Monday 23 September 2019

Rare drug 'too expensive' here but not in nine other EU states

CRUCIAL: FG TD Pat Deering wants drug criteria changed. Photo: Tom Burke
CRUCIAL: FG TD Pat Deering wants drug criteria changed. Photo: Tom Burke
Wayne O'Connor

Wayne O'Connor

The Government's drug treatment assessment process was criticised after a drug available in nine other EU countries to treat a rare genetic disorder was deemed too expensive for use here.

TDs are calling on the Government to change how drugs are deemed cost-effective, saying present measures only favour treatments for more common complaints.

Last month, the NCPE ruled that Kuvan, a drug used to treat the rare genetic disorder Phenylketonuria, was not cost-effective. People with Phenylketonuria (PKU) are unable to break down an amino acid in food. This can cause serious health problems, including, if untreated, severe brain damage.

According to the PKU Association of Ireland, one in every 4,500 babies here is diagnosed with PKU, a significantly higher rate than the UK, where approximately one in every 12,000 babies is diagnosed with PKU.

Kuvan has been assessed by the National Centre for Pharmacoeconomics (NCPE) twice in 2009 and 2017 and rejected on both occasions.

Fine Gael TD Pat Deering said the NCPE's assessment system as it stood only suited treatments for prominent diseases and did not help people suffering with a rare illness.

"I am not looking for the minister to overrule the decision. I am asking him to change the assessment process because this drug will never succeed in being passed under the assessment process at present," said Mr Deering.

"The assessment process at present only suits tests relating to oncology or other more prominent diseases. This is a rare disease and the structure and the criteria that are there will never allow this drug to be used, which is the crucial point."

The PKU Association of Ireland has urged the HSE to engage with Kuvan manufacturers to approve funding for the drug. It said it was the only drug available to treat the disease and it could bring about life-changing results for PKU sufferers.

Children and adults with PKU must limit their intake of meat, fish, poultry, dairy products, bread, chips, pasta and chocolate. This diet can lead to a higher risk of suffering from attention deficit disorders, poor concentration and low energy levels. It can also lead to nourishment issues. These dietary requirements also affect irritability and can lead to stomach pains.

The disorder can be discovered with a heel prick test shortly after a child is born. There are more than 700 people being treated for the disease in Ireland.

Junior Health Minister Jim Daly said his department aimed to secure access to new and innovative drug treatments. However, this had to be done in a cost-effective way, he added.

Kuvan was considered under the national pricing and reimbursement processes in 2009 but insufficient evidence was available to deem it cost-effective. Biomarin, the company that owns the European patents for the drug, submitted a new dossier to the HSE for assessment last year.

"The NCPE assessment of the Biomarin dossier was completed on September 15. The NCPE did not recommend Kuvan for reimbursement as it was not deemed to be cost-effective," said Mr Daly.

"The HSE assessment process is ongoing and the HSE will take into account the expert advice of the NCPE when making its decision in line with the Health (Pricing and Supply of Medical Goods) Act 2013."

People Before Profit TD Gino Kenny called on the Government to override the decision that the drug was not cost-effective. He said the NCPE structures also worked against patients who required treatments for other illnesses and complaints.

"What this comes down to is putting a price on somebody's life," said Mr Kenny.

"This goes further than providing Kuvan for people with PKU; it involves other orphan drugs like Translarna for people with muscular dystrophy."

Sunday Independent

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