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23 March 2016

NICE consults on draft guidance for new cystic fibrosis treatment

NICE – the National Institute for Health and Care Excellence – has issued draft guidance which does not recommend Orkambi (lumacaftor-ivacaftor marketed by Vertex Pharmaceuticals) for treating cystic fibrosis.

The independent appraisal committee concluded that the cost of Orkambi was considerably higher than the current standard of care and it could not be considered a cost effective use of NHS resources.

Cystic fibrosis is an inherited condition where a gene defect causes a person’s lungs and digestive system to become clogged with sticky mucus. This leads to blockages that damage the body’s tubes and passages. Symptoms include persistent cough, recurring infections and malnutrition.

There is currently no cure for cystic fibrosis and current treatments aim to manage individual symptoms.

Orkambi is licensed to treat people who have a specific genetic defect known as the F508del mutation. There are around 2,750 people in England with this genotype. It costs £104,000 per patient for every year of treatment. Most people suitable for Orkambi would also still remain on the current standard of care whilst taking it.

The committee acknowledged that Orkambi does offer benefit because it reduces instances where people experience a sudden worsening of symptoms that requires hospitalisation. However, the benefits to lung function – the test to see how people with cystic fibrosis are improving overall – were modest.

Commenting on the draft guidance, Professor Carole Longson, director of the NICE Centre for Health Technology Evaluation said: Orkambi is a new treatment option and it is disappointing that we are not able to recommend it. However our independent committee found that when compared to the current standard of care, the benefit it offered was modest and comes at a considerable cost. We have to recognise that the NHS is a finite resource and we can only recommend treatments for routine funding that are both clinically effective and represent good value for money.”

Consultees, healthcare professionals and members of the public, are now able to comment on the preliminary recommendations which are available for public consultation until Friday 15 April 2016. Comments received during this consultation will be considered by the committee at a further appraisal meeting before the next draft guidance is issued. 

Until final guidance is issued, NHS organisations should make decisions locally on the funding of specific treatments.


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Notes to Editors

Further info

  1. According to NHS Choices around 1 in every 2,500 babies born in the UK will have cystic fibrosis and there are thought to be more than 9,000 people living with the condition.
  2. The draft guidance will be available at /guidance/indevelopment/gid-tag530.
  3. The committee discussed the clinical evidence from the TRAFFIC, TRANSPORT and PROGRESS trials. The acute improvements in ppFEV1 seen with lumacaftor–ivacaftor were modest and unlikely to be clinically significant. The reductions in pulmonary exacerbations seen with lumacaftor–ivacaftor treatment were clinically significant and important for the management of cystic fibrosis.
  4. The committee considered the company's economic model but concluded that the price reduction applied to lumacaftor–ivacaftor after 12 years was not appropriate. The committee acknowledged that when the company’s arbitrary price reduction was removed, the company’s base-case ICER increased from £218,000 to £349,000 per QALY gained for lumacaftor–ivacaftor plus standard of care compared with standard of care alone. The committee concluded that, even without including all of its preferred assumptions, the estimated ICERs were considerably higher than what is normally considered a cost-effective use of NHS resources.
  5. The cost of lumacaftor–ivacaftor is £8,000 per 112-tablet pack (excluding VAT; company's evidence submission). The cost of a 1-year course of treatment is £104,000 (excluding VAT).
  6. The Scottish Medicines Consortium is due to issue it guidance on this drug in May:

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However our independent committee found that when compared to the current standard of care, the benefit it offered was modest and comes at a considerable cost.

Professor Carole Longson, director of the NICE Centre for Health Technology Evaluation