Drugs: Ultra-rare Diseases

Debate between Cheryl Gillan and Greg Mulholland

Tuesday 16th June 2015 Westminster Hall

[Greg Mulholland]

I hope that we get answers today and a real promise of intervention from the Minister.

[Mrs Cheryl Gillan (Chesham and Amersham) (Con)]

I pay tribute to the hon. Gentleman’s work on this issue. I joined this campaign because of Archie Hill, a constituent of mine aged 10 who has Duchenne muscular dystrophy. No matter what the Minister says about drugs such as Translarna and the process that the hon. Gentleman is about to outline, which has been disgraceful, that drug is available in other European countries and we have still not cleared it for patients in the UK.

[Greg Mulholland]

Indeed. It was a pleasure to meet the right hon. Lady’s constituent, Archie, and his parents. These young people are inspiring us to campaign. She is absolutely right. We are debating the European Union Referendum Bill today in the Chamber. Other EU countries, and some non-EU countries, regard these treatments as effective and affordable, yet we do not.

I will fast-forward from the scrapping of the previous body to October 2014, when NHS England came out with the scorecard system. That is despite one of the clinicians involved, Dr Chris Hendriksz, saying on 22 October in an email:

“I would suggest the scoring is not used at all for decision making this round and I would rather have people acknowledging that they are making random decisions than to try and give some credibility to a process that was deeply flawed.”

That is from one of the senior clinicians.

NHS England none the less went ahead with the scorecard system to decide which funding should be prioritised. Suzanne Mallah and her 10-year-old boy Kamal, who has Morquio and is another inspiring young person whom I have been delighted to meet, saw that that was not only haphazard but discriminatory. With the help of the MPS Society, they threatened legal action on 28 November against NHS England on the basis that the scorecard was clearly discriminatory, that there was no policy explaining it and that there had been no public consultation on its use. Just one week after that, on 2 December, NHS England announced that it was suspending use of the scorecard because the MPS Society and Kamal were right and it was wrong.

Drugs (Ultra-rare Diseases)

Debate between Cheryl Gillan and Greg Mulholland

Tuesday 20th January 2015 Westminster Hall

[Greg Mulholland (Leeds North West) (LD)]

I am delighted to have the opportunity to raise this hugely important issue, which affects a number of children across the country represented here today by a good number of right hon. and hon. Members. Although the debate is scheduled for only half an hour, I will take interventions from Members who wish to raise constituency cases. I am happy to do so because this is an important issue that affects families across the country. At the moment, 88 children in the United Kingdom have Morquio. There are 2,500 people with Duchenne muscular dystrophy, but 50 have a nonsense mutation, which falls into the category of an ultra-rare disease. We are talking about 138 people, 111 of whom are currently on treatment trials with the two drugs that are relevant to this debate.

I am delighted that there was a powerful lobby last week on behalf of the group with the nonsense mutation of Duchenne muscular dystrophy. Families from across the country gathered in Portcullis House and then presented a 24,000-strong petition calling for Translarna. I was with the hon. Member for Blaydon (Mr Anderson), who chairs the all-party group on muscular dystrophy, and the right hon. Member for Chesham and Amersham (Mrs Gillan), who was there with her constituent Archie Hill. We were joined by Liam and Saul, two other boys with the condition. We were all delighted that the hon. Member for Blaydon managed to ask a question at Prime Minister’s Question Time, and the answer gave hope to all those children because the Prime Minister gave a very personal answer comparing his own son to Archie—he had a picture of Archie playing football. The Prime Minister came to speak to us, and he said that he would personally do what he could, which echoes the Minister’s work. I thank the Minister for his personal assistance, which has been extremely helpful. I have met him twice, and I know he is very engaged on this matter, which I appreciate.

[Mrs Cheryl Gillan (Chesham and Amersham) (Con)]

I am grateful to the hon. Gentleman for giving way. I pay tribute to him for his work on this subject, which is second to none. I also thank him for mentioning my constituent Archie Hill and his parents, who have campaigned tirelessly for Translarna for their son. Does the hon. Gentleman agree that it is important that NHS England takes on board what the Prime Minister said to us, and to the families whom we represented, at Downing street last week by introducing a plan that enables Translarna to be available to those children who could benefit now, rather than waiting for the bureaucracy that is tying the drug up in knots? It could be available for those children now.

