Orkambi and Cystic Fibrosis

Rachael Maskell Excerpts

Monday 19th March 2018 Westminster Hall

[Rachael Maskell (York Central) (Lab/Co-op)]

I want to start by thanking the 75 constituents of mine who signed the petition, not least the inspiring parents and patients who have really brought this debate to the fore. I want to mention Dr Aaron Brown, who is based in York and is living proof of what it is to live with cystic fibrosis. He was the first person with cystic fibrosis to graduate to the Royal College of Surgeons and is a GP in York. However, for him, living with cystic fibrosis asks many questions, not least as he has a young son and a child on the way. He says,

“Having CF has given me something to battle against all of my life, to prove that it won’t get me and I can achieve.”

We know that this debate is not just about the impact of cystic fibrosis but the hope that a drug such as Orkambi can bring. Although the drug may not be a panacea to all things, it certainly does slow down the disease process, including scarring of the lungs and deterioration of the lung tissue. It is also paving the way for a new generation of precision medicines. It is so important that we are at the leading edge of that research to give people real hope for the future, not just the 50% of people who will benefit from precision medicines should Orkambi be approved, as it must be, but for the next generation.

In the next five years there is potential for 13 new drugs and in seven years, 18 new drugs, impacting on 90% of the cystic fibrosis population. What an amazing opportunity that presents us. That is why we are resolute to make sure that the Minister moves on the issue.

We have heard a lot about cost, but I want to put a cost in everybody’s mind. We are talking about £285 a day. Let us think about the cumulative impact and cost of hospitalisation, perhaps of transplants, drugs and economic loss. We are talking about just £285 a day, a figure that Ministers of State will be familiar with because it matches how much they earn. We can recognise that this is a cost that the NHS can and must afford. Even if there are final negotiations to be had about a portfolio of medication, which is important, to ensure that we usher in this new generation of drugs, what is the cost of a life? I leave that question in the air. I am sure we would all argue that it is a lot more than £285 a day. If we are determined to save lives, that is affordable.

I worked as a physio in respiratory medicine for 20 years, so I have first-hand experience of making this journey with so many patients. The physio may not be pummelling, but it is certainly intense. I celebrate the way that physio has developed over that time. It has become much more dynamic, rather than passive, and a number of techniques have been developed. We heard about active exercise—that is so important for building lung capacity—positive pressure techniques and postural drainage to ensure that we minimise stress on the lungs as we clear secretions. Just think what a life-changing opportunity those advances in physiotherapy, as well as a good diet and precision medicine, present for people with cystic fibrosis compared with their opportunities just a few years ago.

We have a real opportunity to change people’s life chances, but systems have got in the way, and that is what we plead with the Minister to change. The European Medicines Agency approved these drugs two years ago. It is therefore incumbent on us to stress to the Minister that we need effective movement, quickly. NICE itself recognises the benefits of the drugs: in July 2016, it called them important and effective treatment. So much time has been lost through so-called negotiations.

The Minister cannot hide behind NHS England or other bodies. It is time for leadership. It is time for him to step up, step into the debate and ensure that access to the drugs is delivered. I plead with him to call Vertex, to ensure that discussions are held with NICE and NHS England before Easter and to get the table set for finalising the negotiations. If a portfolio of drugs can be achieved in Northern Ireland and the Netherlands, it can be achieved here. Even if there has to be an interim agreement to ensure that people with cystic fibrosis have immediate hope, it is his responsibility to deliver that. With each day that passes, people’s future lung capacity, and therefore their lives, are seriously impacted.

Of course, some people already have access to these precision drugs, and the outcomes for people who benefit from access to Orkambi are really positive. It really is unethical and both economically and clinically illiterate to make Orkambi available to people who are seriously deteriorating but not to people who could stop their lungs being damaged in the first place if they had access to it in advance.

I am grateful to the many constituents who contacted me, but I want to relay the words of Abigail, who wrote very passionately. She has children aged four and seven, both of whom have been diagnosed with cystic fibrosis, and she expressed the real hope that these drugs would bring:

“Those drugs…are here now, which makes it even more heartbreaking that Orkambi, which could benefit 50% of people with CF, is not available on the NHS. To know that there is a drug out there that could change my children’s lives and allow them to live longer and in better health, but which they have been denied access to is absolutely devastating…simply because of cost reasons…It is absolutely imperative that some kind of agreement is reached that makes these drugs available to everyone with CF who could benefit from them. Living with CF is already difficult enough, please don’t make it any harder.”