11:56, August 12 32 0 theguardian.com

2019-08-12 11:56:04
Cystic fibrosis drugs rejected for use by NHS in Scotland

Cystic fibrosis patients in Scotland have called for an end to their “tragic wait” after the rejection by an NHS watchdog of two very expensive new drugs that they believe would change the lives of children and young adults with the condition.

The Scottish Medicines Consortium came to the same conclusion as its counterpart in England, saying that Orkambi and another drug, Symkevi, were not cost effective.

Despite hearing “strong evidence” from patient groups about the benefits of the first drugs to treat the underlying causes of the life-shortening disease, the SMC was not persuaded that the high cost – the list price of Orkambi is £104,000 per patient per year – was justified.

There were, said the SMC, “uncertainties in the company’s evidence about the long-term health benefits of both medicines in relation to their costs”.

NHS England has been in negotiations with Vertex, the US maker of the drugs, for several years without any sign of an agreement. The National Institute for Health and Care Excellence (Nice), the equivalent of the SMC, assessed Orkambi and also found it was not cost-effective. Nice suspended its assessment of Symkevi after Vertex refused to submit evidence.

Vertex says it needs the high price to invest in new cystic fibrosis medicines that will help more people, but the drug maker is on target to make $21bn worldwide from Orkambi and an earlier drug, Kalydeco, the Guardian has revealed.

David Ramsden, the chief executive of the Cystic Fibrosis Trust, called the decision “upsetting and unsettling”. It would come as a shock to families in Scotland, he said.

“While talk of further negotiation between Vertex Pharmaceuticals and the Scottish government is positive, people living in Scotland need these life-saving drugs now,” he said. “The tragic wait has to end and now is the time for the Scottish government to remain committed and Vertex to do all they can to ensure this chance does not slip away again.”

Some of the campaigners had expected some sort of deal to be clinched in Scotland, where there are 900 people with cystic fibrosis, 40% of whom have the genetic mutation Orkambi targets. The bill would be far higher in England, where there are 10,000 patients. The drugs are not a cure, so would have to be taken for many years, until something better comes along.

NHS England has been highly critical of the company, saying it has made two of its most generous ever offers for access to the medicines. Among all the pharmaceutical companies it has dealt with, “Vertex is an extreme outlier in both pricing and behaviour,” it told the Guardian.

Vertex said it was disappointed but was still in discussions with the NHS in Scotland and England. “Since learning of the SMC decision, Vertex has been working with the Scottish government and other interested parties to develop a solution to allow broad access for all eligible patients in Scotland with cystic fibrosis,” it said.

“We are all deeply committed to reaching such a solution as a matter of urgency and we are working closely with the government to finalise it over the coming weeks.”

There is a compassionate access scheme for patients who have lost a substantial proportion of lung function because of the disease. “Currently, approximately 65 patients eligible for Vertex medicines in Scotland have been approved to receive these medicines under this scheme,” Vertex said.

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