Citing Costs, Scotland Regulators Reject Adding Orkambi and Symkevi to National Health System

The Scottish Medicines Consortium (SMC) is recommending against bringing two of Vertex Pharmaceuticals’ approved cystic fibrosis (CF) treatments — Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) — into routine use by adding them to the country’s national health system.

Each treatment’s “cost in relation to its health benefits was not sufficient” to justify inclusion in the National Health Service (NHS) Scotland,  the SMC said in separate opinions for Orkambi and Symkevi, known as Symdeko outside the EU.

NHS inclusion would open these medications to CF patients in Scotland at no or low cost.

NHS Scotland and Vertex are continuing talks on a price structure that might satisfy both parties. Similar negotiations are underway between Vertex and health authorities in England, who rejected Orkambi’s inclusion because of questions about its cost-effectiveness in 2016. NHS England decisions usually set policy for Wales and Northern Ireland as well.

Treating CF patients with Orkambi or Symkevi — both of which address people with the F508del mutation, the disease’s most common — is estimated to cost about £100,000 (about $121,000) per person each year, the BBC reported. In the U.S., Symdeko carries a list price of $292,000 per patient per year, and Orkambi $272,000.

The NHS acknowledged that clinical trial results showed both treatments improved lung function and quality of life in treated patients.

The decision was called “tragic” by the Cystic Fibrosis Trust, which invited the CF community to join its “campaign day,” set for Aug. 27, to “remind Vertex and the Scottish Government that thousands of people’s lives are in their hands … while deals remain undone,” the group said in a press release.

“The SMC’s advice will come as a shock to people with cystic fibrosis and their families in Scotland. While talk of further negotiation between Vertex Pharmaceuticals and the Scottish Government is positive, people living in Scotland need these life-saving drugs now,” David Ramsden, the Trust’s chief executive, said in the release. “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 that this chance does not slip away again.”

Orkambi and Symkevi are combination therapies that aim to restore the activity of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is damaged by various mutations in the CFTR gene in CF patients.

CF patients in Scotland already being treated with either Orkambi or Symkevi can continue to do so, the CF Trust announced, either through the PACS Tier 2 system set up by Vertex and the Scottish government in December 2018, or under “compassionate use” programs provided by Vertex.

Those who have not yet started using either, but are eligible, may apply for access through the PACS Tier 2 system, the group added. Both treatments are intended for patients with two copies of the F508del mutation in the CFTR gene.

Officials with the CF Trust noted that the SMC’s opposition likely would affect CF patients in England, Wales and Northern Ireland, as it again failed “to demonstrate that routine access on the NHS is possible.”

“The spotlight is now on the company to agree a fair deal, after the Scottish Government and the company confirmed continuing talks,” the group said.