Bobby Aylward (Carlow-Kilkenny, Fianna Fail) | Oireachtas source

The HSE drugs group, in its decision not to reimburse Vimizim, has on both occasions stated that there is a lack of clinical evidence on the effectiveness of the drug. They have acknowledged that the budget impact to cover the cost was relatively low. The drugs group itself agreed that cost was not an issue. When we talk about drugs we worry mostly about the cost. The drug is available in most of the European Union, including Northern Ireland, our neighbours, and is actually manufactured in Ireland. The manufacturing company applied again to the HSE drug group with new evidence late last year after the drug was rejected at the September meeting. This evidence was largely from the managed access agreement that the company are running in the UK, which the UK accepted. If the clinical evidence has proven acceptable in other countries and the HSE has acknowledged that the cost is not the primary barrier to reimbursement, then the drug should be made available to those who need it.

In respect of the cost, there are figures in the public domain which indicate a very high costing for the use of the drug per patient. These figures are factually incorrect as we are dealing with a weight-based drug, which means that the publicly quoted amount is substantially higher than what the price per patient would be because they are small and the weight per patient means less cost. It is also worth bearing in mind that in the decision not to reimburse the drug the cost was not cited as a significant factor in the decision.

In respect of the managed access agreement, BioMarin has an arrangement in the UK with the National Institute for Health and Care Excellence, the NHS and the patient group where patients who suffer from Morquio A syndrome sign a contract which states they must attend all medical sessions and that, if after a year the medical records note that there has not been an improvement in their circumstances, they agree to come off the drug. In England, if the drug does not work, there is an agreement to stop using it if it is not having any effect. This has the benefit of ensuring that patients who do benefit from the drug can use it and that those who do not discontinue treatment, ultimately ensuring that the costs associated with the drug are solely associated with those patients who benefit from the drug. Clinicians who currently work in this space in Ireland have drawn up a similar guideline in Ireland and the patients are also willing to engage on this basis going forward.

In respect of the HSE drugs group challenges, the decision-making process is non-transparent which leaves the patients and the pharmaceutical company in the dark. The current nature of the drugs reimbursement process as a whole is unsustainable. There are problems associated with the quality adjusted life year based analysis for rare and orphan drugs.

There are two little girls looking down on the Minister of State here today. Next Friday, they will not have this drug, which has helped them over the past four years. Will the Minister of State please, on humanitarian grounds, bring this issue back to the task force and explain the situation? It will involve only a small cost to the taxpayers of this country.

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