Drug maker 'committed' to work with NHS

Telford | News | Published:

The maker of a drug which campaigners – including a Telford mother – are calling to be made available on the NHS says it is committed to working with the health authority to find a sustainable funding solution.

Emma Hughes, of Arleston, with her son Chester who has cystic fibrosis

Emma Hughes is one of those calling for Orkambi to be made widely available to help people battle cystic fibrosis (CF) and welcomed an upcoming parliamentary debate on the issue.

Her two-year-old son Chester has been diagnosed with the condition and even though he has a type of CF that means that he is unlikely to benefit, Mrs Hughes said she would welcome any progress to help others.

A spokesman for Vertex, the maker of Orkambi, today said: "We share the CF community’s sense of urgency on access to new Vertex medicines and we are committed to working with the NHS to find a sustainable funding solution.

"That is why we have proposed to the Government a bold new portfolio approach that could make our medicines available to patients as soon as possible.

"It provides budget certainty and value to the NHS − and offers fair and equal access for CF patients to precision medicines that are tailored to specific genetic mutations.

“The proposal follows the recent announcement that two of our triple-combination next generation correctors are going forward into phase III clinical trials.

"At Vertex we are developing medicines to treat 90 per cent of CF patients who will one day be eligible for treatment able to treat the underlying cause of the disease.

“We believe that our approach is a first for England, and a first for the UK. We have already demonstrated it works in other countries."


Mrs Hughes, aged 36, of Arleston, says Orkambi offers a new hope of a cure for sufferers.

She believes that others drugs from the same manufacturer could help her son in the future.

Chester was diagnosed with the condition when he was a week old and spent much of his first year in and out of hospital.

He spent the first three months of life in Birmingham Children’s Hospital, has had three unplanned two-week IV antibiotic admissions for infection, a bronchoscopy to check for infection and attends clinics frequently at Princess Royal Hospital in Telford.


The National Institute for Health and Care Excellence announced a decision to reduce the use of the drug on the NHS because of uncertainty around long-term value and impact, and therefore cost-effectiveness.

A petition calling for Orkambi to be made available on the NHS was signed by more than 107,000 people in the space of just 11 days.

CF is one of the UK’s most common life-threatening inherited diseases.

It is caused by a defective gene and, as a result, the internal organs, especially the lungs and digestive system, become clogged with thick sticky mucus resulting in chronic infections and inflammation in the lungs and difficulty digesting food.

Orkambi is a revolutionary medicine that can tackle the root cause of cystic fibrosis in certain sufferers.


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