York Outer MP raises local child's needs for new spinal muscular atrophy drug with Health Secretary

Julian Sturdy, Member of Parliament for York Outer, yesterday asked the Health Secretary to help ensure people with spinal muscular atrophy (SMA) can access the life-changing new drug Spinraza on the NHS, including 3 year-old York resident Matilda Jamieson, who has Type 3 SMA.

SMA is a serious rare genetic neuromuscular condition that causes progressive muscle weakness and loss of movement through muscle wasting, affecting an estimated 68 babies born every year in England and Wales. Its symptoms include problems with breathing and swallowing, twitching and shaking muscles, bone and joint problems, and difficulty walking. Types 1-3 develop in babies and young children, and Type 1 is generally fatal.

Although there is currently no cure, certain treatment options can help sufferers manage symptoms, and enjoy a higher quality of life. Spinraza is a new treatment, and is already available in many European countries, and has been approved in Scotland.

The independent expert body NICE (National Institute for Health and Care Excellence) which has responsibility for authorising treatments for use in England has stated it does not intend to recommend the drug for use on the NHS on grounds of evidence for long-term effectiveness, and cost. A NICE committee met yesterday to finalise its guidance. If it is not approved, there is still an opportunity for patients to be treated with Spinraza through a managed access arrangement, while further information is gathered on its clinical effectiveness.

During questions to the Secretary of State for Health and Social Care, Matt Hancock, in the House of Commons yesterday morning, Mr Sturdy asked:

NICE has so far declined to recommend the drug Spinraza, despite its ability to transform the lives of patients, such as my young constituent Matilda Jamieson who suffers from Type 3 Spinal Muscular Atrophy.

As NICE meets today to finalise the guidance, can my Right Honourable Friend assure me that he will work with the manufacturers, NHS England and NICE to ensure that patients like Matilda can benefit from it?

Replying, the Health Secretary said that his department worked closely with NICE on these questions, and that it was right that they were able to make objective independent decisions on treatment access.

After leaving the House of Commons, Julian Sturdy said: “While I appreciate the importance of NICE’s independent processes, the fact remains that the Jamieson family and many others are deeply concerned that this life-changing treatment remains unavailable.

Matilda’s parents are very clear on the transformative impact Spinraza could have in her life, and I will continue to do everything I can to enable patients to access this drug. 

Last month I organised a joint cross-party letter from MPs to the NICE Chief Executive expressing concern at their position, and requesting that serious action is taken to put in place a managed access arrangement, covering all types of SMA, so people can start receiving this treatment as soon as possible. Some provision for this should be included in the final guidance being discussed today, to give reassurance to affected families.

I intend to carry on lobbying NICE and the government on this, to help make sure Matilda can access the treatment she needs.”