Political dynamite - England’s Cancer Drugs Fund

Proposals to consider cost in decisions about what will, and, crucially, won't, be paid for by the Cancer Drugs Fund (CDF) in England are the latest changes in the controversial history of the ring-fenced fund. But why is the CDF such a hot topic politically? As a four-week consultation on the CDF by NHS England closes this week, Leela Barham explains what has happened in its four-year history.

A reminder: why the CDF came about

Cast your mind back over four years and remember what it was like before the CDF was created. It was 2009, and headlines screamed out that patients with cancer were being denied life-saving drugs by the National Institute for Health and Care Excellence (NICE), whether that was for kidney cancer, stomach cancer, lung cancer or even bowel cancer.

Not only was the mainstream media highlighting rejections by NICE, but so too were academics who suggested that NICE had been getting 'tougher', rejecting 27 per cent of cancer medicines between 2006 and 2008, compared to 4 per cent from 2000 to 2006. In the year before, the patient group Rarer Cancers Foundation (RCF) had highlighted problems with the funding requests made on behalf of individual patients, many of which were for cancer medicines to treat rare cancers. A postcode lottery existed and something had to be done.

The 'something' turned out to be the CDF. It was first put forward in the Conservative Manifesto for the 2010 General Election and was, arguably, a vote winner. It was then adopted as a policy of the new Coalition Government in May 2010. The RCF takes the credit for "securing commitment to establish a Cancer Drugs Fund".

A temporary measure

The CDF was positioned as a temporary measure, a fund that would enable access whilst the knotty problems that caused all those rejections from NICE could be tackled.

Among those problems – which still remain to be solved – was the use of Quality Adjusted Life Years (QALYs) in cancer which highlighted issues including:

• insensitivity in the underpinning tool preferred by NICE - the EQ 5D – a general tool used to measure health-related quality of life

• the assumption used by economists that people were willing to trade life expectancy for quality of life at a constant rate, irrespective of the closeness of death

• that it was debatable whether it should be those with cancer, or the general public, who should provide valuations used to inform the cost effectiveness calculation

QALYs aren't the only problem that needs addressing. Crossover in trials, while ethically defensible, makes determination of cost effectiveness even harder. That problem remains an issue today, particularly in light of personalised cancer medicines. Predicting long-term outcomes from surrogate outcomes used in trials is also a challenge.

With cost effectiveness driven by both the cost and effectiveness sides of the equation, price plays a role too. That said, there is a debate about what happens when, even if the drug is free, it still isn't cost effective because of the expensive care that people will need if they live longer.

These problems are hard to tackle, so Value Based Pricing (VBP) was going to be developed over time, with the CDF giving "patients access [to] the cancer drugs their doctors think will help them".

Not a universally popular measure

Not everyone was happy with the idea of a CDF back in 2010. Some asked why those with cancer should be given priority over others with different distressing conditions. Even the Department of Health's (DH) impact assessment acknowledged that spending £200 million on the CDF would: "displace 8,000 QALYs, worth £480m from patients elsewhere in the NHS".

Others said it would undermine NICE since, in effect, it would provide funding even when NICE said 'no'. The rules of the fund automatically exclude drugs where the company has not taken part in a NICE appraisal, to avoid sidestepping NICE altogether. Of course, companies might take the chance of a higher price, and a NICE rejection, knowing that they should still get some revenue via the fund.

Practicalities were a concern to the pharmacists who would take a lion's share of the everyday management of the fund, including whether there was enough money to meet demand.

Evolution of the CDF

The CDF has evolved considerably over time, moving from regional funds to a national fund managed by NHS England, and becoming more generous (figure 1). Originally slated to close in 2014, in September 2013 the Prime Minister announced it would continue to 2016. He also pledged that if he was re-elected, he would recommend that it be continued.

Figure 1: CDF: A Timeline

The CDF's spend has also varied (Figure 2), staying in the black until 2013/14. Some might ask where all those 'spare' millions went in the past. Although £15 million went on radiotherapy, it is unclear where the rest ended up.

Figure 2: CDF underspends and overspend

The fund total will reach £1bn by 2016. The number of patients who have benefitted was 55,000 at the last count.

As for the companies that have profited, Roche is the biggest beneficiary of the fund. Roche has been credited with playing a significant part in its genesis and has links with the RCF. Roche isn't alone; according to the media, more than half of the drugs on the CDF are produced by companies who have funded the RCF.

