Today I am leading a Westminster Hall debate on access to drugs for ultra-rare diseases. It is a matter I have campaigned on for the last seven months and, on seeing the number of people and families repeatedly let down by NHS England, is a cause I have grown increasingly passionate about.

I got involved after being approached by my constituents Katy and Simon Brown, whose six year-old son Sam is one of just 88 people in the country with the ultra-rare disease Morquio Syndrome. In December 2014, NHS England were forced to suspend their ‘scorecard system’ for deciding which drugs to prioritise for funding, on the basis that the system was discriminatory against those with ultra-rare diseases. Since then NHS England have failed to put an interim process in place. This has led to many people who suffer from ultra-rare diseases being left in the lurch about their future access to the drugs they need.

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.

 

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