LibLink: Greg Mulholland MP: Drugs for ultra-rare diseases

Yesterday, Greg Mulholland held a Westminster Hall debate after constituents told him that the provision of drugs for their son was under threat because he has a very rare condition. You can read the transcript here.

People with rare conditions not only have to fight their illness, but also, because it’s rare, there is unlikely to be much in the way of research. Friends of mine have raised tens of thousands of pounds for research and to support other families after their baby son died of a rare neurological condition. Another friend set up a charity that brings together sufferers of her daughter’s rare disease from around the world every year and has worked to bring together specialists from various parts of the world.

There are huge question marks over funding for drugs for rare conditions after NHS England’s system was found to be discriminatory. Greg wrote for Politics Home explaining the issues and setting out what he wants the minister to do:

Through the #FundOurDrugsNOW campaign, I have campaigned for 180 people across the UK with Morquio Syndrome, Duchenne Muscular Dystrophy and Tuberous Sclerosis Complex- 180 people who have been let down by this failure of process.

Also worrying is the lack of accountability from NHS England. Ministers have repeatedly said this is a matter for NHS England, yet NHS England have repeatedly failed to turn up to key meetings or respond to correspondence properly and in a timely manner- on Friday I finally received a response to a letter I sent them 11 weeks ago!

There is now real urgency to this issue. NHS England will announce on 25th June whether to grant interim funding for the drugs Vimizim (for treating Morquio Syndrome), Translarna (for treating Duchenne Muscular Dystrophy) and other drugs. My constituent Sam Brown has been receiving his drug Vimizim for the last three and a half years for free from the pharma company BioMarin. If the 25th June meeting decides against funding Vimizim, my constituent Sam Brown’s drug supply will be cut off and his condition will begin to worsen.

This debate is both about holding both NHS England and ministers accountable, but also about trying to work out a long term solution so that those with ultra-rare diseases can receive the treatments they need without discrimination, or uncertainty. As much as it is important for the NHS to act in a transparent, efficient, and professional manner, ultimately, the people who are hurt most by this situation are those being denied the drugs they need for their ultra-rare diseases.

You can read the whole article here.

The Minister said a lot of supportive things but didn’t really offer any solutions to the problems families were facing. He didn’t seem to tell them anything they didn’t already know. For some of them, the announcement of the decisions on the future supply of the drugs they need will be a matter of life and death.

* Caron Lindsay is Editor of Liberal Democrat Voice and blogs at Caron's Musings

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5 Comments

  • From the debate transcript : (16 Jun 2015 : Column 36WH)
    “The list price for Vimizim is £395,000 per person, per year”
    This is an emotionally charged, and desperate situation for the family, but someone has to make a decision on affordability within an NHS budget, and I cannot believe that NICE are making decisions on a crude discriminatory basis? As difficult as it is to eliminate emotion from our thinking, we must,.. (or someone on our behalf must), ask the valid question,.. Has the present NHS funding, got the ability to cover that cost (x 88 patients)? And if the funding simply isn’t there, where are you proposing to make health cuts if you wish to cover it?

  • Why does having a rare disease make your suffering more important than the suffering of someone else who has a more common disease? You’re arguing for money to be spent to help less people, less effectively.

    NICE’s system of assessing drugs based on the benefits is a marvellous feature of our healthcare system and one that deserves our defence and support not hectoring with cheap emotional arguments. However healthcare money is spent, you will always be able to find noble causes of suffering children to roll out and slam those in charge of deciding how to send a finite pot of money but the truth is that favouring these individuals over optimum spending strategies is neither compassionate or humane. It is trading the suffering of those with good PR for greater suffering among those that are less heard from.

    There is a separate issue with research funding for rarer diseases, one which argues for greater government involvement in research funding and less reliance on the charitable and private sectors. And also for research funding to be better allocated according to need. We need to stop spending so disproportionately much on cancer research and bring our spending better into line with the actual burden of disease.

  • John Tilley 18th Jun '15 - 7:50am

    Caron,
    Your assertion that —
    “…There are huge question marks over funding for drugs for rare conditions after NHS England’s system was found to be discriminatory”

    Is this really the situation?
    Or is it just criticising those who have to make the difficult decisions loaded on them by ministers.

    Perhaps we should ask a former junior minister in the Department of Health? I am sure he will remember the briefing provided which might have been something along the lines of —
    The Coalition Governent has set a budget for the NHS. Expenditure on drugs is “x”.
    To fund this super new neurological drug at a cost of £400,000 per patient per year would mean you could kiss goodbye to doing anything new on mental health.
    You have already cut the budgets for mental health each year that you have been a junior minister.
    So your choices are —
    A). Increase the drugs expenditure to pay for this new drug whilst further cutting other NHS budgets
    Or
    B). Shift the blame onto NICE and go to your annual party conference and make a grand gesture statement about future spending on mental health.

  • @Jack – there is in fact rare medicine/orphan drug legislation allowing a little deregulation to encourage drug research into these rare but debilitating diseases. The Labour peer Anne Begg takes some medicine developed from it, if I recall correctly. I think this indicates a balance struck between utilitarian arguments (money based on health burden, help the most people) versus moor idealistic arguments (no-one should be prohibited from living a good life by suffering from treatable disease). I’d imagine both resonate with most people and I think the idea of balancing the two is correct. A purely utilitarian view would probably mean no treatment for these genetic diseases.

    I’m not saying anything about this specific case, I don’t have enough information, but I’m just saying there is a case to be made and it could be argued that Greg is doing his job as a constituency MP by doing it.

  • This article also opens another avenue that no-one really wants to acknowledge, in that there simply isn’t enough money in the system [NHS], to treat all comers with all diseases and injuries, from cradle to grave, and we’re all going to have to face that annoying reality.?
    To save money, and re-route NHS funding it was declared that Nigel Farage, was using the emotional ‘dog whistling’ of hate, when he suggested that the NHS should stop funding the long term health needs of ‘health tourists’, instead asking them to pay for their first 5 years of residence with health insurance cover. And yet this article seems to be blatantly using the same technique, by using a vulnerable child, as a ‘dog whistle’ for sympathy and a re-routing of funding.
    The broader take-away from this article (for us all), is that the NHS is clearly beginning to enter an unwelcome but inevitable era of ‘funding triage’, and we’re going to have to make some very ‘uninvited’, but bold decisions in both the near and long term health care of the Nation.?

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