Children with Life-limiting Conditions

Mary Glindon Excerpts

Tuesday 29th January 2019 Westminster Hall

[Mary Glindon (North Tyneside) (Lab)]

It is an honour to serve under your chairmanship, Ms Dorries.

I congratulate the hon. Member for Strangford (Jim Shannon) on securing the debate. He and I are both members of the all-party group for muscular dystrophy, so I know that his commitment to the issues being discussed this morning is genuine.

Following on from my hon. Friend the Member for West Ham (Lyn Brown), I will talk about Spinraza, because nothing is more fundamental for anyone with a life-limiting or life-threatening condition, or their families, than to have access to treatment that will give the chance of a better quality of life and, possibly, some chance of longevity.

As my hon. Friend said, one of the rare conditions that until the last few years has had no proven treatment is SMA. There are four types, and the most severe is type 1. Infants diagnosed with that have a life expectancy of no more than two years. The condition affects the lower motor neurones in the spinal cord, leading to loss of mobility and eventually of the ability to breathe and swallow.

The drug Spinraza, which was developed and marketed by the pharmaceutical company Biogen, is the only treatment that has proved successful for children with SMA. Spinraza was granted a marketing authorisation by the European Medicines Agency more than 18 months ago. It is available in 24 European countries including Scotland, as has been said, but not in other parts of the UK.

The APPG, which I chair, has supported the work of our excellent secretariat organisation, Muscular Dystrophy UK, and other groups to press for Spinraza to be approved by NICE. Many MPs across the House with constituents who suffer from SMA feel the frustration of families waiting for Spinraza to be approved. So far, however, progress has been slow. That is largely due to the fact that Spinraza has been assessed by NICE under the single technology appraisal, or STA, route, which is not appropriate for such a rare condition. That route is normally used for more common conditions, and it is now a year since the assessment began. Also, in August, when NICE published its initial decision on access to the drug, it did not recommend Spinraza for use on the NHS. That was a bitter blow for all the families, including the family of young Sam McKie from North Tyneside, who has the condition.

Biogen opened an expanded access programme globally in 2016, as an interim solution for patients with infantile-onset SMA. In the UK, the programme was extended to support continued access for those patients until NICE completed its appraisal. To date, more than 80 eligible children in the UK have received the drug free of charge. Under the timeframes provided by NICE, the final appraisal document was scheduled for last November; therefore, disappointingly, Biogen closed its access to the EAP for new patients.

Since August, the APPG has been active in pressing NICE, NHS England and Ministers to be flexible in finding a way forward, and I raised the issue at Prime Minister’s questions in September. There is an impasse, because NICE continues to require that Spinraza should be cost-effective through the STA route, but Biogen has pointed out that, given the smaller patient population in rare diseases, it is inappropriate to expect treatments to achieve the same cost-effectiveness thresholds as medicines in disease areas that have much larger patient populations.