Exclusive: cystic fibrosis drug Orkambi to be rejected by HSE

Cystic fibrosis drug Orkambi will be rejected for use by the HSE. A formal announcement is imminent after the HSE’s drugs committee recommended against funding the drug at a recent meeting.

The committee, which includes various senior clinicians, decided it did not deliver enough benefits to patients to justify its €159,000 annual price tag.

Under the assessment process, the HSE directorate makes the final call. But it will not overturn the decision of the HSE drug’s committee, which was unanimous in recommending against funding the drug.

The decision will be a huge disappointment to patients with cystic fibrosis, a genetic lung disorder. Some 505 people in Ireland have a form of cystic fibrosis that could benefit from the drug, which is the first that treats the underlying cause of the disease for this cohort of patients.

Cystic Fibrosis Ireland dubbed the drug a “game-changer”, and said it would extend the lives of sufferers who rarely live beyond their 40s.

Rejection of the drug is politically sensitive and is likely to heap pressure on Minister for Health Simon Harris, who has already said that he would not overrule clinical decision-making.

In a previous interview with this newspaper, he said: “If you’re asking me whether I am going to fund drugs that experts tell me don’t give a substantial benefit, I won’t.”

Under new rules, the cabinet must approve the use of expensive drugs above a certain cost threshold. But the cabinet is only supposed to get involved if the HSE determines that a drug should be funded.

The state’s medicines watchdog, the National Centre for Pharmacoeconomics (NCPE), assessed Orkambi some months ago.

In its written assessment, the NCPE said that the manufacturer Vertex had “failed to demonstrate the cost-effectiveness of Orkambi” and that the five-year budget impact to the HSE would be €391 million over five years.

Professor Michael Barry, who heads up the NCPE, has said said the drug was “minimally effective”, yielding an average improvement in lung function of approximately 3 per cent. He questioned whether that was clinically significant. Cystic Fibrosis Ireland has said it sustains people’s health and slows their decline.

The HSE and Vertex Pharmaceuticals entered into price negotiations after the NCPE made its decision.

Vertex has failed to strike a deal to have the drug reimbursed by public health systems anywhere in Europe. It was also rejected in Britain.

Some countries including France and Germany facilitated early access programmes pending assessment.

Various doctors in the US have labelled Vertex’s pricing “egregious”.