[Greg Mulholland]

Absolutely. It is a pleasure to be working on this issue with the right hon. Lady and other right hon. and hon. Members from both sides of the House. This is a personal issue for me, too. My attention was drawn to the issue when Simon and Katy Brown came to see me with their son, Sam, in 2012. Sam was then four, and he is now six. Sam is receiving Vimizim, which is the only drug that clinically works for Morquio. Both drugs have been shown to have a very significant impact on the health of these individuals, changing what they are able to do with their lives, which is crucial.

--- Later in debate ---

[Greg Mulholland]

It is crucial, but that also tallies with the medical evidence. It has been shown that that particular treatment stabilises symptoms, slows deterioration and has a hugely positive impact on quality of life. Children can do more and lead more normal lives; they have more energy and stamina. People with Morquio can live full lives and go on to education and employment, but childhood is their only opportunity to take a drug to slow the effects of the disease.

Duchenne muscular dystrophy, also caused by a mutation, affects young boys specifically. It also has no cure and gets worse over time. It begins by affecting a particular group of muscles and then muscles more widely, leading to difficulty walking, running, jumping, standing up and climbing stairs. Children with Duchenne muscular dystrophy may end up in a wheelchair fairly young, and are certainly predicted to become wheelchair-bound between the ages of eight and 14 as their muscles weaken and they lose their ability to walk.

[Mrs Gillan]

The thing that came home to me was that those children need to access such drugs quickly, while they are still walking. Is that not why the time scale is so urgent? As soon as the child is no longer ambulatory, the drug will not have an effect. That is why we must have a speeded-up timetable and access to personal budgets for such drugs.

[Greg Mulholland]

The right hon. Lady is absolutely right. Without such drugs, boys with Duchenne and children with Morquio are deteriorating now while waiting. They were expecting a decision on 15 December about whether they would be able to access those two drugs.

Translarna changes the natural course of the mutation in Duchenne muscular dystrophy, slows the decline in physical functioning and can therefore also play an important role in reducing the burden that the condition places not only on the boys who have it but on their families. They can do more, lead normal lives and see their boys do normal things with their siblings.

The number of people affected by the nonsense mutation in Duchenne is very small: there are only 50, 34 of whom are currently in the Translarna trial. The number expected to be eligible for Translarna is about 80 to 90 people, so we are not talking about huge numbers. Some of those people, incidentally, are not yet diagnosed; it is believed that that is the largest potential figure. Vimizim is already licensed in various countries: more than 20 European countries have access to it outside clinical trials, including France, Germany, Italy, Denmark and the Czech Republic. Translarna has been given conditional approval by the European Medicines Agency to treat boys with the nonsense mutation. Data gathered from clinical trials of Translarna indicates that the drug, as well as being effective, is safe. Results of the phase 2B trial were encouraging. Boys who received a low dose of Translarna could walk an average of 31 metres farther than boys receiving the placebo. Translarna is already available in Italy, Germany, Spain and France.

The clear message from all the families and organisations representing people with both those conditions is that we cannot wait for the drugs. NHS England has a responsibility, but so does the Department of Health, because the abolition of the previous highly specialised commissioning service led to an unfit-for-purpose process that had to be scrapped in the face of the pre-action. There is a moral and potentially a legal responsibility to find a way to make that decision. We are now already more than a month past the day when those families were expecting a decision that could literally be life-changing for them.

We understand, of course, that NHS England must put in place a proper process, but I urge the Minister to carry on doing what he is doing and the Prime Minister, who has taken a personal interest in the issue, to find a way to allow all those children to access these two drugs, which have been shown to be effective and to have a hugely transformative effect on their lives and those of their families. I will carry on working with the Minister and the two drug companies, but I urge him to listen to this message. We cannot wait 90 days. We need an interim solution, and I hope that we can have that by the end of January, soon as that is. I will carry on working with him and colleagues throughout the House until we get that news.

Oral Answers to Questions

Debate between Cheryl Gillan and Greg Mulholland

Wednesday 27th June 2012 Commons Chamber

[Mrs Gillan]

The hon. Gentleman would have had the opportunity to discuss that at the Welsh Grand Committee on Monday at 11.30 am, but I understand that Labour objected to the relevant motion yesterday. I now know that that is because Labour MPs have a problem getting up in the morning and getting to work by 11.30 on a Monday—[Interruption.] I have therefore decided to cancel the Welsh Grand Committee and Labour now has the opportunity to call a debate in its own time.

The Prime Minister has met the First Minister on a number of occasions and I believe that that matter, among others, was discussed. I am not aware of any firm commitments made by the Prime Minister.

[Greg Mulholland (Leeds North West) (LD)]

7. What discussions she has had with ministerial colleagues and others on promoting the Welsh identity.