"Democracy," Winston Churchill famously said, "is the worst form of government, except for all the other ones that have been tried."
The same can be said for our system of funding cancer drugs: As flawed as it may be, we have not found better.
We've seen a striking example of this paradox in recent weeks with the story of Jill Anzarut. Most readers will know that the 35-year-old Toronto Mom has been denied treatment with the "lifesaving" drug trastuzumab (brand name Herceptin.
This seeming injustice has made Ms. Anzarut a cause célèbre, especially in the blogosphere and the Twitterverse.
No one dares suggest that the decision to deny coverage was the right one.
Here's a bit of blasphemy for you: It is the right decision. At the very least, it is entirely justifiable.
We have a publicly funded health system. We owe it to ourselves to spend our health dollars as prudently and efficiently as possible.
Doing so requires making tough decisions, even unpopular ones. That means being guided by evidence, not emotion.
It means sometimes saying "No." And that's the real issue here.
So let's look at the evidence, beginning with the bare bones of Ms. Anzarut's story.
It begins with every woman's worst nightmare: Detection of a small lump. She was diagnosed with breast cancer. Ms. Anzarut's cancerous tumour was caught early; it measured 0.5 centimetres. This early detection and prompt treatment reflects wonderfully on our cancer-care system.
She has undergone surgery and has now started chemotherapy. Her prognosis is excellent.
But her story has made headlines because Ontario has refused to pay the $40,000 cost of Herceptin, deeming that only tumours of one centimetre or larger benefit from the drug treatment in conjunction with chemo. (By contrast, provinces such as British Columbia, Alberta and Saskatchewan cover treatment of smaller tumours.)
This seeming injustice has made Ms. Anzarut a cause célèbre, especially in the blogosphere and the Twitterverse. Cancer Care Ontario, the Ontario Ministry of Health and the Minister of Health have all been deemed cruel and heartless. How dare they say "No."
Are the angry, unabashed supporters of Ms. Anzarut seriously suggesting that an insurance program – be it public or private – has to pay for every single drug and every single treatment no matter how marginal the benefit, how high cost and how grave the risks?
Sure, Herceptin was prescribed by the woman's oncologist. But do we really want individual doctors – no matter how qualified and well-meaning – to have a blank cheque? Do we want no checks and balances?
And what about patients: Should they have the right to demand a treatment, no matter how experimental and no matter how expensive, because they read about it on the Internet or in The Globe and Mail?
Do we really want the provision of healthcare services to be an all-you-can-eat buffet? Do we want treatment priorities determined by a cybermob?
Having processes, rules, policies and cost controls is too often portrayed as bureaucratic, penny-pinching and inhumane. But it is a necessity – a necessary evil, if you will – in every sector, and the health sector is no exception.
There are groups of experts that draft clinical treatment guidelines and those that determine which drugs are funded (or not).
They use the best evidence at their disposal to make recommendations that will benefit the greatest number of people, and minimize risk.
The biggest flaw in the system is not the decisions – like the one to limit Herceptin to those with tumours of one centimetre or larger – it is a failure to communicate how the decisions are made. Committee members are not evil bureaucrats; they struggle with every one of their rulings because the evidence is complex and ever-changing, and they are fully conscious that the decisions matter to patients at the coalface.
It is an infinitely thankless task but a crucial one.
So, let's take a look at the evidence in this case.
Herceptin is a monoclonal antibody approved for treatment of advanced-stage HER2-positive breast cancers and to lower the risk of recurrence in women with HER2-positive, early-stage breast cancer that has a high risk of recurrence.
We are told Herceptin cuts breast cancer recurrence by half. In studies, women with advanced-stage breast cancer who take Herceptin (along with a standard chemotherapy) saw their risk of recurrence fall to 15 per cent after four years, compared with 33 per cent taking chemo alone. And remember, the drug is only for women with who are HER-2 positive (about one in four.) The death rate in this group fell a modest four per cent after four years.
In women with early-stage breast cancer, the gains were more modest: A recurrence of 14 per cent in Herceptin users, compared with 22 per cent in the chemo-only patients, and there was no change in death rates, at least in the short term.
Women with tumours smaller than one centimetre were excluded from these studies. Why? Because they have significantly higher long-term survival rates.
This matters because Herceptin – like all drugs – has side effects, something rarely mentioned in the emotional appeal for providing the drug. Those side effects can be significant: cardiomyopathy (a form of heart failure), pulmonary toxicity and an increased risk of infection; the drug is also toxic to an embryo or fetus, an issue for women of childbearing age.
In determining if a drug is appropriate, a dispassionate risk-benefit analysis is crucial.
There is some circumstantial evidence that Herceptin may benefit women with tumours smaller than one centimetre – in the form of retrospective studies of women with breast cancer that suggest the recurrence rate in those with small tumours is higher than previously believed.
Some provinces decided to fund, others did not. Again, both positions are entirely justifiable. (The absurdity of having province-by-province decisions is a topic for another day.)
Regardless, the essential point is that the line needs to be drawn somewhere. At what point should a woman with HER-2 breast cancer get "lifesaving" Herceptin: when the tumour measures 1 cm? 0.5 cm? 0.1 cm? 0.0001 cm? Should we test all women for HER-2 receptors and give them all the drug on the off chance they may develop breast cancer?
In the drug world, these reductio ad absurdum arguments are commonplace. That's why treatment and funding decisions need to be made on the evidence we have, not the evidence we wish we had.
As much as we all feel for Ms. Anzarut's plight – and who can imagine a more cruel fate than a 35-year-old mother of two young children with cancer? – in her circumstance, Herceptin remains an expensive treatment with a potentially marginal benefit and some not-insignificant risks. No amount of screaming, blogging and tweeting will transform Herceptin into a miracle drug.
The system we have may, at times, seem like the worst possible one for dealing with these heart-wrenching issues. But it is better than the alternative.