NICE rejects Vertex’s £104,000 cystic fibrosis combination

NICE has rejected Vertex’s Orkambi (lumacaftor+ivacaftor) cystic fibrosis drug in draft guidance, saying that its £104,000 per year per patient price tag was too expensive for the National Health Service.

Orkambi is licensed to treat people aged over 12 years who have the F508del mutation. There are around 2,750 people in England with this genotype.

A NICE independent committee acknowledged Orkambi does offer benefit because it reduces instances of sudden worsening of symptoms requiring hospitalisation. However it found that the benefits to lung function were modest.

Vertex had applied an “arbitrary” price cut, NICE said. When the company’s price reduction was removed, Vertex’s base case incremental cost-effectiveness ratio increased from £218,000 to £349,000 per quality adjusted life year gained for Orkambi plus standard of care, compared with standard of care alone.

This places Orkambi well above NICE’s cost-effectiveness threshold of £20,000-£30,000 per QALY gained.

Orkambi combines the new agent lumacaftor with ivacaftor, aka Kalydeco, Vertex’s first CF treatment. Launched in the UK in May 2013, funding for Kalydeco was swifted granted by NHS England, and the drug was never appraised by NICE.

Strikingly, Orkambi actually costs less than Kalydeco, which is priced at £182,000 a year.  Vertex’s logic is that as Orkambi can treat a larger CF population, it can afford to give the combination a lower price. But this clearly hasn’t swayed NICE’s committee.

Vertex’s senior vice president and international general manager, Simon Bedson, said the company believes NICE’s single technology appraisal process is not appropriate for assessing rare disease medicines such as Orkambi.

He said: “We are concerned that application of the wrong appraisal process may lead to no access to Orkambi for eligible patients in England, who currently do not have a medicine that treats the underlying cause of their disease.”

Vertex noted that life science minister George Freeman and patient groups have cited Orkambi as a key example of the need for reform in medicines for rare diseases.

NICE will consult on the draft until 15 April, before publishing a second draft. Until final guidance is issued NHS organisation should make decisions on funding.

The Scottish Medicines Consortium is due to issue guidance on Orkambi in May.

Related articles:

More cystic fibrosis patients to get Kalydeco in England, but delay criticised

Orkambi approval in Europe will extend treatment to more CF patients

Alexion: aiming to break new ground in ultra-rare therapies

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