Drugs: Ultra-rare Diseases

Jo Churchill Excerpts

Tuesday 16th June 2015 Westminster Hall

[Greg Mulholland]

It is absolutely disgraceful and I urge the Minister to properly take that up. We have not had answers or justifications, although there can be no justification for NHS England behaving in that way. NICE’s decision not to recommend approving Vimizim in the short term has already been deemed to be flawed by those involved, including the MPS Society and clinicians, because it fails to consider BioMarin’s offer and has assumed that the cost of the drug will be the original £395,000. How has that happened? NICE also took months to put together the interim guidance, but has given only until next Tuesday to receive the extra evidence that it has asked for. Surely that is an unfair timeline for response.

As of 28 April 2015—which, incidentally, is a year after Vimizim was approved by the European Medicines Agency, meaning that it is approved in 20 European countries, including France, Germany and the Czech Republic—the drug was still not available in the UK, because NHS England has failed to put in place arrangements for funding it. Does the Minister not share the sense of frustration, anger and disbelief that the NHS refuses to fund the drug when so many of our neighbours do? More fundamentally, Earl Howe gave patients an assurance that their access to the drugs that they need would not depend on the cost per quality-adjusted life year measure. Can the Minister tell us why his Department has gone back on that assurance? That is exactly what it appears to have done.

I appreciate that the Minister has taken the time to meet us, but I remind him of the 11-page letter that he asked the organisations to send him some 11 weeks ago. We expected him to respond to that, as it was a complaint about NHS England’s handling of the matter, yet he simply passed it on for NHS England to respond to. That is not what we asked him to do, and the response does not address the points that we made to him, at his request, about how NHS England has failed people. I ask him again to reply directly and properly, and to investigate the mishandling of the situation by NHS England.

Duchenne muscular dystrophy has been mentioned. Again, I highlight the campaigning of organisations such as Muscular Dystrophy UK, Joining Jack, Action Duchenne, the Duchenne Family Support Group, the Duchenne Children’s Trust, Alex’s Wish and the Harrisons Fund. Those groups share the MPS Society’s frustration at the process. As many hon. Members know, Duchenne muscular dystrophy is a condition affecting only boys, and numerous potential treatments are in late clinical trial. Translarna, in particular, received conditional approval funding in the EU in August 2014. This clearly effective drug is being funded in a number of countries, including Greece, even given its economic situation, yet we are still no closer to hearing whether it will be funded here. I hope to hear positive news on that drug today.

I pay tribute to the Tuberous Sclerosis Association and the work of Jayne Spink and her colleagues. For those who do not know, tuberous sclerosis is a condition that causes the growth of tumours in organs, including the brain, eyes, heart, kidneys, skin and lungs, and a range of associated health problems, including epilepsy, learning difficulties and behavioural problems. The drug everolimus has been found to be effective in shrinking the tumours, extending life and improving quality of life, but although it was licensed for use in patients with tuberous sclerosis in February 2013, NHS England has failed to draw up a prescribing policy. At least two people have already died since the drug was licensed; Chris Kingswood, a consultant nephrologist, said that Julie Brooker’s death in January 2013 was “absolutely preventable” if she had been given access to everolimus.

[Jo Churchill (Bury St Edmunds) (Con)]

My constituent William needs that drug. The issue for his family is the timeline, which the hon. Gentleman mentioned. They have waited two years and been told that it may be another year, but they have said to me that William might not have that much time and that, like the woman the hon. Gentleman just mentioned, he might no longer be with them by then. Those parents are fighting for their son.

[Greg Mulholland]

The hon. Lady is right: none of these children or families has time. All those conditions deteriorate irreversibly. She is right that it has been 28 months since the drug was approved, yet patients are no closer to accessing it. What will the Minister do to speed up a commissioning policy for everolimus?

I turn to Batten disease, another condition already mentioned. I pay tribute to the Batten Disease Family Association. Batten disease is another condition that I had not heard of until I was approached by my constituents Duncan and Lynsey Brownnutt. I have been pleased to join Duncan to support some of his amazing fundraising efforts. This summer, he is off on a wonderful cycling trip to the Arctic Circle with his friend Rod to raise money, but the day after the general election, his six-year-old daughter Ellie Mae passed away from Batten disease.

Batten disease is another condition currently without any cure. It includes increasing visual impairment, complex epilepsy with severe seizures, decline of speech, language and swallowing skills, deterioration of motor skills resulting in loss of mobility and ultimately death. Potential treatment for Batten disease is not even being considered for 25 June. If the situation of the other conditions is still unclear and their drugs have been turned down, when will action be taken on treatment for Batten disease?