Drugs: Ultra-rare Diseases

David Simpson Excerpts

Tuesday 16th June 2015 Westminster Hall

[David Simpson (Upper Bann) (DUP)]

My hon. Friend mentions research. As he will know, I am involved in a campaign in Northern Ireland and across the United Kingdom on complex regional pain syndrome. The condition affects children, but it mostly affects adults from the age of 50 onwards, and people can lose limbs to it. One in every 3,000 people is affected, and many lives have been destroyed. We need more research to find a drug to cure this condition, and research funding needs to be put in place so that that research can be done.

[Jim Shannon]

I thank my hon. Friend for his intervention. That is a message that I, too, believe in, and I am sure the Minister will respond positively.

Leaving aside all the statistics we have heard today, we need to imagine the emotional strain these things put on people and their families, and we have had examples of that. Only 35% of patients are aware of a licensed treatment for their condition. There is something wrong when that is the case. How come only 35% of people know there is something there for them? How are the Government addressing that? I am not attacking the Minister—that is not how I work—but how do we move things forward in a positive fashion? Of that 35%, 89% are able to access the treatment, but 11% are not. Therefore, 65% of people are not aware of the drugs, and of the 35% who are, a proportion are not able to get them.

Like others, I want now to touch on Duchenne muscular dystrophy. If Translarna is given at the correct time, we can prolong the sufferer’s mobility. My constituents deserve to have access to that drug as soon as possible, and that is what I would like to see happen. The effects of long waiting times and uncertainty are widespread, and although ultra-rare diseases affect the few, their effects for those who suffer from them are an inescapable reality and should be treated with the utmost seriousness.

Families deserve a solution to the continual failure to establish a lawful, robust and transparent commissioning service that enables the rare disease community to access new drugs in an equitable and timely manner and to avoid situations such as those we have spoken about, where crucial windows of opportunity pass by. This is a crisis—it cannot be described as anything else. People are in trouble, and they need our help now.

Let me quickly pay tribute to the lady who looks after the Northern Ireland Rare Disease Partnership, Christine Collins. Last year, we met the Under-Secretary of State for Health, the hon. Member for Battersea (Jane Ellison), to discuss these matters. We were clearly moving forward, and the Minister was very responsive. The background information for the debate says that, in November 2013, the UK Department of Health and the devolved Governments published the UK strategy for rare diseases. In June 2014, the Northern Ireland Assembly endorsed it and gave a commitment to publish an implementation plan, and last year’s meeting provided an opportunity to underline the need for that to happen. Perhaps the Minister can give us some idea today of what discussions he has had with his fellow Minister to move things forward so that we can deliver on that commitment.

The debate has dealt with access to drugs. It has also given us an opportunity to bring out the gaps in the patient experience. Let us remember the patients, the families, the children and all those who suffer. They require a co-ordinated response from not only the health service and the social services, but research bodies and the relevant charities. I hope that the common experiences we have described signal the urgent need for access to these vital treatments. I remind all those in a position to have a tangible impact on drug access that while we are debating these issues, somebody else is falling into the trap and will, unfortunately, be unable to access the necessary drugs. I urge the Minister to respond positively, and I thank the hon. Member for Leeds North West again for giving us all a chance to speak about this issue.