Drugs (Ultra-rare Diseases)

Alec Shelbrooke Excerpts

Tuesday 20th January 2015 Westminster Hall

[Greg Mulholland]

Absolutely. That is very much the case with Morquio, too. Simon and Katy Brown have told me that the drug is having a huge impact now. I met Sam and saw him running around when he visited my surgery. When there is deterioration in such conditions, the clock can never be turned back, which is why we are urging the Minister to address the situation. I am delighted that we had meetings with him. All the organisations involved, the MPS Society, the Muscular Dystrophy Campaign, Action Duchenne and Joining Jack, are urging the Minister and NHS England to find a way to ensure that all these children, not only the 111 who are currently on the trials—some of whom are, of course, receiving placebos—but all 138 children with these conditions, are able to access the drugs now. We have asked for a decision on that by the end of January.

We are in this situation because a decision was supposed to have been taken by NHS England on 15 December 2014, but a letter was sent by the MPS Society and a young man with Morquio syndrome, Kamal—I am delighted that his family are visiting Parliament today—and on receipt of that letter NHS England, realising that its process was potentially discriminating against people with ultra-rare conditions, pulled the entire process, leaving all these families in limbo. NHS England has a responsibility to put another proper, robust process in place.

[Alec Shelbrooke (Elmet and Rothwell) (Con)]

I am grateful to my hon. Friend for giving way. I will be meeting my constituent Angela Paton on Friday afternoon. She was part of the trial, which helped her immensely, but she is worried that the drug may now be withdrawn. Would my hon. Friend like me to report to him after that meeting on Friday?

[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.