NICE Appraisals: Rare Diseases Treatments

Debate between Thelma Walker and Liz Twist

Thursday 21st March 2019 Commons Chamber

[Liz Twist]

Like the hon. Gentleman, I took part in the PKU diet for a day challenge. It was indeed very restrictive, even though I knew it was only for one day.

Archie is one of those who responds well to Kuvan, and last year, his parents took the difficult decision to pay to buy him the drug. That took a great deal of soul searching on their behalf, and it cost them dear—£25,000 a year, even though Archie is currently on just half a dose. They know that for many people with PKU, or for parents with more than one child who has PKU, it is simply not possible to self-fund, and they are acutely conscious of the unfairness of that. The difference that Kuvan has made to Archie is real and significant: increased concentration and energy, so that he can make the best of his education; no recurring mouth ulcers, which were a real problem; and a chance substantially to increase the number of exchanges he can have, and eat a more normal diet. For Archie, Kuvan has made a real difference.

Archie’s case, and those of many others in other constituencies, prompted us to form the all-party group on phenylketonuria, and to consider how Kuvan could be made available to those who would benefit from it. Nearly 10 years after Kuvan was approved, that treatment does not seem to have been an appraised, and in England it is still not available on the NHS. Since we set up the group, there has been a move for Kuvan to be appraised by NICE, and discussions have been held with NHS England about a managed access agreement. We were disappointed to learn just before Christmas that no agreement had been reached on that managed access agreement, and that the NICE appraisal was to be via the single technology appraisal route, and not the highly specialised technologies programme. I understand that following a legal challenge, the Department of Health and Social Care is again considering the appropriate appraisal route, and the all-party group has made representations on that point.

When talking about the NICE appraisal system it is easy to get lost in technical details—QALYs, and everything else, that means nothing to people on the street—but what really concerns people is whether or not there is a fair chance that the drugs they need will be fairly assessed and made available on the NHS.

[Thelma Walker (Colne Valley) (Lab)]

I thank my hon. Friend for securing this debate. One of my constituents suffers from Batten disease and is receiving cerliponase alfa on compassionate grounds. However, NICE will not now recommend that treatment, which in part is due to cost. Does my hon. Friend share my view that allowing patients on to clinical trials when there is no funding to deliver the treatment, places them and their families in an incredibly difficult and uncertain position regarding their future?

[Liz Twist]

I certainly agree, and such things happen too often. We need a system that properly evaluates those drugs.

British Sign Language: National Curriculum

Debate between Thelma Walker and Liz Twist

Monday 5th March 2018 Westminster Hall

[Liz Twist]

I thank my hon. Friend for her intervention. She gave a good example of how everyone benefits from BSL and signing, which is an idea that I will touch on later.

As I was saying, yesterday “The Silent Child” won an Oscar. Starring six-year-old Maisie Sly and Rachel Shenton, it tells the story of a four-year-old profoundly deaf girl who struggles to communicate until she learns sign language. I am sure that all Members will join me in sending our congratulations to Maisie, Rachel and the team that produced the film. Now all I have to do is follow that.

Moving on to the petition itself, the petitioners ask for BSL to be part of the national curriculum. They point out that about 50,000 people in the UK use BSL, that many children are born deaf and that those children should be given

“a better chance at a more integrated future.”

[Thelma Walker (Colne Valley) (Lab)]

I commend my hon. Friend on securing today’s debate. Deaf children should be able to interact with their peers as much as those children who can hear. Does she agree that a simple solution to make our education system truly inclusive would be for the Department for Education to include British Sign Language in the national curriculum for all schools and all children?

[Liz Twist]

I thank my hon. Friend for that intervention. I agree with that statement, and I hope to explain why.

The petitioners want BSL to be part of the national curriculum, giving better life chances to young people who are deaf. They believe that if BSL becomes part of the national curriculum, that will even up the chances of deaf young people being able to play a full part in school and attain the best results they can.

Let us look at the case. Research by the National Deaf Children’s Society into the attainment of deaf children in 2017 shows that deaf children continue to underachieve throughout their education compared with other children. Although the Department for Education claimed recently that attainment for deaf children is at an all-time high, the latest figures show that the attainment gap between deaf children and children with no identified special needs is widening, with the gap at GCSE level being particularly worrying. In 2016, 41.3% of deaf children achieved the expected benchmark of five GCSEs at A* to C grade, compared with 69.3% of children with no identified special needs. That is a difference of more than 20%, which is just not acceptable in this day and age.

All that is in the context of a reduction of 14% in the number of qualified teachers of the deaf since 2011, and a 2% reduction in just one year—2016-17. We know that we have to do more to help deaf pupils to achieve their full potential and that we need to reverse the reduction in the number of teachers of the deaf. We can do that partly by ensuring that young deaf pupils are able to have effective communication. For many, that will be through BSL. BSL has been a recognised language since 2003, but unlike other languages it is not recognised as a GCSE that can be taught in schools.