Price of CF drug may be health cuts elsewhere

Kalydeco, at the original price quoted by its manufacturers, came in at 10-20 times this threshold; a deal approved by the Minister won’t do much to change that multiple. It is off the spectrum, and there doesn’t seem to be much point in having an assessment system if it’s going to be disregarded.

Besides, why the rush? The decision, and the negotiations involved, were supposed to take a few months, but have been finalised within a few weeks. The marginal benefit to patients has to be set against the long-term aim of securing a fair price. There is a broader point to be made here in relation to the way we approve new medicines. Generally, it takes years to fully assess the efficacy and safety of a drug. However, pharmaceutical companies need to get their products on the market as quickly as possible to maximise profits before their patent runs out. Sometimes it makes sense not to withhold the benefits of a new drug from patients who clearly need it. Kalydeco has clearly been on the fast track since birth. In the US, the Food and Drug Administration gave approval in three months instead of the normal 10 months. Two clinical studies of the drug, which found it resulted in significant and sustained improvement in lung function in CF patients, lasted just 48 weeks and involved 213 patients, not particularly large numbers. This may be why the the Irish regulator, the National Centre for Pharmacoeconomics gave a less than ringing endorsement: “Whilst ivacaftor [Kalydeco] may represent an innovation for the treatment of patients with cystic fibrosis there are significant uncertainties, including the absence of long-term health outcome data,” it noted.

If Kalydeco were the only new drug coming on stream, there wouldn’t be much of an issue about funding it. But this isn’t the case – other treatments nearing the market are likely to match this price and, in some cases, to exceed it. Some, like the gene therapy treatment Glybera, are projected to cost more than $1 million per patient per year. Kalydeco as currently authorised treats only about 10 per cent of CF patients in Ireland. There is hope that it could be used for more common forms of the disease when used with another drug. That could increase spending on the drug by a factor of five.

Perhaps you can see a pattern here. Perhaps you read in the past about agreements made with the pharmaceutical companies which were supposed to deliver massive savings but never did. This was because the price cuts on older drugs were more than offset by the cost of providing newer on-patent drugs.

With the ink barely dry on the most recent deal with the industry, it seems like the same thing is happening again. That provided for savings of €120 million this year, offset by new drug costs of €70 million. However, already that figure of €70 million is starting to look optimistic.

And if those targets are not met – and let’s face it, the health system doesn’t have a good record here – the result is likely to be cuts in other areas of the service.

PAUL CULLENis Health Correspondent