Health October 28, 2019
Life-saving drugs for cystic fibrosis to be made available across England

By Francesco Tamilia - Accountable Care Journal

After a long period of negotiation, NHS England and Vertex Pharmaceuticals have reached a compromise agreement to produce the lifesaving cystic fibrosis (CF) drug Orkambi for patients in England.

The agreement was previously caught in a stalemate dating back to 2016, as NICE deemed the originally proposed price too high for the NHS. While details of the new agreement remain confidential, the final annual price of the drug is expected to be much less than the £100,000 originally posed by Vertex. A deal with NHS England was reached with support from the Scottish Parliament, which reached its own agreement with Vertex for Orkambi six weeks ago.

The new agreement will see more than 4,000 people across England gain access to the lifesaving drug and it is hoped that similar deals can be reached in Northern Island and Wales soon.

Cystic fibrosis is a genetic condition that affects around 11,000 people in the UK. It causes the body to produce a thick mucus from the airways, leading to lung problems and often premature death. According to the Cystic fibrosis Trust, one in 25 people carry the faulty gene that causes it without being aware. A person with CF inherits the condition when both parents carry a defective gene and cannot be developed later in life.

Orkambi counters the F508del mutation which causes around 50 per cent of CF cases. The drug will help patients in several ways, including improvement of lung function and reduction of breathing difficulties.

NHS England has said that clinicians will be able to begin prescribing these drugs within 30 days and will be available from children as young as two.

Simon Stevens, NHS England Chief Executive, said that he was “pleased” that Vertex accepted a deal that is fairer for patients as well as for British taxpayers. “It is an important and long hoped moment for children and adults living with cystic fibrosis,” Mr Stevens added.

Also commenting on the announcement, Matt Hancock, the Health Secretary, said the deal is “wonderful news” adding that, “the deal is great value for money for the NHS, and crucially, will improve thousands of lives. It is another reason to be so proud of our NHS. ”

David Ramsden, Chief Executive of the Cystic Fibrosis Trust, said: “This is a very special day and I want to thank people with cystic fibrosis, their families and everyone who has been part of this campaign for their persistence and determination to keep on fighting.

“We will continue to fight for similar access in Wales and Northern Ireland so that all people with cystic fibrosis in the UK are able to benefit from these life-saving drugs. ”


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