Parents of young Cystic Fibrosis (CF) patients staged a protest at Parliament Square on Wednesday 28 February calling on the Government to make a new treatment available on the NHS.
The demonstrators braved wintry conditions to gather on Rare Disease Day and delivered a petition with over 113,000 signatures demanding urgent action to provide access to the new drug Orkambi.
Parliament will debate the petition on 19 March 2018.
In July 2016, the National Institute of Clinical Excellence (NICE) recognised Orkambi as an important treatment however it did not recommend the drug for use within the NHS on grounds of cost and a lack of long-term data.
Michelle Frank, whose daughter suffers from CF, said: “We’re not just talking about a drug that will ultimately give them a few months, we’re talking about a drug that is going to give them a whole life and you can’t put a price on that.
“No one is going to tell me that price is too big for my six-year-old daughter.”
CF is a life-shortening genetic condition that affects over 10,000 people in the UK and causes sticky mucus to build up in the lungs and digestive system of sufferers.
This can lead to chronic infections and means that people with CF struggle with greatly reduced lung function.
Orkambi is a specific drug for CF created by pharmaceutical company Vertex and, instead of being an antibiotic, it acts to mimic the missing gene that causes the disease.
Data from the Cystic Fibrosis Trust suggests the drug has been proven to slow decline in lung function by 42 per cent and cut the number of infections requiring hospitalisation by 61 per cent.
Routine use of Orkambi costs £104,000 per patient per year.
In response to the petition the Department of Health and Social Care said: “We want patients to benefit from clinically and cost effective treatments. We welcome the dialogue between Vertex and NHS England to agree a deal that would make Orkambi available to NHS patients.”
