Gillian is working to ensure people with Cystic Fibrosis get access to new medicines that could prolong and enhance their lives and is supporting the ongoing campaign by the Cystic Fibrosis Trust to ensure access to precision medicines.
Orkambi, is a precision medicine that 40 per cent of people with CF in the UK could benefit from. While conventional Cystic Fibrosis treatments target the symptoms, precision medicines tackle the underlying genetic mutations which cause the condition.
Orkambi is not a cure, but it has been found to slow decline in lung function – the most common cause of death for people with cystic fibrosis. It has also been shown to reduce pulmonary exacerbations requiring hospitalisation by up to 61 per cent.
Orkambi received its European license in November 2015 and in June the following year the National Institute of Health and Care Excellence (NICE) recognised Orkambi as an important treatment. They did not recommend the drug, though, on grounds of cost-effectiveness and a lack of long-term data. This means it is unavailable to most people with cystic fibrosis across the UK.
However, more recent 96-week clinical data - not available at the time of appraisal - shows that Orkambi can reduce lung function decline by up to 42 per cent.
Cystic fibrosis is a life-shortening, inherited disorder leading to severely reduced quality of life and life expectancy. Half of all people who died with CF last year were under the age of 31.
People living with this devastating condition experience a build-up of thick, sticky mucus that causes chronic lung infections and progressive lung damage. Daily life can be a struggle and people with the condition face a huge burden of daily treatments, with the most basic tasks causing such breathlessness that oxygen is required to help them breathe.
For more details on Cystic fibrosis go online to cysticfibrosis.org.uk