Cystic fibrosis patients like my daughter can wait no longer

When I first heard about Orkambi, the drug that could treat the root cause of our daughter's type of cystic fibrosis, I was hopeful that she would soon have access to it. But the years have crept by. It is now 4 years since Orkambi was approved for use in the US. My daughter recently celebrated her 4th birthday. Talks between the UK government and Vertex (the pharmaceutical company that makes the drugs) have stalled and restarted multiple times - and we are still waiting. The awful fact is that some people with cystic fibrosis cannot afford to wait - 250 people have died in the time that has passed.

So where does this leave us?

I never imagined that I would be considering a buyers club as a viable option for getting up-to-date treatments for my daughter in 2019.

At the start of June the CF Buyers Club popped up in my news feed. My first thoughts were: isn't that a film - the one that propelled Matthew McConaughey from romcom pretty-boy to credible actor? Wasn't that something that happened with HIV in the 80s?

The CF Buyers Club is proposing a way for people with cystic fibrosis to access medicines that are not available on the NHS. An Argentinian pharmaceutical company currently provides a generic version of Orkambi to patients in Argentina. They are offering to supply their version of Orkambi for less than a quarter of the price Vertex is offering.

It is astonishing to me that, in a country with a world-class National Health Service, the cystic fibrosis community has been left with no other option than to form a buyers club to get our hands on these drugs. I thought that Dallas Buyers Club (starring McConaughey) is the sort of film you watched on a Friday night with a takeaway; the sort of film that made you appreciate that we have access to drugs that can control a devastating disease like HIV. I never imagined that I would be considering a buyers club as a viable option for getting up-to-date treatments for my daughter in 2019.

If 500 people sign up to the CF buyers club, the pharmaceutical company will drop the price to £18-20,000 per person, per year. Considering that some people are paying Vertex the shocking amount of £104,000 privately for a year's supply of Orkambi, this is a very attractive prospect. However, it is still a colossal amount of money, and money that we, as a family, don't have. And if we did manage to raise £20,000, how could we be sure that we could raise it the next year to continue her treatment?

For some families, £20,000 a year is affordable. But it's not right that people with the cash get the treatment and people without it have to do without. Surely this is a window into a world without the NHS - where people who can afford a doctor are treated and it is tough luck for the poor; where parents have to make the unthinkable decision of which of their sick children to treat.

We pay our taxes to fund the NHS to deliver equal care for every person. If my family are forced to fundraise for my daughter's cystic fibrosis treatments, we would all be effectively paying twice.

But what other options do we have? Vertex reportedly has $3 billion in the bank, so they are in no hurry to secure a deal for their drugs.

it’s not right that people with the cash get the treatment and people without it have to do without.

But now that we know a generic version of the medicine exists, I want to call on the UK government to make it available on the NHS for every person with cystic fibrosis. Not just for those who can afford it. Vertex have had their opportunity and I'm not willing to let these interminable talks drag on any longer.

In a debate on Orkambi in parliament on 10th June the government seemed to move closer to this view - saying they have a ‘moral obligation’ to pursue means of providing access to the generic version of the medicine in the face of Vertex’s continued refusal to negotiate in good faith. I want to call on the UK government to recognise the extremity of the situation for the cystic fibrosis community and see this new commitment through until patients all have access.

Anna Bennett lives in Birmingham with her husband and 4 year old daughter who lives with cystic fibrosis. She works as an ESOL teacher and enjoys reading, playing the violin and watching for the Spring.

Elizabeth Baines