Cystic fibrosis patients are unlikely to get access to Vertex’ Orkambi on the National Health Service in England and Wales after cost regulators rejected the therapy’s use despite its ability to reduce hospitalisations. 

Orkambi (lumacaftor/ivacaftor) is the first drug for CF directed at treating the cause of the disease in people who have two copies of the F508del mutation, accounting for around 45% of cases. This mutation causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.

Data from the Phase III TRAFFIC and TRANSPORT studies show that the therapy significantly improved lung function (as measured by FEV1) by 2.6-4.0 percent, and reduced the rate of pulmonary exacerbations by 30-39 percent in target CF patients. 

But the National Institute for Health and Care Excellence has now issued draft guidelines rejecting its use, after its appraisal committee concluded that Orkambi’s cost was “considerably higher” than the current standard of care, and that it could not be considered a cost effective use of NHS resources.

There are around 2,750 people in England who carry two copies of the gene, and Orkambi’s list price is £104,000 per patient for every year of treatment. Moreover, most people suitable for treatment would also still remain on the current standard of care whilst taking it.

The committee acknowledged that Orkambi does offer benefit in reducing instances where people experience a sudden worsening of symptoms that requires hospitalisation, but said benefits to lung function - used to measure how patients with  cystic fibrosis are improving overall - were modest.

Too expensive

After removing an arbitrary price reduction (after 12 years’ treatment) included in the company’s base case estimate, the incremental cost-effectiveness ratio (ICER) increased from £218,000 to £349,000 per QALY gained for Orkambi plus standard of care versus standard of care alone. Even without including all of the committee’s preferred assumptions, the estimated ICERs were considerably higher than what is normally considered a cost-effective use of NHS resources. 

“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,” said Carole Longson, director of the NICE Centre for Health Technology Evaluation, commenting on the decision.