Reilly approves costly CF drug

Minister for Health James Reilly has given his approval for a new cystic fibrosis drug despite concerns expressed by his officials about the claimed benefits of the treatment, the asking price and the effects on funding for other services.

The budget for Kalydeco, which will be used to treat 120 cystic fibrosis patients, is €220 million over 10 years, HSE director designate Tony O’Brien has told the Minister in a letter seen by The Irish Times.

Officials had also warned that approval for the drug might encourage the pharmaceutical industry to demand higher prices for new treatments. With a number of other new high-tech drugs in the approval pipeline, some costing more than €1 million for each patient, HSE sources say they fear this deal could set a precedent.

Mr O’Brien wrote to the Minister on Thursday seeking direction in view of the “profound ethical and financial issues” involved. He had warned that the HSE Drugs Group was concerned this decision “may have implications for the long-term affordability of new technologies and also for other services seeking funding”.

Drugs Group assessment

In its assessment, the group expressed considerable scepticism about the benefits claimed for the drug, which manufacturers Vertex Pharmaceuticals say will extend the life of CF patients by over 29 years. This has not been formally demonstrated and any estimate of the survival benefit was “prone to great uncertainty”, it said.

The group said it had “serious concerns” about the original and the revised pricing proposed by Vertex, which is subject to a confidentiality clause.

The company originally said Kalydeco would be supplied at a cost of €235,000 a patient a year, or €28 million. The National Centre for Pharmacoeconomics recommended against approval at that price and suggested a reduction in price or a cost-sharing arrangement.

Dr Reilly, announcing his decision in Cork yesterday, would only say he had provided funding of “€20 million-plus” for the drug. The annual budget for new drugs is €70 million. “I think this is a very positive story and it’s certainly going to improve the quality of life for 120 Irish citizens with a particular strain of cystic fibrosis,” he said.

Universally welcomed

The Minister’s decision, taken following discussions with the Taoiseach, was welcomed by patients and the political parties yesterday. CF campaigner Orla Tinsley said it offered great hope.

In his letter, Mr O’Brien had pointed out that his officials advised that the decision meant less funding would be available for other services.

Before the deal was done, the HSE Drugs Group had pointed out that the proposed price for Kalydeco is 10-20 times the threshold for cost-effectiveness under normal Irish assessment procedures. It expressed concern that the pharma industry might believe the HSE considers this level of pricing to be “acceptable or reasonable”.However, “on balance, the group felt that society would wish that the medicine be funded, given the possibility of significant survival benefits”.

Kalydeco can be used to treat cystic fibrosis patients with the G551D mutation, present in about 10 per cent of CF patients in Ireland.