[Ip-health] NICE rejects Vertex's £104,000 cystic fibrosis combination

Jamie Love james.love at keionline.org
Wed Mar 23 03:23:32 PDT 2016

We have to end the madness of high prices on drugs for rare diseases.
 Delinkage is the only approach that will do this.



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

Richard Staines

23rd March 2016

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drug in draft guidance, saying that its £104,000 per year per patient price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

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

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

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

Related articles:

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

Orkambi approval in Europe will extend treatment to more CF patients

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


James Love.  Knowledge Ecology International
KEI DC tel: +1.202.332.2670, US Mobile: +1.202.361.3040, Geneva Mobile:
+41.76.413.6584, twitter.com/jamie_love

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