National Institute for Health and Clinical Excellence (NICE)
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Cost of cystic fibrosis treatment too high for benefit offered, says NICE

NICE - the National Institute for Health and Care Excellence – has said it will not be able to recommend Orkambi (lumacaftor-ivacaftor, Vertex Pharmaceuticals) for treating cystic fibrosis.

In March, NICE publicly consulted on draft guidance which concluded that compared to the current standard of care, the benefit Orkambi offered did not justify its considerable cost. Although Orkambi was shown to reduce instances where people with cystic fibrosis are admitted to hospital, the benefits to lung function – one of the tests used to see how people are improving overall – appeared modest in the short term whilst the long-term benefits were uncertain.

Orkambi costs £104,000 per patient for every year of treatment. It is licensed to treat people who have a specific genetic defect known as the F508del mutation. There are around 2,750 people who would be eligible for treatment with Orkambi in England.

In response to the public consultation, the Cystic Fibrosis Trust suggested that additional real-world data could be collected which would demonstrate that Orkambi does offer significant benefits in the long-term. However the committee concluded that if the drug were to be made available to patients on this basis, the company would have to provide Orkambi to the NHS in a cost effective way. Vertex Pharmaceuticals did not put this proposal forward to the NICE appraisal committee to consider.

Professor Carole Longson, director of the NICE Centre for Health Technology Evaluation said“We know how important a new treatment option would be for people with cystic fibrosis; but for the benefits it offers, the cost of Orkambi is too high. We can only recommend treatments when we are certain they are both clinically effective and represent good value for money. If the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it.”

NICE has now issued further draft guidance to consultees who have the opportunity to appeal against the decision. If there are no appeals, the decision will be issued as final guidance in July.

Once NICE issues final guidance, companies are still able to submit a new cost-effective proposal which demonstrates their drug is cost-effective. This will be considered under a rapid review process.

Notes to Editors

Further info

  1. The draft guidance is available at /guidance/indevelopment/gid-tag530.
  2. Lumacaftor–ivacaftor has a marketing authorisation in the UK for treating cystic fibrosis in people 12 years and older who are homozygous for the F508del mutation (that is, have 2 copies of the mutation) in the CFTR gene.
  3. The committee discussed the clinical evidence from the TRAFFIC, TRANSPORT and PROGRESS trials. Although the acute improvement in ppFEV1 was modest, when combined with the improvement in rates of exacerbations, the clinical trials provide evidence that lumacaftor–ivacaftor may significantly improve the long-term outcome for patients. The committee concluded that longitudinal changes rather than acute changes in ppFEV1 were more clinically relevant for assessing long-term outcomes of cystic fibrosis, and both the observed and extrapolated benefits of lumacaftor–ivacaftor on ppFEV1 were taken into account in the company’s cost-effectiveness analysis.
  4. 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).
  5. The committee considered the company's economic model but concluded that the price reduction applied to lumacaftor–ivacaftor after 12 years (assuming the introduction of a future low-cost generic) 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 any of its preferred assumptions, the estimated ICERs were considerably higher than what is normally considered a cost-effective use of NHS resources.
  6. The Scottish Medicines Consortium issued guidance in May which does not recommend Orkambi:https://www.scottishmedicines.org.uk/SMC_Advice/Advice/1136_16_lumacaftor_ivacaftor_Orkambi/lumacaftor_ivacaftor_Orkambi

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