Cookie policy: This site uses cookies (small files stored on your computer) to simplify and improve your experience of this website. Cookies are small text files stored on the device you are using to access this website. For more information please take a look at our terms and conditions. Some parts of the site may not work properly if you choose not to accept cookies.

Join

Subscribe or Register

Existing user? Login

Technology appraisals

NICE rejects £104,000 lumacaftor/ivacaftor therapy for cystic fibrosis

The high cost of combination treatment lumacaftor/ivacaftor (Orkambi) means it has not been recommended for use in the NHS in England and Wales for cystic fibrosis (CF), draft guidance from the National Institute for Health and Care Excellence (NICE) says. 

NICE says the product’s price – £104,000 per patient per year – is not justified by the benefits of the treatment.

Carole Longson, director of the NICE centre for health technology evaluation, says: “We know how important a new treatment option would be for people with CF, but for the benefits it offers, the cost of Orkambi is too high.”

The Cystic Fibrosis Trust has written an open letter, published on 17 June 2016, calling for the product’s manufacturer Vertex and NHS England to find a way to make the treatment available to patients. 

The letter says NICE has not seen data which shows that the product reduced the rate of lung function decline over two years by almost half.

The charity adds that NICE is unsure of the treatment’s long-term value and has proposed that patients should be granted access to the drug and the long-term efficacy assessed via the Trust’s CF registry, which records health data on consenting people with CF in England, Wales, Scotland and Northern Ireland. 

Ed Owen, chief executive of the Cystic Fibrosis Trust, says: “It is now vital that NHS England and Vertex sit down and negotiate a means by which this drug becomes available to those who need it while we put in place a system to measure the real-world impact through the CF registry.” 

He adds: “We want to see meaningful discussions underway by the end of July [2016] to design an access solution for Orkambi and we will not sit idly by if this does not materialise.” 

NICE says that final guidance will be issued in July 2016, until which time consultees are able to appeal the decision. It adds that if the manufacturer submits a cost-effective proposal after that time, it will still be considered under rapid review.

Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2016.20201314

Have your say

For commenting, please login or register as a user and agree to our Community Guidelines. You will be re-directed back to this page where you will have the ability to comment.

Recommended from Pharmaceutical Press

  • Basic Pharmacokinetics

    Basic Pharmacokinetics

    A clear and concise basic pharmacokinetics textbook. Shows how to apply the principles to achieve successful drug therapy.

    £54.00Buy now
  • Drugs in Use

    Drugs in Use

    Optimise drug therapy for your patients. These case studies help you bridge the gap between theoretical medicines knowledge and practical applications.

    £43.00Buy now

Search an extensive range of the world’s most trusted resources

Powered by MedicinesComplete
  • Print
  • Share
  • Comment
  • Save
  • Print Friendly Version of this pagePrint Get a PDF version of this webpagePDF

Supplementary images

  • NICE office logo

Newsletter Sign-up

Want to keep up with the latest news, comment and CPD articles in pharmacy and science? Subscribe to our free alerts.