A happier Christmas this year for sufferers of ultra-rare diseases

3 min read21 December 2015

Leeds North West MP Greg Mulholland writes at the end of 13 month campaign to get a drug approved which treats Morquio Syndrome, a rare disease affecting only 88 people in England.

For 88 people across the country, Christmas will be extra special this year. Last week, the National Institute of Health and Care Excellence (NICE), the national body which approves drug treatments for NHS England, finally approved the Vimizim drug. This drug is for treating Morquio Syndrome, an ultra-rare disease which only 88 people in England have. For the last 13 months, I have worked closely with families, charities and campaigners and we have battled to get this drug approved.

Morquio Syndrome inhibits growth of the skeleton but organs carry on growing which without treatment leads to deformities and ultimately to death, without treatment. Although one’s mental abilities are fortunately not affected, physical damage caused by this degenerative condition is irreversible and if left untreated, life expectancy of Morquio sufferers is just 25 years of age. With the UK being one of the pioneers of the Vimizim drug, with it having been approved by the European Medicines Agency in April 2014 and being available in over 20 other countries, it was baffling that the drug was not available in the UK.
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In addition to Morquio Syndrome, I learnt that people suffering from Tuberous Sclerosis Complex and a rare form of Duchenne Muscular Dystrophy were also waiting for the drugs they needed to be approved. That’s 218 people in total having to wait for the bureaucratic cogs to turn so they could live as normal a life as possible. And so the #FundOurDrugsNOW campaign was born.

The last 13 months have highlighted repeated failure of process from NHS England, NICE and the Department of Health. It began with a legal challenge from a Morquio sufferer that forced NHS England to scrap their flawed process for approving drugs. The following months saw repeated delays, a failure by officials to turn up to meetings or respond to correspondence, or to give clear and consistent information. That was 218 people and their families being let down time and again. In addition to raising the campaign at Prime Minister’s Questions three times in four months, I secured two parliamentary debates, met health minister George Freeman three times, sponsored five parliamentary motions and organised numerous rallies and protests outside Parliament, 10 Downing Street and the Department of Health.

What kept our campaign going was the sheer inspiration from those suffering with these ultra-rare diseases. Families were ready to beat down every door so their loved ones, already suffering enough, did not have to go through further pain when it could be avoided. Last week’s Vimizim announcement was fantastic news for those suffering from Morquio Syndrome, and for that, full credit must go to the families who fought so hard, the MPS Society, a fantastic charity representing Morquio sufferers, and to parliamentary colleagues who joined our campaign. Of course, drugs have not yet been approved for those with Tuberous Sclerosis Complex and Duchenne Muscular Dystrophy, so the #FundOurDrugsNOW campaign continues full steam ahead- and if there is one thing the last 13 months and the Vimizim result show, it’s that campaigning works.

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