Orkambi and Cystic Fibrosis

James Heappey Excerpts

Monday 19th March 2018 Westminster Hall

[Luke Hall]

Absolutely—that example highlights the positive work that the Cystic Fibrosis Trust is doing. I want to put on record my thanks and appreciation to all the parents and campaigners, whom I find completely inspirational. Not only are they parents, which is challenging enough in itself sometimes, but they are parents of children with CF, and on top of that they manage to find time to lobby their MPs, to get groups of people together, and to get support for petitions and debates such as today’s.

[James Heappey (Wells) (Con)]

My hon. Friend speaks eloquently about the many parents and children who have been campaigning for this cause. I add to them the voice of Helen Mann, my constituent, who has written the most wonderful, compelling book about her daughter’s life with cystic fibrosis and, indeed, her treatment with Orkambi. Her daughter, Clara, came to knock on my door yesterday lunchtime to make sure that I came to today’s debate. It was amazing to see her stood on my doorstep so full of life—a living example of just what an amazing drug it is, and how many others could benefit if only it were available to all.

[Luke Hall]

I pay tribute to all the young people such as Clara who are getting involved and making a difference in the campaign to ensure that Orkambi is available.

I was probably as disappointed as every Member in the Chamber to read the statements that came out on Friday about how a deal on the February offer by Vertex had not been reached. The petition for today’s debate was signed by 470 people from my constituency. I am here speaking on their behalf, and on behalf of Annabelle Brennan, and her parents Elizabeth and Shane. Annabelle is three years old, and is a lovely young girl. I met her at a CF campaigning event in my constituency. She does things that other three-year-olds do, of course, but she also has to endure a complex daily routine of physiotherapy, nebulisers, medication and antibiotics to help her to manage and live with the illness.

I received an email from Annabelle’s nursery leader, who is also my son’s nursery leader, explaining that Annabelle’s time at nursery, and her interaction with the other children in the group, is affected by her condition, because she is under constant supervision and is not always able to be involved in all the activities like the other children. Every winter, there is an added risk of further infection due to the common cold, and, as time passes, the potential for more complications increases. I remember hearing a devastating description from Annabelle’s parents of how every day is like waking up on a cliff edge, not knowing when irreparable damage caused by CF will come. That highlights the importance not only of these debates and the pressure, but of getting a deal, so that Orkambi can be made available as quickly as possible. That would ensure that children such as Annabelle, and people living with CF throughout the country, can realise their full potential.

A number of colleagues have laid out evidence today that shows that Orkambi works. We heard about Carly’s story from the hon. Member for Dudley North (Ian Austin)—I thank him for all he has done. We have seen international examples of the difference that Orkambi has made, but the reality is that progress on reaching a deal has been devastatingly slow. We know that Orkambi can help about 50% of people with CF. The other point we should remember is that precision medicines such as Orkambi are not just the better option—for some people, they are the only option. People have stated the countries around the world where it is already available: Austria, Germany, France, Ireland, Italy, the USA, Luxembourg, the Netherlands, Greece, Denmark, and many more. Despite the prevalence of CF in the UK, it is still not available here.

The set of statements released on Friday was disappointing, but although an agreement has not been reached, that must not be the end of those discussions; they should continue, ensuring that a deal is reached as soon as possible. It is also important that NHS England ensures that any deal is sustainable in the long term, and flexible enough to enable us to take into account any future advances in CF medication, either by Vertex or another company. The aim of NHS England and the National Institute for Health and Care Excellence should be to negotiate the best possible long-term access for people living with CF. That has to be reflected in the deal.

I ask the Minister to convey to both Vertex and NICE the importance of ensuring that a deal is reached as quickly as possible, and I encourage him to take a seat round the table himself, so that Annabelle and other people around the country have access to these drugs. I also ask him to update us on what conversations his Department has had with NHS England and Vertex since the announcement on Friday to see where progress is likely to be made in the coming weeks and months. I finish by saying to the Minister, NHS England and Vertex, “Can we please ensure that a deal is reached as quickly as possible, because this drug has the potential to change thousands and thousands of lives?”