I first heard of the drug Spinraza last year when Katie Prescott contacted me about her 10-year-old daughter, Heidi. She wrote:
“I have a daughter, Heidi, with a rare muscle wasting disease called Spinal Muscular Atrophy (SMA). SMA ranges from the very severe type 1 through to type 4. Heidi has type 3 and she is fast losing the ability to walk. She is 10 years old and it’s a devastating prospect for her. For the most severe cases of SMA, life expectancy is only 2 years without access to this drug.”
There is a treatment available for SMA, and it is called Spinraza. It is the one and only available treatment for SMA, and 22 other countries, including Scotland, have approved it. However, NICE is recommending that NHS England should not fund Spinraza for any types of SMA, meaning that thousands of people in the UK with SMA are left without a treatment. This is practically a death sentence for babies diagnosed with type 1, and it means that type 2 and type 3 children are destined for a life of deterioration. The issue is that NHS England and Biogen cannot agree on the costs. We need the Government, NHS England and Biogen to sit down and negotiate an agreement that has the SMA community at its heart.
In January 2019, I wrote to the Health Secretary about this. He replied:
“NICE is an independent body, and it would be inappropriate for me to comment on its guidance”.
When I wrote to NICE for clarification, it said:
“I fully accept that we, the company and NHS England have a responsibility to bring this matter to a conclusion quickly. We are working hard to do this through discussions with the company and I am hopeful of reaching a positive outcome.”
I also asked the Prime Minister this question:
“Why can this treatment not be accessible to my constituent Heidi and other children in England with this disease?”—[Official Report,
Vol. 654, c. 1462.]
She answered that the next meeting between the parties would take place in March. Since then NICE, NHS England and Biogen have met, on
When a child is born with a condition such as SMA, there is hope in every parent’s heart that a cure will be found—that a new drug will be developed to treat it. This is an example of that, but the drug has been denied to children suffering with SMA, and we must find a mechanism to resolve the problems. I understand that there are structural issues within NICE, which means that the treatments of conditions such as SMA end up being assessed in the same way as more common conditions, therefore making it extremely difficult for treatments to be assessed as being cost-effective. In the meantime, Heidi’s health continues to deteriorate. When I met her recently in my office, I was deeply impressed with her bravery and optimism, but her mother said to me:
“It is very frustrating that still no decision has been made. Heidi cannot walk unaided anymore and even then can only take a few steps. To know there is something that can help her, but she can’t access it, is very difficult and Heidi finds that hard to deal with too.”
Up and down the country, families like Heidi’s are fighting for treatment. Surely NHS England, NICE and the company have a duty of care and a moral obligation to give treatment to patients like Heidi when it is available. We must remember that there are human beings behind the statistics—caring families who are suffering because a medicine has been denied in this country.