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

More patients with the life-limiting disease cystic fibrosis are to receive new treatment Kalydeco, after NHS England agreed to fund the drug.

But charity the Cystic Fibrosis Trust has hit out at the slow decision-making from health service chiefs at NHS England, saying quicker rulings are needed for groundbreaking drugs.

Vertex’s Kalydeco (ivacaftor) can dramatically improve lung function and reduce hospital admissions, leading to hopes it could normalise life expectancy – but only a subset of patients with certain gene mutations have been shown to benefit so far.

Like so many drugs for rare diseases, Kalydeco is very expensive – it costs £182,000 a year per patient, who will have to keep taking the drug for the rest of their lives.

NHS England first approved the drug for patients with the G551D mutation in 2013, and has now extended that to patients with eight rare non-G551D gating mutations – just 40 patients in England.

The charity says these patients have had to wait a whole year for the decision following European approval for the expanded use.

Ed Owen, chief executive for the Cystic Fibrosis Trust, said: “This is a long overdue but welcome decision which should help to transform the lives of dozens of people with cystic fibrosis.

“But this delayed decision illustrates why the system to fund transformational therapies of this sort is flawed and needs to change.”

NHS Wales has yet to agree funding for the expansion, while there are no currently no cystic fibrosis (CF) patients carrying the eight mutations living in Scotland or Ireland.

Ed Owen added: “We also need this decision to be made in Wales as quickly as possible. It would be unacceptable for people in one part of the UK to have access to life-changing treatments available to others elsewhere.

“The Cystic Fibrosis Trust will continue to fight for the right of children and adults living with CF who are set to benefit from new therapies in the near future. We will not stop until the system, which is currently obstructed by bureaucracy, is made logical, transparent and fair.”

New drug Orkambi around the corner

The NHS England announcement has coincided with news that the US has just approved Vertex’s next cystic fibrosis treatment, Orkambi. The drug is a combination of ivakaftor and a new molecule, lumacaftor, and will mean many more people with CF can be treated. The combination treats patients who have two copies of the F508del gene, around 50 per cent of the UK CF population.

Illustrating the confusion around how new specialist drugs are approved in England, Orkambi is due to be reviewed not by NHS England, but by NICE. Orkambi is expected to gain European approval later this year, after which point Vertex will give public indications about its price.

In the US, the price of the drug has been announced as $259,000, less than the $300,000 it charges for Kalydeco – a lower price it can afford thanks to the larger patient population.

NHS England’s spending decisions

The decision on Kalydeco is just one of a raft of new spending commitments that NHS England announced yesterday in specialised services and treatments.

The NHS is under huge pressure to meet demand for new drugs, surgery and other treatments, but its leaders have warned that its funds will have to be rationed.

James Palmer, NHS England’s Clinical Director for Specialised Commissioning warned the pharma industry earlier this week that market access for its products would inevitably become harder over the next five years.

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