Drugs: Ultra-rare Diseases

Rehman Chishti Excerpts

Tuesday 16th June 2015 Westminster Hall

[Greg Mulholland]

The recommendation from NICE is strange—I will come on to that—given that, clearly, the drug is effective.

Sam and other children and adults with Morquio disease are not the only people being let down. There are other conditions. I have been working with Members and organisations on the mutation of Duchenne muscular dystrophy and tuberous sclerosis. We have come together to campaign as one to say that we need a better way of approving drugs for ultra-rare conditions. At the moment we have a system in this country where people with ultra-rare diseases are discriminated against, and that must stop.

[Rehman Chishti (Gillingham and Rainham) (Con)]

I pay tribute to the hon. Gentleman for the work that he has done and for securing this debate. On other rare conditions, I have a very sick two-year-old in my constituency who suffers from neuroblastoma, a rare form of cancer that only 100 children suffer from each year. It is difficult to accept that my constituent has to raise money and travel to the United States to get treatment. We should ensure that children or anyone suffering from rare conditions, such as Ruby Young and those in the hon. Gentleman’s constituency, get the treatment they need at the first port of call in their own country.

[Greg Mulholland]

The hon. Gentleman is right to say there are other such conditions. I will not be able to mention them all today, but other Members may wish to do so. I will concentrate on the three conditions that I have been working on: Morquio, Duchenne and tuberous sclerosis. Some 180 people suffer from those conditions. I am sorry to say that all those people and their families have been hugely let down by the repeated failure of process by NHS England and by the thick wall of bureaucracy and utter lack of accountability.

--- Later in debate ---

[Greg Mulholland]

The Batten Disease Family Association explained that to me when I met with representatives, but unfortunately that is not even in the consideration for 25 June. That is why we need an overhaul.

We have a five-year Parliament. I hope that the Minister will serve as the Life Sciences Minister for a considerable time, if not for the whole Parliament. His challenge as the Life Sciences Minister, as well as dealing with the accountability deficit that clearly exists in NHS England’s decision making, must now be to initiate a proper process for the approval of drugs for rare conditions. Of course there are cost implications, and of course drugs must be effective, but the situation is that there are effective drugs that this country is not funding, while other countries with less strong economies are finding the money in their health services to fund them.

[Rehman Chishti]

The hon. Gentleman talks about funding, but one aspect that precedes funding is awareness of such diseases. For example, the Government’s “Be Clear on Cancer” campaign does not take into account rare conditions and cancers such as neuroblastoma, from which my constituent, who is near death, is suffering. The Government must ensure that rare conditions are part of the bigger campaign, so that the people suffering from them get the help that they need as well.