The CDF hasn't speeded up access according to academics, instead it's mostly provided access to those drugs not seen as cost effective by NICE. That continues to be the case; for 2014/15 so far, 63 per cent of applications are for indications not seen as cost effective by NICE. An important minority, 12 per cent, are for indications not being considered by NICE, and 3 per cent are for off-label indications.

No-one seems to know the impact of the drugs that the CDF has paid for in terms of survival or quality of life. Audit is included as part of the fund but, as yet, no results have reached the public domain. Though unproven, perversely, it could be that success, in terms of patients living longer and going on to have further courses of treatment, could be behind the overspend. Disentangling that from the impact of prices of the drugs covered in the fund or the increase in cancer cases is not straightforward, though.

Consultation on a new approach

Since going into the red, the fund has been given an £80 million/year boost, which was announced in August this year. That comes with strings attached; NHS England is now consulting on changing its approach to deciding what is on the list, and what will be removed.

The consultation title is an oxymoron; 'Proposals for a sustainable Cancer Drug Fund' for a temporary policy that is due to end by March 2016. NHS England wants to know people's views on a re-evaluation process that adds in cost, and which could either see drugs removed from the fund or discounts from companies to keep them on. NHS England says it may need to make adjustments again, so there could be more delists or more discounts in future.

NHS England always had the chance to make changes to the list. In fact, it reduced the list when it moved from regional to one national fund. It also secured what were described as 'confidential commercial schemes' to include Aflibercept in second line colorectal cancer, Bevacizumab in second line Ovarian Cancer and Pertuzumab in breast cancer in May 2013. A good question is why NHS England has chosen now, with only a few months until the next General Election, to take a harder line?

Discussion also includes greater 'alignment between CDF and NICE assessment processes, and evaluation through commissioning option'. The latter is untested for medicines to date, but is the idea of exploring real-world use with limited funding in small patient groups.

NICE itself reportedly wants more of a role, pointing out that the fund 'makes no sense'. But since NICE was part of the 'problem' in 2009 before the fund came in, wouldn't that just bring out the same frustrations as before? Reform of NICE would be needed (especially as NICE has rejected treatments more often since the CDF has been in place for cancer drugs than it did before the CDF existed). Or lower prices, most likely through Patient Access Schemes (PAS) that allow confidential discounts to be given to the NHS and allow NICE to say 'yes'. There have been a lot of these in cancer; 22 since 2007 and 14 of those are simple discounts.

What else has changed?

The much talked about VBP (now known as Value Based Assessment or VBA) has been delayed until after the 2015 General Election.

Patient groups have also become more influential and sophisticated, and may well be willing to play a role, not only in commenting on the processes of the CDF but also in being part of decision making.

Regulators have been changing their approach, encouraging earlier access through the Adaptive Licensing pilot from the European Medicines Agency, and the Early Access to Medicines Scheme from the Medicines and Healthcare products Regulatory Agency.

The debate on pricing has changed too; Roche in particular has been singled out for high prices not only by NICE, but also with an online petition calling for the company to cut the price of breast cancer drug Kadcyla. Comments have turned personal suggesting that Roche's head of health economist should be 'ashamed of herself'.

That's not the only industry angle though; the 2014 Pharmaceutical Price Regulation Scheme (PPRS) has also brought in what is, in effect, an industry-wide rebate scheme. If NHS spend on branded medicines goes above a pre-agreed growth rate then the DH gets money back - totalling £150million so far this year. Not only that, but these are real pounds flowing back compared to those tricky productivity or efficiency gains typical of the NHS. Quite how the CDF and the 2014 PPRS work together is not clear.

What could happen next?

It's difficult to know what will happen next. The consultation closes this Friday 31 October and there are bound to be a lot of responses. Many will simply say that they don't support the proposals; whether that's a company with a drug on the fund, or one which might want to have its product on the fund some time soon, or a patient group which fears that its patients will have to go through the individual funding request process or somehow find the cash itself to pay for delisted drugs.

By March 2016 the UK may have a new Prime Minister, and the Fund could disappear altogether. If it does go, what won't disappear is the focus on cancer.

About the author:

Leela Barham is an independent health economist and policy expert who has worked with all stakeholders across the health care system, both in the UK and internationally. Leela works on a variety of issues: from the health and wellbeing of NHS staff to pricing and reimbursement of medicines and policies such as the Cancer Drugs Fund and Patient Access Schemes. Find out more here and you can contact Leela on leels@btinternet.com

Have your say: What would you do with the Cancer Drugs Fund?