Charities Commission Registration CC22512

NZORD commentary on Pharmac's decision to fund Glivec for Chronic Myeloid Leukaemia - Nov 2002

In late October Pharmac approved funding of Glivec, the new therapy for Chronic Myeloid Leukaemia, after a long and public campaign by the patient group. John Forman, Executive Director of NZORD, the New Zealand Organisation for Rare Disorders, looks at the decision and its implications for other costly treatments for rare diseases.

Pharmac's decision to approve funding of Glivec is significant in many ways. But in the first instance, congratulations are certainly due to the Leukaemia and Blood Foundation for the very effective publicity campaign they waged over the past 6 months or more. Jim Hamilton and his team, plus the individual patients who braved the glare of media publicity, can be very pleased with the outcome that richly rewards their efforts.

In all the debate over this drug, Pharmac has been held out as the villain of the piece for failing to allocate the money, or to act sooner on an application that was filed by the pharmaceutical company, Novartis, over a year ago. I agree that Pharmac should be criticised for the delay. It was unacceptable by any measure. But it was not all about resistence, pressure and final victory. There is more to this issue than meets the eye……...

Glivec is one of the first success stories of the modern ability to get precise identification of the genetic defect that causes the disease. Using this knowledge, Novartis produced a targeted drug so precise, that in the phase one trial to study safety and dose levels (most drugs require two further stages of trials before marketing approval), 53 of the 54 trial patients were in total remission. A remarkable outcome for any drug, and a remarkable outcome for a disease that was invariably fatal after about five years or so, and till now only held at bay by drugs with tough side effects for many patients. Glivec is the most effective cancer drug ever produced, and a great herald of further benefits we can expect to see over coming years from similar advances through the Human Genome Project. But at a cost……..

When issues of cost are so significant, Pharmac does have to work within the budget given to it each year by the Government, and will inevitably negotiate with the pharmaceutical companies for the best price it can get. None of us would expect any less of them. We want good drugs available, but we want value for money too. Pharmac applied cost/benefit criteria and said "No, the cost is too high". And so the game of cat and mouse between Pharmac and Novartis continued, and the patients waited, suffered on older less effective treatments, or dipped into their savings…….

Was Pharmac right? I think they took too little notice of the provision in the Act that directs them to get "the best health outcomes that are reasonably achievable from pharmaceutical treatment", and put too much emphasis on the secondary phrase "and from within the amount of funding provided". Let me set aside the latest news of Pharmac's underspend of its budget, and the implications this has for all funding decisions that are pending right now. Assuming this debate is in the context of a fully allocated budget, with Pharmac applying cost/benefit criteria to a life-saving treatment, against the whole range of other treatments on the Pharmaceutical schedule, begs the question of whether such cost/benefit criteria could ever be valid, let alone ethical. But in doing so they were also at risk of offending against some of the significant public law obligations contained in the UN Charters, the Bill of Rights, and the Human Rights Act. These issues of the right to health care and treatment were the matters that NZORD addressed in our submission on the issue, and in the other lobbying we did in the background, in support of the patient group. So what of the outcome……..

Significantly, Pharmac have moved to make Glivec available to all stages of the disease for CML patients, something that a number of other developed countries have not yet done. That is perhaps remarkable in the light of frequent claims that we are denied drugs here that are subsidised elsewhere in the world. And Pharmac have also approved it for another patient group not even included in the Foundation's campaign. Patients with non-operable Gastro-Intestinal Tumors will have the drug available to them also, with every chance of dramatically improved outcomes. So it is clear that while the publicity campaign certainly had an effect, Pharmac also took notice of the evidence of the drug's effectiveness, and in their final decision did act according to their primary directive - to get the best outcomes for patients. They swung some significant savings in the overall drug bill as well, it seems, in their deal with Novartis. We may well have the true market cost of the drug established now, with a willing seller and willing buyer agreed on a price. A nice added bonus. But what other implications…………

The Human Genome Project will undoubtedly produce many new smart drugs that work better with less side effect, but many of them will be very expensive to start with. The development cost of each new medicine is approximately US$800 million, and for rare diseases with a small customer base, that will mean  high end costs. So the ability of patients to benefit from new generation smart drugs may be as dependent on increased drug funding for Pharmac, as on the ability of support groups to wage effective publicity campaigns. If Pharmac and government could be persuaded to see these funds as an investment in the health of the population, rather than a burden on the budget, we may all be better off sooner……..

And for those doubters who question whether high cost drugs are worth it, and shouldn't we be investing in other areas of health instead…... Remember that the most expensive drug ever produced, relative to the costs of its day, was so precious the urine of treated patients was collected and what had passed through their system was refined out for reuse. Today Pharmac pays on average less than 8 cents a tablet for this medicine that saves thousands of lives every year. It is penicillin - proof that investing in the new and best medicines, pays off in lives saved, quality of life, and cost reductions over time.

The overall outcome……… Another rare disease out of the "too-expensive, too-hard, too-bad" basket, a triumph of health gain and evidence of effectiveness over narrow cost considerations, and a significant example of the value and importance of effective political lobbying as a part of the total decision making process.

Final score……… Top marks to the Leukaemia and Blood Foundation. Life for their patient group. And for Pharmac, several demerits for unacceptable delays, but a big tick for an excellent final decision.