Paul Scully: We must act now to bring breakthrough cystic fibrosis medicine to the UK

Posted On: 
8th July 2019

A breakthrough medicine for cystic fibrosis is available from a US drugs firm, but negotiations to make it available in the UK are in stalemate. We must act now to end the deadlock, says Paul Scully

Cystic fibrosis campaigners protest outside Downing Street
Credit: 
PA Images

Cystic fibrosis (CF) may be rare, but it can be devastating. Children having to live with a regime as punishing as that of a professional athlete just to loosen the mucus in their lungs and be able to function every day; sufferers having to consume sometimes 3,000 calories or more a day to address the suppression of nutrient intake; the inability for many to meet other people because of a risk of infection and the overall risk of a sudden drop in lung function, perhaps to just 20% – CF can be truly crippling.

Half the people who die of cystic fibrosis are under the age of 31. The average life expectancy for someone born now with the condition is still only 47, though this is thankfully lengthening. It is a genetic condition with a significant proportion of sufferers in northern Europe. Around 12% of the global CF community are in the UK. More than two million people in the UK are carrying the faulty CF gene, often without knowing. With more than 2,000 identified mutations of the CF gene, cystic fibrosis is a complex condition with significant challenges to find an effective way to treat this incurable disease.

Until recently it was only possible to limit the effects of the symptoms of this disease, but a series of drugs on the market and in the pipeline have changed the battle, by treating the underlying condition. Some sufferers are seeing a reversal in their decline of lung function. Others have been able to survive long enough to secure a lung transplant thanks to these drugs produced by the large American pharmaceutical company, Vertex.

Each drug along the pipeline works with a greater number of genetic mutations, so allowing more and more people living with CF to benefit. Vertex’s first licensed treatment, Kalydeco, can be used for around 8% of patients. Orkambi extended efficacy to around half of patients, and Symkevi, the next drug yet to be released, increases this further.

A triple therapy currently in development will provide a huge boost to the CF community, with clinical trials showing a potential benefit to 90% of patients and an increase of 10% in some people’s lung capacity in a single week of using the drug.

'This is about human beings, their life expectancy and their quality of life' 

While Kalydeco remains available on the NHS under legacy arrangements, negotiations to provide Orkambi have stalled. To date, Vertex has rejected offers from NHS England and has not engaged with the National Institute for Health and Care Excellence (Nice) process which assesses the clinical and cost effectiveness of new drugs, believing it does not cater for the portfolio approach in this case, having a single offer for all drugs as they become available. Meanwhile the CF community looks on incredulously as people die and others suffer.

Some have even looked at setting up a buyers’ club, having noticed that Orkambi is patent pending in Argentina and so copies are available at £18,000 per patient rather than the eye-watering £104,000. That £18,000 comes out of their own pocket which defeats the idea of the NHS being free at the point of need.

Another solution mooted by campaigners and being considered by government is the Crown use of patents which would allow the use of generic medicines. However, this does have consequences in terms of investment by other pharmaceutical companies into research, should they see patents being removed from private companies.

Nice was set up to remove the politics from the purchasing of medicine but as politicians we must have a view. Members packed Westminster Hall for both the debates on this topic that I led on behalf of the Petitions Committee because of the representations made by their constituents and because, as we all know, CF sufferers are real people, not just entries on a spreadsheet.

This is about human beings, their life expectancy and their quality of life. We need Nice and Vertex to act now and make Orkambi available.

Paul Scully is Conservative MP for Sutton and Cheam