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NICE’s draft guidance against use of Vertex therapies may upset new cystic fibrosis patients

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Patients who are already using the Vertex therapies can continue to use them, the guideline is open for consultation until 24 November

Modulator therapies, manufactured by Vertex Pharmaceuticals (VRTX.O), may no longer be available for new cystic fibrosis (CF) patients in the UK.

A draft guidance recently published by the National Institute for Health and Care Excellence (NICE) stated that it would not recommend the use of Kaftrio, Symkevi and Orkambi on new CF patients.

Kaftrio and Symkevi are currently recommended for the treatment of cystic fibrosis in patients aged 6 years and older, while Orkambi is used for patients aged 1 and older.

The NICE draft guidance highlighted medicine costs and the strain on resources and finances faced by the National Health Service (NHS) as reasons for removing these drugs.

The guidance stated: “Even when considering the condition’s severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates for IVA–TEZ–ELX, LUM–IVA and TEZ–IVA are above the range that NICE considers an acceptable use of NHS resources. So, they are not recommended.”

The list price of a 56-tablet pack of Kaftrio is £8,346.30, £6,293.91 for a 28-tablet pack of Symkevi and £8,000 for a 112-tablet pack of Orkambi (excluding VAT).

However, the NICE’s draft guidance will not affect patients who are already using the Vertex therapies.

The proposals, if enacted, would restrict the use of these therapies on new patients under National Health Service (NHS) care. The guideline is open for consultation until 24 November.

Cystic Fibrosis is a common inherited condition that affects around 100,000 people worldwide. In the UK, it is estimated to affect about one in every 2,500 newborn babies.

The lungs of those with the condition become filled with thick sticky mucus, which makes them vulnerable to recurrent chest infections, and breathing and digestion problems.

Over the years, the affected lungs become damaged and may eventually stop working.

The Vertex drugs “treat the underlying cause of cystic fibrosis” rather than just addressing symptoms of the disease. The drugs, when approved, were hailed as game-changing, life-extending advances.

In 2019, the NHS signed a deal with Vertex and included these drugs in its prescription list.

The deal allowed the pharmaceutical company to collect clinical data in the real world, ahead of a formal appraisal by NICE.

Vertex has expressed disappointment with the draft NICE appraisal guidance issued about their drugs.

Reuters cited a statement from the company that read: “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.”

Further, the company stated that the long-term costs of caring for cystic fibrosis patients are being underestimated by NICE.

However, John Stewart, national director for specialised commissioning at NHS England, told the publication said the health service would continue to work with Vertex and NICE to make the medicines available “in a way that is fair to patients, and fair to taxpayers.”

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