Cystic fibrosis drugs to cost €650m over 10 years

The Government’s decision to provide potentially life-saving new drugs for more than 900 cystic fibrosis patients is likely to cost more than €650 million over the next decade.

The price agreed by Cabinet – equivalent to the original estimated cost of the new national children’s hospital – includes €400 million on a single drug, Orkambi.

The balance is accounted for by the provision of a second drug, Kalydeco, to small children as well as access to other, as yet undeveloped drugs from its manufacturer, Vertex Pharmaceuticals.

The HSE reached broad agreement with Vertex on the deal earlier this month but it was only at last week’s Cabinet meeting that approval was given. The cost of the deal has not been officially disclosed.

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A national cystic fibrosis treatment programme is to be established by the HSE to manage the State’s investment in the CF drugs and its agreement with Vertex, according to the Department of Health.

This programme, to start next autumn, will be similar to the national hepatitis C treatment programme, which was set up to monitor the provision of life-saving drugs to patients with the disease.

The HSE estimates that up to 740 Irish patients will receive treatment with Kalydeco or Orkambi this year. By 2021, the number of patients receiving treatment could increase to more than 900.

Mixed feelings

The agreement has been warmly welcomed by the cystic fibrosis community and Opposition politicians, but health experts have been more circumspect.

Prof Michael Barry, head of the National Centre for Pharmacoeconomics, which measures the cost-effectiveness of new drugs, described Orkambi as “minimally effective for some patients”.

One-quarter of treated patients could expect to reach a 10 per cent improvement in lung function, he said.

The provision of Orkambi, and Kalydeco for two- to five-year-olds, is expected to start within weeks.

“The Minister for Health, [Simon Harris], has highlighted to the director general of the HSE the importance of the HSE making every effort to ensure patients receive access to their treatments without delay,” a spokeswoman for the Minister said.

The spokeswoman said no new funding was required to pay for the deal this year, while the funding for subsequent years “has been committed to by Government based on the Cabinet decision”.

Vertex and the HSE are meeting this week in relation to the contractual arrangements to facilitate the agreement, a spokeswoman for the company confirmed. She said Vertex hoped to begin supplying treatments to all applicable patients “as soon as possible”.

As well as Orkambi and Kalydeco, the deal will cover other “pipeline” treatments once they receive market authorisation in Europe for relevant patients, she added.

More than 40 other new drugs, with a combined cost of more than €1 billion, are awaiting a decision by the HSE on reimbursement.