Campaigners unite in cystic fibrosis drug fight

Paul, Cerys and Catherine Upstone NNL-181209-171507001
Paul, Cerys and Catherine Upstone NNL-181209-171507001

The mother of a Brackley teenager who has cystic fibrosis (CF) took her fight for the availability of precision drugs to London this month.

Catherine Upstone travelled to London with dozens of other families and advocates of those living with CF to petition the drug manufacturer, the NHS and members of parliament for a resolution regarding the availability of Orkambi.

Three years ago the drug, known as a precision drug, was approved for use in the UK. One thousand days later the drug is still not being prescribed in the UK as its manufacturer, Vertex, the NHS and the National Institute for Health and Care Excellence (NICE) became embroiled in a financial dispute preventing its access.

People with CF, a condition which causes a build-up of thick sticky mucus in the lungs, cannot meet due to the risk of cross infection so the fight for precision drugs has been taken up by advocates groups across the country.

The London trip was a call to arms on behalf of those with CF, taking direct action, including the handing in of a petition to Vertex by Luis Walker a young CF patient, a community protest and march culminating in a candlelight vigil in Parliament Square.

The overriding community message was that time has run out for people with CF and immediate access to Orkambi is required. Last year, half of all people who died with cystic fibrosis were under the age of 31.

Organiser Sue Barnes said: “The UK isn’t keeping up in providing access to medicines for cystic fibrosis patients.

“We shouldn’t see family members needlessly suffering and, in some cases dying because the NHS and Vertex can’t come to an agreement. We don’t have time to wait.”

The drug is available in many other EU countries in addition to North America and Australia. There are an estimated 10,000 people with CF in the UK and precision medications, which tackle the underlying cause rather than the symptoms, will benefit half of these.

During the time the UK has remained in a stalemate with Vertex, other more advanced drugs have been trialed and approved and the fear is the UK will remain in a constant race to catch-up with medical breakthroughs.

On November 1, another precision medicine in the Vertex pipeline, Symkevi, was licensed in the EU. Symkevi could benefit more than 5,000 children and adults with cystic fibrosis.

David Ramsden, Cystic Fibrosis Trust chief executive, said: “These precision drugs are essential to improve the quality and length of people’s lives.

“Three years of waiting and failed negotiations for access to Orkambi have been unbearable, with emotional and psychological stress for people with cystic fibrosis and those that care and support them.”

Ian Austin MP also sponsored a cross-party parliamentary event to discuss the issues raised by the CF advocates.

Mrs Upstone said: “The event was attended by 63 MPs. It was really useful to engage with them, especially those who were less informed.

“It was an opportunity to share my story and explain the importance of precision medication. However, still three years later thousands of CF sufferers are left suffering needlessly in this county, while in 11 other European countries the outcome for those with CF is much more positive.

“This leaves me feeling frustrated and helpless. We need the leaders of this country to intervene in the negotiations to keep us in line with other countries and the government’s Life Science Industrial Strategy.”