Drugs: Ultra-rare Diseases

David Anderson Excerpts

Tuesday 16th June 2015 Westminster Hall

[Greg Mulholland]

Indeed. It was a pleasure to meet the right hon. Lady’s constituent, Archie, and his parents. These young people are inspiring us to campaign. She is absolutely right. We are debating the European Union Referendum Bill today in the Chamber. Other EU countries, and some non-EU countries, regard these treatments as effective and affordable, yet we do not.

I will fast-forward from the scrapping of the previous body to October 2014, when NHS England came out with the scorecard system. That is despite one of the clinicians involved, Dr Chris Hendriksz, saying on 22 October in an email:

“I would suggest the scoring is not used at all for decision making this round and I would rather have people acknowledging that they are making random decisions than to try and give some credibility to a process that was deeply flawed.”

That is from one of the senior clinicians.

NHS England none the less went ahead with the scorecard system to decide which funding should be prioritised. Suzanne Mallah and her 10-year-old boy Kamal, who has Morquio and is another inspiring young person whom I have been delighted to meet, saw that that was not only haphazard but discriminatory. With the help of the MPS Society, they threatened legal action on 28 November against NHS England on the basis that the scorecard was clearly discriminatory, that there was no policy explaining it and that there had been no public consultation on its use. Just one week after that, on 2 December, NHS England announced that it was suspending use of the scorecard because the MPS Society and Kamal were right and it was wrong.

[Mr David Anderson (Blaydon) (Lab)]

The hon. Gentleman makes a good case. Is it not also the case that clinicians have not been listened to all the way through this, in the same way that they were not listened to when the Health and Social Care Act went through? That is what has led us to where we are. I have been the chairman of the all-party group on muscular dystrophy for 10 years. We had a very good working relationship with the specialised commissioning groups, which were effective in getting medication of this type to people, but the bureaucracy created by the Act was against clinicians’ wishes, which is why we are here today. NHS England has a lot to answer for. The Government’s decision to ignore the voice of professionals has put us in this position.

[Greg Mulholland]

It has been a pleasure to work with the hon. Gentleman and the APPG on muscular dystrophy on the Translarna part of the campaign. He is absolutely right. We want not only an acknowledgment from the Minister that the current processes are not fit for purpose and not fair on those with ultra-rare diseases, but a drive to overhaul them.

--- Later in debate ---

[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.