As the Scottish Medicines Consortium (SMC) opts not to recommend the gene-specific medicines Orkambi for people over 12 and Kalydeco for 2–5 year olds to NHS Scotland, the Cystic Fibrosis Trust has put forward a solution for the drugs to be considered by the Scottish New Medicines Fund (NMF).
The NMF, unique to Scotland, is designed specifically to provide ongoing funding to make access to new medicines for rare diseases easier. The NMF already funds Kalydeco for people over six years old, and the Trust is also calling for funding to be extended to 2–5 year olds.
Orkambi has been shown to increase lung function and significantly reduce infection and hospital stays for those carrying two copies of the most common mutation of the cystic fibrosis gene, F508del. In today’s decision, the SMC acknowledges these benefits but opts not to approve Orkambi due to concerns around cost and long-term impact.
The Trust’s solution would see Orkambi provided to all who need it with funding from the NMF, while further evidence is collected on its long-term clinical impact using the UK Cystic Fibrosis Registry.
Ed Owen, Chief Executive of the Cystic Fibrosis Trust said: "People with cystic fibrosis in Scotland must not be denied Orkambi because of a battle between the NHS and Vertex over price and longer term impact.
“The New Medicines Fund is a credit to Scotland, providing a perfect opportunity to provide access to Orkambi and enable the UK Cystic Fibrosis Registry to gather much-needed data on its long-term impact. Vertex, the NHS in Scotland and the New Medicines Fund must come together and make this happen.”
“Kalydeco is already being provided by the New Medicines Fund for those eligible for it and over six years old. It is inconceivable that a drug that has been shown to slow the progression of cystic fibrosis would be denied for younger people who stand to benefit so much at such a crucial time in their development, fighting a progressive disease.”
In a letter to Shona Robison, Cabinet Secretary for Health, Wellbeing and Sport, Ed Owen today urges intervention from the Scottish Government to work with the company, Vertex, and support the Trust's solution for Orkambi and to ensure equality of access to Kalydeco.
Next week, when the election in Scotland is settled, we will be publishing a letter for the CF community in Scotland to send to their MSP, urging the Scottish Government to support the Trust’s proposals. As ever, your support will be crucial, so be ready to make your voice heard.