm but the fundamental problem remains: that the prices charged for new drugs (and especially cancer drugs) do not reflect how much the NHS can afford to pay for the benefits they offer.
“It is proposed that drugs will be paid for as part of the Cancer Drugs Fund if there is a ‘chance’ that they might meet NICE criteria within 24 months once other data have been collected. This means that the NHS will continue to pay unaffordable prices for drugs while data (without proper controls) is collected in the hope that new evidence will suggest they are worthwhile. The alterative, of course, is to not approve drugs at unaffordable prices but wait until either the manufacturer reduces the price or provides the evidence that they are worthwhile at the price being charged. Alternatively, wait until independent and properly conducted research is completed (e.g. randomised controlled trials), which could be funded at a small fraction of the current cost of the Cancer Drugs Fund. Even if research does show that a drug that was funded as part of the new Cancer Drugs Fund meets the NICE criteria after two years, the evidence suggest that these criteria are far too lax anyway, i.e. more health is lost for other NHS patients than is gained when NICE approves drugs even using its existing criteria.
“What is, and has always been required, is to establish price negotiation mechanisms which will allow manufacturers to agree rebates so the effective prices reflect how much the NHS can afford to pay for the benefits. Until the political will is found to address this fundamental problem, it is NHS patients who do and will continue to bear the cost of this failure.”
Additional comment from Prof. Klaxton
“There is a real danger that these proposals will undermine the evidence base for clinical practice in the long run, which may be an even greater threat to health outcomes for cancer and other NHS patients than paying the currently unaffordable prices through the CDF while the type of ‘research’ envisaged is undertaken.
“So called ‘real world data’ really means observing what happens to patients using the new drug but often without proper controls (observing similar patients not taking the new drug), or even when controls are available, any comparisons will be riddled with selection bias (that patients using and not using the new drug differ in important ways we can’t observe) so it becomes impossible to properly assess what would have happened to survival and quality of life without access to the drug.
“The presumption, that we can necessarily gain useful and unbiased evidence in this way without seriously addressing these profound difficulties, ignores all we know about good research design, basic principle of clinical epidemiology and Nobel Prize-winning work on how to analyse observational data. It ignores all the reasons why randomised controlled trials are, quite rightly, the cornerstone of evidence-based medicine.
“The apparent faith in the type of ‘real world data’ that can be collected is either ill-informed and dangerously cavalier, or is a cynical disregard for science and the interests of cancer and other NHS patients in order to avoid addressing the real problem – that the prices charged for new drugs (and especially cancer drugs) do not reflect how much the NHS can afford to pay for the benefits they offer.
“This type of cynicism has a long track-record – the multiple sclerosis risk sharing scheme which was and is a disaster for the NHS but was of great benefit to manufacturers of expensive but ineffective medicines; see McCabe C, Chilcott J, Claxton K, Tappenden P, Cooper C, Roberts J, Cooper N, Abrams K. Continuing the multiple sclerosis risk sharing scheme is unjustified. British Medical Journal 2010;340.”
Dr Alison Cook, Cancer Research UK’s director of policy, said:
“All cancer patients should be able to get the best, evidence-based treatments for their disease. The Cancer Drugs Fund has given thousands of patients treatments that they would otherwise not have had, but it’s been clear for some time that it’s not sustainable.
“The cancer strategy published earlier this year has already set out the need to reform the system and we want to see a long term solution, including a review of how NICE works. We’d like to see a single system for drugs approvals that can respond to the needs of patients.
“We welcome this consultation and now need to assess whether the proposals it sets out will be an improvement.”
Alison Clough, acting CEO of ABPI (Association of the British Pharmaceutical Industry), said:
“Whilst we need time to fully assess the proposals in detail, we are encouraged by today’s announcement.
“NHS England and NICE have listened to the industry on a number of important issues – the process will be NICE led and managed and will allow a period of conditional acceptance of new medicines while they are given time to prove their worth. We believe this is the right move.
