THE families of cystic fibrosis (CF) patients protested in London on November 19 to mark three years to the day since precision drug Orkambi was licensed for use in the UK.

Now they have launched a new online petition, urging the NHS to supply new lifesaving drugs for CF like 11 other EU countries.

Hartley Wintney teenager Elizabeth Sheahan has been at the forefront of the campaign, which began by targeting international drugs company Vertex Pharmaceuticals, which they have been pushing to agree a price “the NHS can afford” so people like her can access Orkambi – a drug that could prolong her life.

Only available on the NHS on compassionate grounds, Orkambi currently costs £100,000 a year for each patient – and is considered too expensive for general use.

But for 15-year old Elizabeth, it could afford her the time to enable her to go to Oxford and fulfil her dreams.

Orkambi is a precision medicine that nearly half the 10,000-plus people in the UK with cystic fibrosis (CF), including children aged six or over, could benefit from.

While conventional CF treatments target the symptoms of the disease, precision medicines such as Orkambi tackle the underlying genetic defects that cause the condition.

Orkambi received its European licence on November 20, 2015, and in June the following year the UK government agency the National Institute for Health and Care Excellence (NICE) recognised Orkambi as an important treatment.

However, NICE was unable to recommend Orkambi for general use on grounds of cost-effectiveness and a lack of long-term data.

Now an investigation has been launched by the Health and Social Care Committee, which has formally requested documents from all interested parties with regards to the ongoing negotiations for the provision of Orkambi – and other drugs for the treatment of cystic fibrosis – on the NHS.

As part of the campaign Elizabeth joined fellow CF sufferer Luis in filming a video, literally begging for their lives.

Their message to the NHS and politicians was clear: “You have the chance to save somebody’s life – and you can’t get back a life once they’ve gone.”

Orkambi campaigner Susan Barnes said: “I spent an afternoon filming with Elizabeth – she is an amazing, vivacious young woman and society would be much the poorer for losing someone like her, either to ill health or the inevitable consequences of cystic fibrosis.

“We will never give up fighting for precision medicines like Orkambi that can transform the lives of many cystic fibrosis patients. We will certainly never give up fighting for our loved ones to live a longer, healthier life.”

At the beginning of November Symkevi, another precision CF medicine, was licensed for use in Europe.

Symkevi has been shown to be effective in a far wider group of patients and the CF community is keen to broker a‘portfolio deal’, such as the one agreed last year in Ireland, to open up access to new medicines, like Symkevi, as they become available.

“Despite almost three years of campaigning, it feels as though we are no closer to gaining access to these life-saving drugs” says Gemma Weir of the CF Trust community.

“The CF community entered 2018 with huge determination to step up our campaigning activity and finally get access to a drug that is already available in many other countries.”

In January, the CF community’s Orkambi petition attracted 119,000 signatures, which led to a government debate in March.

A protest march in June resulted in an emotional confrontation with key Vertex personnel.

But, says Gemma: “It seems Vertex and NHS England are still miles apart in terms of agreeing a price for CF drugs.”

The latest petition has already attracted 23,205 signatures. It can be found at https://petition.parliament.uk/petitions/231602 .