The assessment of Orkambi® continues today as The National Institute for Health and Care Excellence (NICE) takes evidence from people with cystic fibrosis, clinical experts and the drugs manufacturer Vertex Pharmaceuticals Ltd.
Lynsey Beswick, Public Affairs Officer for the Cystic Fibrosis Trust, will be speaking from the perspective of a person living with cystic fibrosis at the first of two NICE evaluation committees.
NICE, an independent public body, accountable to the Department of Health, has responsibility for assessing the clinical- and cost-effectiveness of new treatments. Its advice covers England but also influences decisions made by the Welsh and Northern Irish health systems. Scotland has a separate process , which the Trust is also working with.
Orkambi, which combines ivacaftor and another compound called lumacaftor, could bring significant health benefits for those people with cystic fibrosis age 12 and over who have two copies of the F508del mutation. According to UK CF Registry data (2014) 2,789 people in England, 236 in Scotland, 113 in Wales and 111 in Northern Ireland have this genotype.
As part of its Stopping the Clock campaign, the Trust is calling on NICE, the NHS across all four nations of the UK, and Vertex Pharmaceuticals, the manufacturer of Orkambi, to ensure that precision medicines like Orkambi are available to those that need them as soon as possible.
Today’s meeting is an important milestone towards achieving the Trust's goal of ensuring access to precision medicines to as many people as possible by 2020. However, access is not guaranteed and the Trust is prepared for a sustained campaign in each of the four UK nations, which have separate processes.
The next step in the campaign will be handing in the petition urging greater access to life-changing treatments - which a phenomenal 20,000 people signed - to Vertex Pharmaceuticals and the Governments across the UK.
Watch out for Lynsey’s blog, where she will share her experience of appearing before the NICE committee.