England's young cystic fibrosis patients face new treatment worry

[November 07, 2023]  By Andy Bruce

MANCHESTER, England (Reuters) - Young English sufferers of cystic fibrosis (CF) face new uncertainty after regulators published proposals which, if enacted, would restrict access to potentially life-changing treatments on cost grounds.

The National Institute for Health and Care Excellence (NICE) on Friday published draft guidance that it would not recommend three treatments known as modulator therapies, manufactured by Vertex Pharmaceuticals for use on new patients.

The draft guidance, open for consultation until Nov. 24, would not affect those who already take Kaftrio or Symkevi, which are currently recommended for patients aged 6 and over, or Orkambi, which can be used at age 1 and over.

If passed, young children or babies who are yet to receive these therapies would not be guaranteed them under National Health Service (NHS) care, despite NICE's view that there are major benefits for CF patients.

Cystic fibrosis is one of the world's most common lethal inherited diseases, affecting around 100,000 people globally. Patients' lungs become filled with thick sticky mucus and they are vulnerable to recurrent chest infections, which eventually destroy the lungs.

"Even when considering the condition's severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates ... are above the range that NICE considers an acceptable use of NHS resources. So, they are not recommended," the NICE report said of the Vertex therapies.

The Vertex CF treatments - the first to address the causes rather than just symptoms of the disease - were hailed as game changing, life-extending advances when approved.

Kayley Dempsey from Manchester, whose four-year-old son has cystic fibrosis and takes Orkambi, told Reuters that the proposals would generate fear and uncertainty for families. She had not expected Britain could go backwards in terms of medical access.

"When the news came that we would get Orkambi... we were over the moon, I cried with happiness," she said.

A sign hangs in front of the world headquarters of Vertex Pharmaceuticals in Boston, Massachusetts, U.S., October 23, 2019. REUTERS/Brian Snyder/File Photo

The modulator therapies have been used in the NHS since 2019 when it struck a deal with Vertex that would allow clinical data to be collected in the real world, ahead of a formal appraisal by NICE.

The NICE draft guidance underlines the strain on resources and finances faced by the health service, with waiting times in Britain soaring - an increasing political but also economic problem.

NICE said a 56-tablet pack of Kaftrio costs 8,346.30 pounds ($10,337.73), excluding VAT, and 6,293.91 for a 28-tablet pack of Symkevi.

"Vertex is disappointed with the draft NICE appraisal guidance issued about our CFTR modulators that treat the underlying cause of cystic fibrosis," the company said in a statement.

"We believe that the committee has not fully considered all of (the) real-world evidence and we will be providing additional information and analysis to be taken into account," Vertex said.

The company also said NICE had under-estimated the long-term costs of caring for people with CF.

John Stewart, national director for specialized commissioning at NHS England, said the health service would continue to work to make the life-changing medicines available, now and in the future.

"(We) will continue to work with Vertex and NICE through the next stages of the appraisal process to make this possible in a way that is fair to patients, and fair to taxpayers," he said.

($1 = 0.8074 pounds)

(Reporting by Andy Bruce; Editing by Bill Berkrot)

[© 2023 Thomson Reuters. All rights reserved.]This material may not be published, broadcast, rewritten or redistributed.  Thompson Reuters is solely responsible for this content.