The CDF: friend and foe?
pharmafile | November 11, 2013 | Feature | Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing | ABPI, CDF, Cancer, NICE, eric low
Few things in my professional life have troubled me more than the Cancer Drugs Fund (CDF) since its inception in 2011 as a quick political response to newspaper headlines about cancer patients dying, because their ‘life-saving’ treatment had been turned down by NICE.
Had it been introduced ten years ago, I might have called it the best thing since sliced bread, but as I have come to understand our healthcare environment better, I am not convinced.
I am sure the CDF was introduced with good intentions. However, it is evidently a policy conjured up at the last minute, just before the publication of the Tory party manifesto, with little thought about its long-term implications.
There is no doubt that patients are alive today as result of the CDF and thousands have enjoyed precious extra life thanks to the fund. But it was only ever meant to be a short-term solution to buy some time in which to find a long-term fix to the systemic issues facing the introduction of new treatments.
Given the recent news that the fund is to be extended for a further two years, the time seems right to consider its pros and cons, and the issues that needed fixing in the first place.
The situation before the CDF
There is no doubt that, for a variety of reasons, some effective new drugs were rejected by NICE with the result that some patients were denied access to treatments that had the potential to prolong their life.
But we should not forget that NICE was introduced to tackle the so-called postcode lottery and cancer was the area most challenged by the variation in access.
Getting new treatments approved for use within the NHS is getting harder – and rightly so. For too long, the NHS didn’t set the bar high enough for the level of benefit needed and it seemed that any drug at any price could be approved for use. This changed almost overnight with the introduction of NICE about 12 years ago.
Since then, NICE has determined which new drugs should and shouldn’t be used in the NHS. It does this through a fairly sophisticated appraisal process by which it establishes both the clinical and cost- effectiveness of a new drug, measuring its value to patients and the NHS by using tools such as EQ5D and the QALY.
NICE has a nominal QALY threshold of between £20,000 & £30,000. Put simply, if a new drug falls within this range it will generally be approved for use; if it has a higher cost it is extremely likely that it will not, although there have been exceptions.
Everyone is happy when ‘their’ drug is approved but when it isn’t, a media circus often ensues. Over recent years, an increasing number of new cancer medicines have been turned down, especially when they add little extension of life – although this has been less of an issue with the introduction of the end of life criteria, patient access schemes and the ability of the manufacturer to offer a direct ‘commercial in confidence’ discount to bring a drug within the cost-effectiveness threshold.
A NICE appraisal is a risky business for all concerned as it brings together data from global Phase III licensing studies and the robust, but not perfect, world of HTA appraisals. This is not a marriage made in heaven. Phase III studies are designed to prove a compound’s safety and efficacy and to get marketing authorisation as early as possible.
The trial design – endpoints, comparator and patient group characteristics – is at odds with the data needs of HTA and real-world clinical practice. As a result, the data is often difficult to interpret; add a price that is as high as the manufacturer thinks the market will bear, and NICE has a difficult challenge trying to decipher what all of this means from a UK perspective. This is an issue that is undermining the image and trust in the industry.
There are other issues, too. NICE is not perfect: it does not appraise the majority of new medicines and too its guidance is restrictive and does not reflect state-of-the-art clinical practice. As a result, doctors do not always have freedom to use new drugs in the most clinically effective and efficient ways.
Because of the way we introduce new treatments, the results from academic clinical research cannot be readily introduced into clinical practice at a national level, denying patients care that is considered state of the art outside the UK.
As a consequence, many patients are still denied access to the best possible treatment. So there are big issues that need urgent solutions – but how much of a solution is the CDF?
CDF pros and cons
The upsides of the CDF are obvious: thousands of cancer patients have benefited from access to drugs that they otherwise would have had been denied. We cannot underestimate the positive impact that this has had. This benefit is what we are all, collectively, working towards.
However, in achieving this, we need to consider the potential downsides. Rigid, high barriers to market entry are critical drivers of innovation and creativity in drug discovery, design and development.
A weak customer- or demand-side is not helpful in the long run, and in some ways the introduction of the CDF has weakened demand-side and is not giving the right signals to industry about what is needed.
There is no assessment of value or cost- effectiveness of CDF-approved drugs and the CDF has no remit to negotiate a price discount. Thus, there could be a scenario where NICE has declined a drug, even when the manufacturer has offered a discount (PAS), but subsequently the drug could be approved by the CDF without the discount.
This doesn’t make for a good use of limited NHS resources: although extra funding has been provided for the CDF, we should still demand value for money – it’s this that I think is important, not just affordability.
Although there are some very good drugs in appropriate indications on the CDF, others that have been approved give some cause for concern. Many that have been approved for use at the end of life only work in about one in four of patients.
With no way of identifying the one in four in which it will work, we have to give it to all four to find out. This may only give that one patient an extra few weeks or months over the standard of care. Such a limited time is, of course, hugely important and no patient should be denied access, but how much are we, or should we, be prepared to pay for this?
There are also concerns about the extent to which the CDF is sustainable, even in the short-term. Reports predict a huge overspend and the list of applications to the fund is becoming almost unmanageable.
This means that unless the government significantly increases CDF funding, its options would be to introduce cost-effectiveness criteria to help make decisions, or introduce a procurement remit to negotiate prices.
Without these options it will be forced to remove a drug from the list every time a new drug is approved. The CDF could become a whole new battleground – a mini-NICE – and a rich source of Daily Mail headlines.
I also don’t think that, in policy terms, the CDF looks good for the government. When you
look at the totality of its healthcare policy and the rhetoric that surrounds it, it is hard not to wonder whether policy is indeed about looking good, pleasing people and winning votes at the expense of serious, sensible, sustainable long-term solutions.
One challenge posed by the CDF is seldom discussed: the impact it has on clinical studies in the UK. Normal practice is that the comparator in UK academic studies should be standard NHS clinical practice. This has often been problematic where UK standard of care is out of kilter with global practice.
Study design in the UK has always been odd as a result since it is difficult to do novel, competitive studies where the control arm is not universally recognised as standard of care. Drugs approved for use through the CDF are not approved as a comparator in clinical studies: how then can they be used in trials to compare the role of future medicines?
Conclusion
Notwithstanding the short-term benefits to patients, the CDF is an anomaly. Those that have called for its introduction and continuation now need to put their admirable advocacy talents to better use and to focus on calling for long-term, systemic solutions.
I implore policymakers to put an end to the ‘sticking plaster’ culture of policy development and to think more carefully about the policy changes they make. Patients deserve better and unless we become smarter and more courageous, the number of cancer patients in the future who will pay the price for our actions today, will far outnumber those that have benefited to date from the CDF.
Eric Low, Myeloma UK chief executive
Related Content
Combination treatments: Takeda’s Implementation Framework and the broader landscape
Pharmafile talks to Emma Roffe, Oncology Country Head (UK & Ireland) about the combination treatment …
Central nervous system cancer metastases – the evolution of diagnostics and treatment
The current forms of immunotherapy, how T cell therapy works and what the future holds
BioMed X and Servier launch Europe’s first XSeed Labs to advance AI-powered antibody design
BioMed X and Servier have announced the launch of Europe’s first XSeed Labs research project, …
