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 drugs that have caused such a stir are made by the US biotech company Vertex. Its groundbreaking Kalydeco drug was licensed in Europe in 2012 and useful at first to only a small percentage of sufferers. Then came Orkambi in 2015 and Symkevi in 2018 – both two-drug combinations. Since 2020, there has been Kaftrio, which is a triple combination. Hopes rose in the UK as the combination drugs were shown to target more genetic mutations and help more people, only to be dashed by the verdict of the cost-effectiveness watchdog body, the National Institute for Health and Care Excellence (Nice). Kaftrio is the latest to be labelled unaffordable for a cash-strapped NHS.
