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The editorial’s perspective on treatments for cystic fibrosis: a turbulent journey for those who are ill.

Cystic fibrosis is a distressing disease, clogging the lungs of children who have to fight to breathe, and shortening lives. Parents – and young adults who also suffer from it – have been on a rollercoaster ride in the last few years. They have been taken to the giddy heights of hope by the invention of drugs that for the first time work on the underlying causes of the illness where previous treatment just alleviated symptoms. And they have been plunged to the depths when those new drugs have been denied them because of their cost.

The medications causing significant controversy are produced by American biotech company Vertex. In 2012, their revolutionary drug Kalydeco received approval in Europe, but initially only benefited a small portion of patients. Later, Orkambi (2015) and Symkevi (2018) were introduced as two-drug combinations. In 2020, Kaftrio, a triple combination, was released. There was optimism in the UK as these combination drugs showed potential for targeting more genetic mutations and helping more individuals, but the National Institute for Health and Care Excellence (Nice), a body responsible for assessing cost-effectiveness, ultimately deemed Kaftrio too expensive for the financially strained NHS.

NHS England is committed to providing access to beneficial medication for children with cystic fibrosis, even if there are limited options available. The negotiations between NHS England and Vertex were challenging, but agreements have been reached. In 2019, NHS England announced a two-year deal with Vertex to purchase Orkambi and Symkevi. In 2020, NHS England quickly secured access to Kaftrio, immediately following its approval, through a prearranged agreement with Vertex.

However, we are currently experiencing a decline in the rollercoaster ride. As per the agreements, data was gathered on the effectiveness of the drugs for patients with cystic fibrosis, including trial information from Vertex. Nice has now determined that Kaftrio, also known as Trikafta in the US, is not financially feasible. Existing patients on the drug should continue to receive it, but new patients will not be prescribed it. This has understandably caused distress within the cystic fibrosis community.


The role of Nice as the antagonist in this situation may seem obvious, but it is simply fulfilling its designated role. The exorbitant cost of Kaftrio is the main obstacle preventing its approval. While a price tag of £180,000 per patient per year may be justifiable for a cure, this drug does not offer a permanent solution. In comparison, Orkambi was only suitable for half of patients with specific genetic mutations, while Kaftrio has the potential to benefit over 90% of those with cystic fibrosis in the UK. If the NHS were to allocate funds for these drugs, it would have to make difficult choices and potentially cut essential services such as hip replacements and cancer care.

Vertex did a tremendous thing in developing drugs for a relatively rare but horrible disease that other pharma companies had ignored. But it is demanding prices that health services here and in other countries cannot pay. Vertex, predicted to make about $9bn this year from Kaftrio, says that it needs billions to invest in research. But what is the point if children are still condemned to suffer and die prematurely because nobody can afford to buy the drugs?

Source: theguardian.com