Orkambi, once too expensive for the NHS, has brought hope to thousands. A new UK-US trade deal could see its price rise again
Wed 20 Nov 2019 10.09 EST
Last modified on Wed 20 Nov 2019 10.11 EST
For the first time in my life I now feel able to consider a more fulfilled and “normal” life. Picking up a cold and ending up in hospital may no longer be my reality and the thought of reaching middle-age seems more realistic. This follows the huge victory of cystic fibrosis patients and the parents of children with the condition to get access to life-changing drugs on the NHS across the UK.
I was diagnosed with cystic fibrosis when I was six. I’m now 21, and since then have been under a strict medical regime which sees me spend three hours a day undergoing treatment; a combination of 30 tablets a day, inhaled medications and constant physiotherapy. Despite my daily routine, I also get admitted into hospital when I pick up a lung infection, leaving me in a hospital isolation room for two weeks at a time. This takes me away from my family and friends, and has had a huge impact on my education and work opportunities.
Cystic fibrosis is an invisible, life-shortening genetic condition. It causes passageways in the lungs and digestive system to become blocked with thick, sticky secretions, causing irreversible fatal lung damage as well as other health problems. The average life expectancy is about 47 years for half of those born with cystic fibrosis. Much less for others. But now a price deal between the US drug manufacturer, Vertex, and the NHS means that about 5,000 adults and children such as me can look forward to a longer life. One of the drugs, Orkambi, has been shown to increase a patient’s lung function by up to 20%.
The years of struggle by people with CF to gain access to these drugs has taken its toll
A few months before the deal, my consultant told me I had the correct CF mutations to take Symkevi, a sister drug to Orkambi and one that has, along with Kalydeco, also been made available on the NHS. At the time, access to these medications, which carried the unaffordable price tag of £100,000 per patient per year, still didn’t seem very hopeful, and the four-year battle to get Vertex to lower its price saw many lose hope. My doctor warned me that it could still be a long wait, so the news that a deal had finally been done was unbelievable at first.
These drugs will significantly improve the quality of life for so many with CF, reducing the daily treatment times, amount of tablets needed, hospital admissions and hopefully avoiding the need for lung transplants. However, the years of struggle by people with CF to gain access to these drugs has taken its toll. Parents watched as their children became more unwell, their lungs filling with mucus, causing lasting damage and knowing their children may die without better treatments. After the tireless efforts of everyone in the CF community, it is a huge victory to be able to get hold of these drugs.
But how secure is this arrangement? Will Britain’s proposed exit from the EU hinder access to these medications? Vertex has assured me that: “When the UK leaves the European Union, all eligible patients across the UK will continue to have access to Vertex licensed CF medicines, as contractually agreed.”
Yet recent reports suggest that a post-Brexit US-UK trade deal could see US drug companies drive up the costs of the drugs they currently sell to the NHS. Considering how hard we had to fight to get these medications, it is terrifying to know that if political circumstances change, our lives may once again hang in the balance. These decisions are in the hands of people unaffected by this debilitating illness, who will not value Orkambi in the same way those with CF do. We have to hope that the fight for our lives wasn’t in vain.
• Isabelle Jani-Friend is a journalist
Source: https://www.theguardian.com/commentisfree/2019/nov/20/cystic-fibrosis-orkambi-brexit-trade-deal
