Five questions I want answered

On Thursday 7th March the Health and Social Care Select Committee will be grilling the NHS, NICE (the body decides on which medicines should be made available on the NHS) and Vertex Pharmaceuticals on the ongoing standoff over access to Orkambi. Anna Bennett, mum to a child with CF, has some questions she wants answered.

I have lived with the uncertainty about whether or not my daughter will have access to precision medicines for cystic fibrosis for three and a half years. At times, it feels unbearable to see what medicines are out there and to know that my daughter cannot use them. So when I tune in to the select committee hearing tomorrow I have five questions that I want answered.

  1. What is the government’s Plan B?
    More than three years have passed since Orkambi was given regulatory approval, and well over six months since negotiations reached a standstill. I have heard that the government’s first preference is for agreement to be reached with Vertex, but if there that does not happen, what is their plan B? As a parent anxiously waiting for action, I want to call on the government to be clear about what they plan to do if this situation continues.

  2. How do we ensure the NHS stays as a world class health service?
    Our brilliant NHS has a global reputation for excellence. Many of my daughter's CF consultants have relocated from around the world to the UK to work for the NHS. However, how can the NHS’s reputation for clinical excellence continue when the medicines that treat the root cause of the most common form of CF is not available? Inevitably, life expectancy for CF patients in the UK will plateau. I do not accept that if Vertex refuse to do a deal, that the NHS will take no further action and allow cystic fibrosis patients to die without access to these medicines. Ever higher drug prices are a threat to our NHS - how is the government going to deal with this problem without making patients pay the price for big pharma’s greed?

  3. What steps have you taken to investigate a Crown use licence?
    As a parent, I feel overwhelmingly powerless to influence a pharmaceutical giant like Vertex; and as the demonstration outside Vertex’s headquarters last year showed, they are not swayed by the voices of CF sufferers and their loved ones. The government agrees that they have the legal power to enact a Crown use licence to get an affordable version of Orkambi for the NHS. We’ve heard why it might be complicated (we don’t think it’s actually as complicated as the government makes out) but we now need to know what they have done to pursue this avenue. It could be the most powerful tool we have for putting pressure on Vertex to lower their prices.

  4. Why haven’t Vertex paid a penny in tax in the UK?
    Since my daughter was diagnosed in 2015, I have fundraised with friends and family to enable research into CF medicines. My father-in-law, who is currently on the kidney transplant list, swam the equivalent of the Channel and my 14 year old cousin gave up part of her summer holiday to complete an exercise challenge to raise money for research into new CF medicines. Vertex have avoided paying tax in the UK and benefited from millions in donations and tax breaks. My question is: How much funding and support have Vertex received from UK taxpayers and the UK CF community? I am distressed by the thought that Vertex could have profited by these means when CF patients and their families cannot access the medicines they helped to develop and fund.

  5. Who is profiting from Vertex’s high price strategy?
    We’ve learnt that it is not only Vertex CEO, Jeff Leiden, making millions every year - senior people in Vertex UK have been made millionaires too. I hope the committee ask Mr Leiden how he feels about the millions he’s putting in the bank whilst patients die without access. I want to know how many staff members’ bonuses are tied to the price agreed with the NHS. And finally, the question that concerns me most: How long does Vertex expect to maintain their monopoly (and the profits that come with it) in the cystic fibrosis market? If a deal for Orkambi cannot be struck, where does that leave CF patients, when Vertex is currently the only pharmaceutical company with licensed CF medicines?

I, along with thousands of others whose lives are affected by CF, are waiting with bated breath to hear how Vertex, the NHS and NICE respond to these questions. Let’s hope the committee members are ready to give them a grilling.

Anna Bennett lives in Birmingham with her husband and 3 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.

Diarmaid McDonald