When will the All-Wales Medicines Strategy Group approve Orkambi for use in the Welsh NHS?
Orkambi® (lumacaftor/ivacaftor) is indicated for the treatment of cystic fibrosis in patients aged 12 years or older with the F508del gene mutation.
In 2016, the National Institute for Health and Care Excellence (NICE) re-issued their Technology Appraisal guidance under their “Do Not Do” guidance, emphasising that Orkambi® should not be made routinely available.
The All-Wales Medicines Strategy Group (AWMSG) has contacted the pharmaceutical company, Vertex Pharmaceuticals, and has strongly encouraged them to make a submission to the AWMSG for appraisal. Whilst Vertex has agreed in principle to submit clinical data for appraisal by AWMSG, they have not committed to any firm date for doing so. However, discussions have commenced with Vertex on the most effective approaches to appraisal for the additional license extensions due to come on stream over the next few years. My officials will ensure the future appraisal of lumacaftor/ivacaftor (Orkambi®) is covered.
In the interim, the Welsh Health Specialised Services Committee (WHSSC) has agreed a patient access scheme with Vertex Pharmaceuticals and it is available in the Welsh NHS, where clinically appropriate.