NICE Appraisals: Rare Diseases Treatments

Jim Shannon Excerpts

Thursday 21st March 2019 Commons Chamber

[Jim Shannon (Strangford) (DUP)]

It is a pleasure to follow the hon. Member for South Shields (Mrs Lewell-Buck), with her passion for the subject matter and on behalf of her constituents, so well done to her. I thank the Backbench Business Committee for enabling this debate to take place. I was very pleased to go along to it with the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon)—two formidable, diligent ladies who have put down their marker on this subject matter. I was very pleased to be the third in the trio involved, and well done to them for giving us the opportunity, on behalf of our constituents, to express our concerns today.

I am sure that, like me, most Members in the Chamber have been inundated with emails from people begging to be helped, and begging for the drug that they need to be made available. Every one of them has said that, and I do not believe that anyone can ignore it. It is not only in the emails, but in the interaction we have with our constituents when we hear their stories face to face.

I want to start with a good story and to say what happened—not, I have to say, through this Minister, but through the Home Office. I fought very hard to see medicinal cannabis legalised for my beautiful young constituent Sophia Gibson, and what a difference that has made in her life, as it has to the lives of others. Her parents had no option other than to uproot their family and move to Holland to get the treatment that Sophia needed. At the same time, they were respectfully knocking on doors and following the system through to a Home Office Minister, the Minister for Policing and the Fire Service, and getting clinical assistance in order to bring about a change. Eight months later, their daughter has been at school more than ever before in her whole lifetime. She has had substantially fewer admissions to hospital, and has attended school parties that were impossible—they were too much for her—without this medication. She is a very different young girl today because her parents, Darren and Danielle, fought the battle in the right way and in the right place. Today, we have all presented our cases for the right battle, in the right place, at the right time and with the right request.

Since that time, I have had many other parents coming to me and asking me to help secure the drug that their child needs. A mother in my constituency has an absolutely gorgeous two-year-old son—Lorcán they call him—whom Orkambi would help. Orkambi is a second precision medicine, which targets the root cause of the disease, and it would benefit about half the people with cystic fibrosis in the United Kingdom of Great Britain and Northern Ireland. Cystic fibrosis is a terrible illness that affects the lungs and digestive system of people with the condition, who have a median life expectancy of just 31 years old.

I say this very respectfully, and I hope it will be taken into account: Vertex Pharmaceuticals, NICE and NHS England must end the protracted negotiations for the drugs Orkambi and Symkevi. We must break the stalemate between the three parties and provide access to these drugs, which could so vastly improve the quality of life of my constituents and those of everybody here, as they deserve. They want and need that drug desperately.

I have often spoken about the need to allow people to access Orkambi on prescription, and I have often been beyond frustrated with the lack of movement between the drug company and NICE. There must be—indeed, there has to be—something that can be done to find a way forward, and I believe that it must be done in this House. Today, we look to our Minister and our Government to give us answers and, respectfully, the answers that we need. We must instruct the Department to negotiate a way forward to ensure that my young constituent and so many others like him can live a better life.

I was contacted by the Muscular Dystrophy Association regarding spinal muscular atrophy—other hon. Members have spoken about that condition on behalf of their constituents, and I will do the same. SMA is a rare inherited neuromuscular condition that affects lower motor neurons in the spinal cord. It leads to the gradual loss of someone’s ability to walk, crawl, move, breathe and swallow, and it requires complex medical support. About 100 children are born with the condition each year, and around 2,000 children and adults in the United Kingdom are living with SMA. Spinraza is the first and only treatment for patients with spinal muscular atrophy. It is meant to increase the body’s ability to produce a protein called survival motor neuron, which is essential for motor neuron health. The treatment is administered through an injection into the spinal canal. It is never an easy treatment, but if it provides an opportunity for better health, people should take it.

For children with SMA type 1, life expectancy is rarely longer than two years. Some children who have received Spinraza have seen their muscle strength improve, and have lived long enough to crawl and even walk. For those children, Spinraza has been a lifeline—perhaps I should say that Spinraza has been life itself, as that is the level we are talking about. Spinraza proved so effective in clinical trials for children with SMA type 1 —the most severe form of the condition—that the trial was stopped early so that all children affected by it could potentially access that treatment.

The Spinraza appraisal took almost 14 months, and currently 25 European countries—including Scotland in the United Kingdom of Great Britain and Northern Ireland—have already made it available, nearly two years after the European Medicines Agency granted it a licence. Although Translarna was eventually approved by NICE in July 2016, that was more than two years after the European Medicines Agency gave it a licence. Such lengthy delays to a process that could and should be significantly shorter have resulted in frustration and anxiety for many families who see a life-changing treatment within touching distance. They can almost reach out for it, but they can never get it, and that is where frustration creeps in. That group of people see a better life but are prevented from accessing it. There must be a better way of dealing with these issues, and we must find it or instruct the Department to find it.

My heart aches as a father and grandfather who would do anything for his children and grandchildren, and other right hon. and hon. Members would do the same for theirs. The block on life-changing drugs affects not only the child but the entire family. If a child or adult has a physical disability, their family also feel that and live with it every day. Many families in the UK do not have access to life-changing medication, and I wholeheartedly and sincerely urge the Minister and his Department to enter into talks, find a way forward, and do better so that people can live better. The Government have set aside £40 billion for extra health support. I welcome that, as do all hon. Members, and I gently suggest that some of that money should be set aside for life-saving drugs.