The Speaker
Friday, 14 June 2024 – 10:06

Cystic Fibrosis ‘Miracle Drug’ Made Available on the NHS

The life-changing CF drug Orkambi has be made available, after years of campaigning from CF sufferers and their families.

Orkambi- a drug proven to increase lung function and slow deterioration- is to be made available on the NHS in England, saving around 5000 lives

The deal comes after campaigners from across the UK demanded effortlessly for over four years to force the government and Orkambi manufacturers Vertex Pharmaceuticals, to reach a price deal which would make it more accessible to NHS clients. Before the deal was made, it was expected that the price of the drug would have cost the NHS £104,000 per year per person.

According to statistics compiled by the British Lung Foundation, around 1 in 25 people in the UK suffer from Cystic Fibrosis- a life limiting, inherited disease. It affects lung function significantly due to over-production of sticky mucus that naturally lines the respiratory system. It is caused by a set of hereditary faulty genes, but only by a person receiving two recessive genes, making it more rare than other hereditary diseases. A new agreement between governing bodies and the drug manufacturer means that CF sufferers will now have access to the drug within the next 30 days.

NICE (National Institute for Health and Care Excellence) initially rejected Orkambi due to the high price, however, they have finally come to an agreement with Vertex. This is due largely to the binding condition that Vertex must submit its full portfolio of its triple therapy treatment- using Orkambi and the two other drugs, Smykevi and Kalydeco- for a full NICE appraisal, and the agreement of ‘confidential commercial terms’ submitted by the national body. The triple treatment has already received approval from the NICE equivalent in the US- the FDA- and is thought to be effective on up to 90% of patients.

NHS Chief Executive Simon Stevens has responded to the new deal by saying:

“The UK has the second highest prevalence of cystic fibrosis of any country in the world, so today is an important and long hoped for moment for children and adults living with cystic fibrosis,” 

However, the news has not come without criticism, mainly in relation to the length of time it has taken to reach the agreement and Vertex’s inflexibility on price and non-compliance with the NICE appraisal process. Two months before the news of the NHS’ success story, it was reported that by making the drug unaffordable Vertex would have been making $21 billion from treating CF. The drug is also only being made available on the NHS in England, with families in Wales and Northern Ireland still battling with devolved powers and pharmaceutical companies. The drug has already been made available on the NHS in Scotland.

In politics, the success of the deal has led to further challenge from the left in the UK around ‘big phrama’, especially as the race to acquire drugs for other serious illnesses such as hepatitis C and breast cancer is ongoing. In an article written for Labour Link, Shadow Business Secretary Rebecca Long-Bailey met the decision with expressions of optimistic endeavours to reduce the cost of pharmaceutical products in the UK, by stating:

“Labour’s solution, which will tackle both high prices and the additional cost of public health priorities not being met, is to “delink” financial rewards and high product prices from the costs associated with R&D. We’ll do this by providing direct financial support for health innovation through a combination of upfront grants, subsidies and prizes.”

Appearing on ITV morning show This Morning, Labour leader Jeremy Corbyn identified that the high cost of drugs often comes down to issues with the patent, saying that for this to be brought down he thinks ‘the public should have ownership or part-ownership of the patent’ and that the “NHS should have the ability to manufacture it’s own generic medicines which would be a lot a cheaper”.

Government Health Secretary Matt Hancock- who played a tough role in negotiating for the drug- said he would ‘trash out’ a deal to provide the vital drug at an affordable price back in August. 116 MPs overall joined the fight to end the deadlock. Mr Hancock welcomes the overwhelming news with hope, knowing that so many people will be positively affected.

Photo by Sharon McCutcheon on Unsplash

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