Scottish Cystic Fibrosis sufferers finally get access to life-extending drug Orkambi

12 September 2019, 17:53

Orkambi, a life-extending drug for those living with cystic fibrosis, has been made available to patients in Scotland
Orkambi, a life-extending drug for those living with cystic fibrosis, has been made available to patients in Scotland. Picture: PA

By Kate Buck

Drugs that could transform the lives of cystic fibrosis sufferers will be available to patients in Scotland after a deal was struck on the cost.

The Scottish government has announced a five-year pricing agreement with Pharmaceutical firm Vertex International, who make Orkambi and Symkevi.

The medicines cost £100,000 per patient each year, but the drug maker has said they will be provided at 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 the "cost in relation to its health benefits was not sufficient".

The cost of Orkambi means that 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".

Patients in England still do not have access to the drugs
Patients in England still do not have access to the drugs. Picture: PA

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 which creates a mucus build-up that causes chronic lung infections and progressive lung damage.

Orkambi is used to treat patients aged two and above, while Symkevi treats those aged 12 and up.

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.

Ludovic Fenaux, vice president at Vertex, 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."

A spokeswoman for the UK Government's Department of Health and Social Care said: "We always follow clinical advice on what medicines should be made available to patients, ensuring they provide value for money for the NHS and taxpayers.

"NHS England has put forward a very generous offer, the largest in its 70-year history, that would make Orkambi available to NHS patients in England and we urge Vertex to accept the deal so patients can benefit from treatment as soon as possible.

"The Health Secretary will be bringing in Vertex representatives to explore what the barriers are to reaching an agreement."