“We agree there needs to be rigorous scrutiny of medicines, and medicines pricing, so that the NHS is confident it is getting value for money. But too many patients are routinely missing out, hence, these are just the first steps in what needs to be wholesale reform of NICE and the way new medicines are fairly assessed.
“If we want the best for cancer patients, we have to invest. We still have some way to go if we are to deliver world class cancer outcomes and catch up with the rest of Western Europe. This is despite having amongst the lowest prices for medicines compared to these countries, and a unique scheme where we pay hundreds of millions of pounds back to the Government each year to help them fund new medicines.
“We are pleased that the NHS England consultation has now been announced and we hope this is the first step towards a new and holistic system which will provide all patients with greater access to new medicines.
“The current system of ring-fenced funding for cancer doesn’t work. We have always said that the current cancer drugs fund was meant to be an interim solution, and what is now needed is wholesale change and reform of the medicines assessment process used by the National Institute of Health and Care Excellence (NICE).
“We are committed to working with the Department of Health, NHS England, NICE, charities and patient groups on key areas such as NICE reform and how best to optimise the use of innovative medicines with the ultimate aim of getting the right medicines to all patients at the right time.
“Under its deal with the NHS to keep medicines spending to agreed levels during times of austerity, the pharmaceutical industry is giving £4billion over five years to the Department of Health. This agreement was meant to provide greater access to all new medicines, including cancer, without the need for ring-fenced funds.”
Prof. Paul Workman, Chief Executive of The Institute of Cancer Research, London, said:
“Today’s announcement signals a welcome move towards a more rational and unified system of drug evaluation. It does make sense for the Cancer Drugs Fund to pay for new drugs only after a provisional green light from NICE, to avoid duplicating the processes for deciding whether drugs should be made available on the NHS.
“There are also promising signs of a desire to address the bottleneck we have in cancer drugs being approved for use on the NHS. The proposals could speed up the drug evaluation system – which is good news for everyone – and would increase the number of drugs which would be available for consideration under end of life criteria.
“I welcome the fact that new patients will benefit from early access to promising treatments while further evidence is collected. It’s a good solution for the Cancer Drugs Fund to provide early access to drugs in this way.
“Drug evaluation is enormously complex, and the devil will be in the detail. The proof of success will be in more, and particularly more innovative, drugs actually reaching patients on the NHS.”
Dr Matt Kaiser, Head of Research at blood cancer charity Bloodwise, said:
“Thousands of patients have benefitted from the Cancer Drugs Fund since it was introduced, but the system as it stands is clearly broken. We live in an era of tailored medicine and if we are truly serious about increasing survival rates to the level of the rest of Europe, cancer patients must have access to the drugs that are likely to be most effective in treating their individual disease.
“It’s encouraging to see the Government consulting patients, charities and the pharmaceutical industry to come up with a long-term and financially viable solution.”
* https://www.engage.england.nhs.uk/consultation/cdf-consultation/consult_view
Declared interests
Dr Charlotte Chamberlain: “Recently funded by the National Institute for Health Research for three years to investigate access to cancer drugs in the NHS. Currently employed as a public health doctor with an honorary contract with the University of Bristol.”
Prof. Karl Claxton is a faculty member of the Roche Payer Evidence Council.
ABPI: About the ABPI – The ABPI represents innovative research-based biopharmaceutical companies, large, medium and small, leading an exciting new era of biosciences in the UK.
Our industry, a major contributor to the economy of the UK, brings life-saving and life-enhancing medicines to patients. Our members supply 90 per cent of all medicines used by the NHS, and are researching and developing over two-thirds of the current medicines pipeline, ensuring that the UK remains at the forefront of helping patients prevent and overcome diseases. The ABPI is recognised by government as the industry body negotiating on behalf of the branded pharmaceutical industry, for statutory consultation requirements including the pricing scheme for medicines in the UK.
Dr Matt Kaiser declares that he has no relevant interests.
No others received