Drugs (Ultra-rare Diseases)

Debate between Greg Mulholland and Kerry McCarthy

Tuesday 20th January 2015 Westminster Hall

[Greg Mulholland]

Absolutely. It is critical that we all work together on this issue. Indeed, I would like to hear from the MPs for all these families across the country so that we can have one voice to say to NHS England and the Minister, who has been very helpful, that we need a solution and that we need to hear some news by the end of January.

NHS England is now consulting on a new process, and it has said that it will take 90 days. That may seem a reasonable time to come up with a process, but considering that the old process was flawed, there needs to be something to fill the gap that enables all these children to access the drugs now. At the moment, the drugs in this case are being supplied through the good will of the drug companies: BioMarin in the case of Vimizim and PTC in the case of Translarna. Both companies are engaged in the process, both have a part to play and both are involved in dialogue with the Minister and NHS England.

I will briefly explain the two conditions so that people understand. Morquio is caused by the lack of an enzyme needed to break down certain chains of sugar molecules used in building bones, cartilage, tendons and other bodily tissues. Those unbroken molecules are stored in parts of cells called lysosomes, which become swollen, disrupt cell functions and cause progressive damage. Babies with the syndrome grow normally, but growth slows significantly after 18 months. Those severely affected stop growing at about age eight, and their final height may be three or four feet, which has many effects on their quality of life. There is no cure for Morquio, but the enzyme replacement therapy Vimizim, for which clinical trials are ongoing at the moment, has been shown to be effective. As we have said, any delay with the drug will cause damage that cannot then be reversed.

[Kerry McCarthy (Bristol East) (Lab)]

Before I came in, I was speaking to my hon. Friend the Member for Alyn and Deeside (Mark Tami), who would have liked to be here but who has a meeting and sends his apologies. When reading the testimonies from the MPS Society UK, I was struck by how significant a difference the drug makes to children’s energy levels. Obviously, clinical trials and other formal assessments are important, but the personal testimonies from the families about the changes that they have seen in the children and how much energy the drug gives them are far more compelling than any scientific assessment could be. Does the hon. Gentleman agree that listening to the families is important?

[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.