Scotland’s NHS could reimburse Vertex CF drugs, price row ongoing in England

Vertex is to file its two latest cystic fibrosis drugs with Scotland’s drug cost watchdog in a bid for NHS reimbursement – but an ongoing row over price in England is still unresolved. The US pharma said it will submit files for Orkambi (lumacaftor+ivacaftor), and Symkevi (tezacaftor+ivacaftor) with the Scottish Medicines Consortium, potentially allowing more patients with CF access new therapies that treat the underlying cause of the disease. Vertex has been developing a series of drug combinations that have allowed more and more patients to receive therapies that correct the genetic defect that causes symptoms including a build-up of sticky mucus in the lungs.
If the SMC agrees that the NHS should fund the drugs, eligible patients in Scotland could have access to the medicines – but this is cold comfort for patients in the rest of the UK who are still waiting for access to Orkambi more than three years after it was first approved in Europe. In England, NICE said Orkambi was too expensive for the NHS in 2016, and since then Vertex and NICE have been unable to reach an agreement that will bring the drug to patients. Vertex has also refused to file a dossier with NICE for Symkevi, which was approved in Europe at the beginning of November, saying that the cost-effectiveness body must change the way it assesses targeted rare disease drugs like Orkambi.