Access to spinal muscular atrophy (SMA) treatment Spinraza featured prominently in a House of Commons debate yesterday on the NICE appraisal processes for treatments for rare diseases.
The debate was introduced by Liz Twist MP, one of the vice chairs of the All Party Parliamentary Group (APPG) for Muscular Dystrophy, and supported by Mary Glindon MP, Chair of the APPG for Muscular Dystrophy, who both made powerful speeches outlining the problems and concerns about the way in which treatments such as Spinraza are being assessed and highlighting constituents’ frustrations.
Their speeches were followed by passionate contributions from other MPs highlighting Spinraza, including Eleanor Smith, Crispin Blunt, Kerry McCarthy, Lyn Brown, Neil O’Brien, Ian Austin, Jim Shannon, Marion Fellows and Sharon Hodgson.
What did MPs say in the debate?
MPs made several key points focused on why the system is not currently fit for purpose for rare disease treatments. Their comments on Spinraza and the overall process included:
- existence of just two appraisal routes and treatments being stuck in the middle – not meeting criteria of the Highly Specialised Technologies (HST) route and therefore being assessed by Single Technology Appraisal (STA) route designed for non-rare treatments
- responsibility of pharmaceutical companies to make drugs affordable for the NHS
- Spinraza available in 24 European countries and in the USA
- Scottish Medicines Consortium approved Spinraza for SMA Type 1 in May 2018, and speedily reassessed Spinraza under the new ultra-orphan pathway for SMA Types 2 and 3, and is set to be available from next month
- NICE emphasis on cost effectiveness calculation in contrast to focus on flexibility and data gathering for further review in Scotland and across Europe
- either put Spinraza through the route for rare medicines, or find or invent a new mechanism.
- Spinraza appraisal has taken 14 months and there are several months in between the different stages of the process
- no news announced yet following the NICE meeting on 6th March
- issue of availability of mental health support and counselling
- frustration and anxiety for many families who see a life-changing treatment within touching distance
- call for funding to be set aside for life-saving drugs
- call for the Minister to work with Muscular Dystrophy UK to create a new and fairer system like in Scotland that will deliver for patients
Health Minister’s response
Health Minister Steve Brine MP responded to the MP speeches, commenting that he will reflect on the call for a third appraisal route for rare disease not eligible for the HST programme. He went on to say about Spinraza:
The hon. Member for North Tyneside and others raised the issue of the drug Spinraza for the treatment of spinal muscular atrophy. I understand that NICE’s independent appraisal committee met earlier this month to consider its recommendation on Spinraza following new evidence being put forward by the company. NICE wrote to the company and patient groups last week to say that it was not yet able to provide an update on the outcome of the meeting, but that it would provide an update soon. Again, I encourage that to happen even sooner. I recognise that the protracted process in this instance is hugely frustrating for patients and their families and, whatever our differences across the Dispatch Box, of course I feel the deep hurt that the hon. Ladies who spoke on the subject have laid out. I hope they will appreciate that a final decision has not yet been made and that NICE must be allowed to complete its work free from political interference.
We continue to urge NICE to release their decision on access to Spinraza as quickly as possible and will be working with other charities to press for a fairer NICE process for assessing new treatments.
Contact your MP to ask for their support on access to Spinraza.