Image: 'Chemo' from Ryan Ozawa's Flickr Photostream

Who’s who and what’s what in the war over cancer drugs

Over the last few days a very nice man from Ipswich called Tim Mason has been appearing in the local media where I live. Tim has an illness called GIST and has been taking a drug called Regorafenib. He only gets that treatment from the NHS because his illness is “refractory” (it hasn’t responded to the surgery and the two chemo drugs that are used on GIST). Tim is appalled that, following a recent NHS ruling, future patients won’t have access to the “third line” drug that he feels has helped him so much. And, as is the way with 21st century patients, he’s not taking it lying down. He’s fighting back by leading the GIST Support UK campaign to get the decision reversed.

Of course everyone knows nowadays that there are “tough choices” to be made about health care. I mean, how many times have you seen some boring geezer from the King’s Fund on the telly banging on about the ageing population, the need to reign in government spending, heightened public expectations, rapid scientific progress ….yadayadayada. In my house it’s usually a cue to nip out and put the kettle on. But given that we’re now all familiar with that well described stage set, it’s worth taking a look at the protagonists who act out the drama. What roles do they actually play? And what motivates their actions? Tim’s campaign, it turns out, provides both a typical cast list and what is fast becoming an oft-repeated plot line.

First up is the Cancer Drugs Fund, which is nowadays a sub-committee of NHS England (keep up!!). The CDF is the body that decides how to spend the special-money-for-expensive-drugs that David Cameron earmarked a couple of years ago in a fit of mid-term populist largesse. It’s budget for 2015-16 is going to be £340 million, so it’s not peanuts even in the extreme world of eye-watering pharmaceutical prices. It’s part of NHS England because that is the post-Landsley body that buys the ‘specialist services’ that your local Clinical Commissioning Group is too small and too ignorant to know about. It’s the CDF that has to make lots of “tough choices” between the many whiz-bang and partially evaluated medicines that big pharma is constantly bringing into the market place. Yes, that’s right, market place. Feeling queasy yet?

In the case of Regorafenib for GIST, the CDF recently put the data through their “tough choices” methodology. Not unreasonably this involved assessing the clinical effectiveness of the drug in two ways. One was how it compared to placebo in terms of the overall survival (OS) of the patients in a trial and the other was the amount of time it gave progression free survival (PFS). The latter means that the patient not only lives but also has a better quality of life while they live. Another assessment was about nasty side effects. Finally, the CDF looked at “unmet need” – a kind of “is this the only the game in town?” question. It’s ethically difficult, scientifically challenging work – but someone’s got to do it.

Unfortunately for NHS patients with refractory GIST, the CDF crunched the numbers and came up with the answer “3B” and said “no”.

In the explanation of their working and their decision, the cost of the treatment also comes into the equation. For Regorafenib this was £4493 (including VAT) per 4-week treatment cycle, and the median treatment was for 22.9 weeks (it works out at about £25,700 per patient) and this was deemed not value for money.

At this point you might wonder: “ok, what’s the cut-off, then?” And this is where it all gets even murkier, because we find that there are two values for money cut-off points. And Regorafenib was assessed at the lower one and didn’t qualify as a special drug “developed for very rare indications”. This is because it is already licensed for use in bowel cancer and thus “did not fulfil the condition” for the higher scoring system. To make this even less transparent, we are told by the CDF that the committee used a “confidential CDF cost scoring system” which “resulted in an overall score which represented insufficient value”. It seems strange, to say the least, that the most important bit was “confidential” – but there you go.

So who are the drug’s inventers, testers, owners and sellers – the people who have come up with that grand-a-week price tag? They are a large international company called Bayer, whose most recent balance sheets can be seen on their website. Blimey! I thought that National Savings was a sensible place to put money – but check out 2013’s 41% share price rise! I think the technical term in high finance and investment is “kerching!” So you don’t need to be a social scientist to work out what their motivations and priorities are.

And this is the nub of it. Whether future Tim Masons get their Regorafenib or not is down to a straight fight. Shareholder’s interests v Patient’s interests. As ever in the context of ‘Big Private Pharma’ and a (still just about) unitary state-funded NHS, this is the battle that constantly has to take place.

Just as the GIST patients have a view, so do the professionals who treat them. Through the website of pressure group Sarcoma UK Professor Ian Judson, Head of the Sarcoma Unit at the Royal Marsden Hospital observed that the decision “…will be a bitter blow… I cannot understand how they were scored 0 for unmet need, which is clearly untrue. Once again patients with rare diseases find themselves being discriminated against unfairly”.

And in a plainly frustrated press release, Andrew Wilson, Chief Executive of the Rarer Cancers Foundation, said: “Patients should never be the victim of a standoff between industry and the NHS. Both sides bear responsibility for this mess and both sides have a duty to try to fix it…. we urge NHS England and pharma to work together in the interests of cancer patients to find a way to ensure these treatments remain available. Neither side should give up on cancer patients.”

So – good luck with that, then. And good luck to Tim and all those with refractory GIST. I’ve signed their petition.