‘Landmark moment’ as CF patients in Scotland get access to Orkambi drug

Hundreds of cystic fibrosis sufferers in Scotland will be able to get drugs which can help extend their life, after a deal on costs was reached.

Pharmaceutical firm Vertex International has reached a pricing agreement with the Scottish Government – and said as a result approximately 400 patients would be able to benefit from Orkambi and Symkevi.

The drugs will be made available for five years with a “confidential discount”.

It comes just a month after the Scottish Medicines Consortium (SMC) – the body which approves drugs for use by the NHS in Scotland – rejected the treatments, saying that the “cost in relation to its health benefits was not sufficient”.

The cost of Orkambi – reported as being £100,000 a year per patient – means in England the National Institute for Health and Care Excellence (Nice) has already deemed it not cost effective.

David Ramsden, chief executive of the Cystic Fibrosis Trust, hailed the access agreement in Scotland as a “landmark moment for the hundreds of people with cystic fibrosis and their families across Scotland who have tirelessly campaigned for years to access these drugs”.

He said: “This breakthrough is a victory for their perseverance and enduring hope. It means 350 eligible people living in Scotland will have access to drugs that stabilise their lung health and reduce the need for hospital admissions.

“We celebrate the news in Scotland today, but our campaign must continue to focus on Vertex and all parties in England, Wales and Northern Ireland.

“Those in need of the drugs have already waited too long and we must ensure that thousands more people are not subjected to a postcode lottery. Scotland’s success must now be replicated across the UK without further damaging delay.”

Cystic fibrosis is a debilitating life-shortening illness that creates a mucus build-up that causes chronic lung infections and progressive lung damage.

Orkambi is used to treat patients aged two and older while Symkevi treats those aged 12 and up, and while the drugs are not a cure for the disease, they can slow the decline in lung function, which is the main cause of death.

Scottish Health Secretary Jeane Freeman said: “This is fantastic news for those living in Scotland with cystic fibrosis and their families. This new pricing agreement with medicines manufacturer Vertex Pharmaceuticals, secured by the Scottish Government, will allow more people with cystic fibrosis to benefit from medicines Orkambi and Symkevi and live fuller lives for longer.”

She added: “The agreement has been reached after extensive discussions between the Scottish Government and Vertex Pharmaceuticals and means the medicines will now be made available to patients on the NHS in Scotland, subject to a confidential discount.”

As part of the five-year access agreement Vertex International will collect data on the effectiveness of the drugs, which will be used in future submissions to the SMC.

Ludovic Fenaux, senior vice president at Vertex International, said: “We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way that we have worked together to find this access solution.

“It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to Orkambi or Symkevi.”