NICE Appraisals: Rare Diseases Treatments

Marion Fellows Excerpts

Thursday 21st March 2019 Commons Chamber

[Marion Fellows (Motherwell and Wishaw) (SNP)]

It is a pleasure to follow the hon. Member for Strangford (Jim Shannon), and I congratulate the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon), and the Backbench Business Committee, on securing this debate.

This is not the first time that I have spoken in such a debate, and I declare an interest because my two-year-old granddaughter, Saoirse Fellows, has cystic fibrosis. We are very fortunate because she is pancreatic sufficient, but it is a real worry for our family. I should also say that I am waiting for granddaughter No. 4 as we speak, so if I am a bit lightheaded that is why. I have never listened to a debate in either Westminster Hall or the Chamber in which so many people have cited Scotland as a great example of how to do things. That is normally my job in these debates, so I am grateful to hon. Members who have already done part of that job for me.

An estimated 7,000 rare diseases affect about 3.5 million people in the UK, and around 80% of those are genetic. It has been reported that 95% of rare diseases have no approved treatments available. The default NICE referral route for the majority of orphan medicines is the single technology appraisal, but many will be close to meeting the selection criteria for NICE’s highly specialised technology programme, which has already been mentioned. Stakeholders are frequently sought to make the case for an HST referral, which is a more appropriate route for orphan medicines.

The Government have maintained the position that it is appropriate for orphan medicines to be considered under NICE’s standard STA and that orphan medicines have been successfully reviewed under the STA programme. However, many Members have said how that is not working for their constituents. The Scottish Medicines Consortium has adapted its processes to increase the input of clinicians and patient group experts in decisions for certain orphan medicines.

Mr Deputy Speaker, I am cutting my speech because I want to allow more time for both the Opposition spokesperson and the Minister. Scotland and the SMC has led from the front, and NICE has adopted many of its methods in the past. I encourage the Minister to look again at what Scotland is doing. We are a small country and a devolved nation, but we can do it, so I can see no reason why the England NHS should not be getting the same medicines as we do. We have a higher approval rate: 69% in Scotland to 55% in England. We have a new medicines fund specifically for orphan drugs, which is instrumental in helping to secure access to medicines for rare diseases. That does not exist in the other devolved nations. Both Scotland and Wales have taken a proactive approach to addressing concerns about the applicability of the standard processes for orphan treatments.

The new medicines fund has really helped to deliver better medicines more quickly to Scotland. The fund has covered the cost of orphaned drugs for individual patients where the condition affects fewer than one in 2,000 people. A total of £21 million was made available for the fund by the SNP Scottish Government. The Scottish Government have since made further improvements, reforming access to new medicines by creating the peer approved clinical system tier 2. The system allows clinicians, on behalf of their patients, to ask a PACS panel whether they can access a medicine that has not yet been recommended by the Scottish Medicines Consortium. That is exactly why Orkambi and Symkevi can be used in Scotland. I would suggest it is absolutely imperative that this is looked at in England. Spinraza, which has been mentioned in the debate, is already being used in Scotland. I ask the Minister this: why is it not possible for NHS England to use those drugs in England to the same extent that they are in my own country?

I do not want to bring too sour a note to the end of this debate—there is real consensus across the House, and Members have spoken sincerely and passionately on behalf of their constituents and others across the country—but I worry, and I think many other Members across the Chamber will be worried, that we are heading towards Brexit. No one knows where it is going or the impact it will have on the availability of medicines generally, and, more importantly for this debate, medicines that are not yet even available in England as they are in Scotland.