Fiona McQueen: Expense condemns some to life of pain

If I were to develop rheumatoid arthritis tomorrow and if I were unlucky enough to have the severe joint-deforming version, I would have to seriously consider leaving New Zealand.

If my arthritis was progressive and did not respond to conventional therapies so I was becoming unable to work and looking at going on to a sickness benefit, there would be little choice. I would go.

In Australia, or Britain or anywhere in the European Union, or the United States or Canada, assuming I could gain citizenship and wait the prescribed period of time, I would have access to a new class of drug that has revolutionised therapy for rheumatoid arthritis sufferers.

These drugs are the TNF-inhibitors or anti-TNF agents. TNF stands for tumour necrosis factor and this refers to a molecule that plays an important role in inflammation and is found normally at low levels in many tissues within the body.

During the 1970s it was discovered this molecule (and others that perform a similar function) was present at high levels in the bloodstream and particularly in the joints of patients with rheumatoid arthritis.

It caused ongoing pain, redness and swelling of the joints and also damage to the underlying cartilage and bone. A great deal of research followed and eventually drugs were developed that specifically targeted the TNF molecule.

These drugs were found to be remarkably effective. They could reduce the pain and stiffness of arthritis so patients who had previously given up work were able to return, and sometimes take up sport.

As time went on compelling evidence appeared suggesting these drugs could also stop the progression of bone damage and joint deformities, characteristically seen in the hands and feet of rheumatoid arthritis patients. They were much more effective than standard drug therapy in this respect.

Why are TNF-inhibitors not available in New Zealand? They are expensive - between $21,000 and $25,000 a year - and the patient needs to stay on the drug long term. They are not a cure, but all studies have shown they are the most effective therapy available.

There is a downside: in some patients there are side-effects and in a proportion the arthritis does not respond. In countries where these drugs are available, specialists monitor patients receiving them carefully so they are stopped rapidly if they are ineffective or causing toxicity.

In New Zealand, they have been made available (for the past 12 months) to the very small group of patients with the childhood form of severe rheumatoid arthritis. To date, 26 patients have started one of these drugs. Unfortunately, when those people reach 18, if the present situation continues, they will have to stop.

One of the drugs is also licensed for use in adults with rheumatoid arthritis if patients can pay privately, but few can afford it. Those who are really in need are often unable to work and on benefits.

Why are these new drugs so expensive? This is a complex issue and brings up many important ethical questions. Research and, in particular, medical research has become a profitable enterprise. Biotechnology companies have invested huge amounts in research and development, recruiting the brightest scientists and using state-of-the-art equipment to push ahead the frontiers of medical knowledge. They have been remarkably successful in some areas, producing drugs such as the TNF-inhibitors and Glivec, used in a form of leukaemia, but other products have failed to reach the market, sometimes after 10 or more years of development.

Thus the products that work and are profitable must cover the shortfall for those that do not, as well as subsidising research costs for the future.

Having said that, it has been estimated that more than 600,000 rheumatoid arthritis patients are receiving TNF-inhibitor therapy worldwide, making an income of about $15 billion a year for the two or three companies involved.

Presumably the shareholders are doing well and enjoying their holidays in the south of France. These holidays come at a cost borne by the unfortunate patients whose health systems cannot afford to pay for these drugs. Capitalism does not sit well with the precepts of the Hippocratic oath.

Putting aside these ethical issues, we should consider whether TNF-inhibitors should be funded by Pharmac, the Government's pharmaceutical watchdog, which has managed, with great difficulty, to keep some sort of control on drug spending.

Taxpayers fund Pharmac and need to be aware of its decisions. In this case, funding for these new drugs should go ahead as soon as possible. Although they are enormously expensive, only a small number of patients would need such therapy.

The Rheumatology Association estimates that between 300 and 400 people would fit stringent criteria (as in Australia and Britain) for arthritis severity and lack of response to other drugs. The total cost to the country for this group would be $6 million to $8 million a year.

This needs to be contrasted with the cost these patients already incur. Standard intensive drug therapy can cost up to $7000 a year. On top of that are costs for hospitalisation often required by these people ($600 a day) and of surgery to joints destroyed by arthritis.

One of my patients, who developed rheumatoid arthritis 10 years ago, has had six joint replacement operations (replacement of knuckles, fusion of the wrists, several bouts of surgery to the feet, stabilisation of the spine) at a total cost of more than $60,000, not to mention that she is now permanently on a sickness benefit.

The Rheumatology Association has lobbied Pharmac to make these drugs available to the needy group for whom they may be life-changing. It has gone through the official channels time and time again. It has not been heard or understood.

New Zealand likes to think of itself as a civilised Western nation. A definition of civilisation is that society cares for its most disadvantaged members - the sick and the needy.

It is time these values were adhered to.

* Fiona McQueen is an associate professor in rheumatology at Auckland University.