Drugs: Ultra-rare Diseases

Jim Shannon Excerpts

Tuesday 16th June 2015 Westminster Hall

[Jim Shannon (Strangford) (DUP)]

First, I congratulate the hon. Member for Leeds North West (Greg Mulholland) on bringing this matter to Westminster Hall for consideration. Westminster Hall is well filled today because we all have constituents who are suffering and do not have access to the drugs needed to combat these rare diseases. I also congratulate the hon. Gentleman on his hard work on this issue, for which he is well renowned; we have all said that, but it is the truth, and we all want him to know that we know it.

I am glad this debate has occurred, because it is on a subject that affects many people in my constituency. We have heard some stories and we will hear more before this debate is over.

The diseases we are considering may be rare, but collectively they affect the lives of 3 million people across the United Kingdom. That emphasises that everything must be done to create a comprehensive initiative for providing care to those affected by these difficult and challenging diseases.

Rare diseases tend to be life-threatening or chronically debilitating. There are between 6,000 and 8,000 rare diseases. Each one affects less than 0.1% of the UK’s population, but Rare Disease UK calculates that 75% of these illnesses affect children.

We are here today on behalf of our constituents, but we are also focusing very much on young people across the United Kingdom of Great Britain and Northern Ireland who have these problems.

The ultra-rare diseases that have been mentioned include Morquio disease, Duchenne muscular dystrophy and tuberous sclerosis. I would also add Prader-Willi syndrome, which some of my constituents suffer from.

The chance of improving people’s quality of life depends very much on a narrow timescale. It requires quick diagnosis, treatment and drug provision, so that drugs can be accessed when they are proven to be most effective. In other words, as every Member who has spoken has said, time is of the essence—the people who are suffering need help now, not in six or 12 months. It is our duty to make that timeline as transparent and effective as possible within the finite resources we have, and I understand the problems the Minister has. There must be adequate assistance for practitioners, to allow for timely diagnosis and the timely provision of drugs and treatment.

[Mr Anderson]

The hon. Gentleman has been very consistent on this issue, and he is right: as those of us in the all-party group on muscular dystrophy have found, one of the main reasons for delays is that clinicians—particularly GPs—do not see these diseases very often, and when they do, they are sometimes lost as to where to go. Once a disease is diagnosed, the people suffering from it should have no worse access to treatment than people with much more common diseases—surely that is the issue that has to be addressed. Once a disease is identified, we have to get to grips with it, and people have to get the medication and the support they need, so that they can get on and live the best life they can.

[Jim Shannon]

I agree wholeheartedly. I am sure the Minister has heard us all say that time is of the essence and that we should strike right away. That is what we are about.

The health and social care professionals involved in the diagnosis, treatment and care of these patients face difficult tasks. As I was saying, there must be adequate assistance for them, to allow for timely diagnosis and the timely provision of drugs and treatment. There also needs to be sufficient funding UK-wide.

In Adjournment debates and other debates about these issues, I have always referred to Queen’s University in Belfast and to the importance of research and development. Queen’s University is one of the universities that do research, and it works in conjunction with the Health Department. Perhaps the Minister could therefore give us some idea what the Government are doing on research and development to ensure that new drugs are found.

[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.

[Several hon. Members]

rose—

--- Later in debate ---

[Dr Whitford]

Obviously, devolution gave us the power to do things differently, but I do not think that we should re-invent the wheel. Often, we will accept work done by NICE or re-evaluate it quickly, to see whether things should be applied differently, but we do not just go back to the beginning. However, I am sure that ideas can be shared in both directions.

It seems that certain drugs were left as orphans when the system changed. We know that patients with the brain tumour form of tuberous sclerosis, which the hon. Member for Carmarthen East and Dinefwr (Jonathan Edwards) mentioned, can access the drug through the cancer drugs fund, but if they have a kidney tumour and are treated by urologists they are not part of that system and simply will not be aware of it. Such random unfairness exists.

[Jim Shannon]

There is a forum and association, driven by the Health Minister, that discusses matters together with the three regions. A UK-wide strategy is already in place. The process is allowing that to happen already. However, it is delayed and has not happened yet; that is why we are concerned.

[Dr Whitford]

I think it is a matter of what ideas go on the table and what is being discussed in the meetings. Good ideas are going ahead. I commend the idea of including patients and clinicians in evaluations, because the numerical data from trials will often be small due to the nature of the diseases in question, and we will have to look wider. The problem for children is that if these drugs are to prevent deformity, they have to be got in early. People with Morquio already have the changes. We do not know yet how much change could be prevented, or how much saving there could be on a person’s disability in the long term if metastatic breast cancer treatments, which eventually become adjuvant treatments, are given earlier.

I commend the system I have talked about. I know it is difficult and challenging, but it is clearly fair, with an interim period for compassionate reasons, and people know where their voice should be heard.