Treatment for rare disease gets official sign-off
After more than a year of campaigning, an Otley schoolboy living with an ultra-rare disease has finally had the drug he needs signed off by health authorities.
Sam Brown is one of just 88 people in the UK suffering from morquio syndrome.
They've been lobbying NHS England to fund vimizim - the only treatment available to them.
Last month, NHS England and NICE announced they had secured a five-year, fixed-fee agreement with the drugs company BioMarin, and today that agreement was officially signed off.
Morquio sufferers will now need to visit one of the eight metabolic centres around the country and get a clinician to confirm their diagnosis. They will then undergo baseline testing, which will be used to measure their progress annually over the next five years.
After the visit and tests have been done, NHS England will release funding for the Vimizim drug.
Leeds North West MP Greg Mulholland led the #FundOurDrugsNOW campaign and worked closely with families and charities, including the family of Otley seven-year-old Sam Brown.
The drug was approved on a Europe-wide level in April 2014 and is already in use in 20 other European countries, but attempts to make it available in England have seen repeated delays.
In the last 13 months, Mr Mulholland has secured two parliamentary debates and raised the matter three times with David Cameron at Prime Minister's Questions.
There have also been a number of rallies outside Parliament, the Department of Health and 10 Downing Street.
"This is exactly the news we wanted and I am delighted for Sam and for all the people with Morquio who will now get access to the treatment they need," Mr Mulholland said.
"It is a great victory for people power and all those involved in the campaign including the MPS Society and families up and down the country.
Sam Brown’s mother Katy added: “**We are relieved and delighted by today’s decision, and would like to thank Greg for his unwavering support.
"Without him, and the work of the MPS Society, none of this would have been possible. We would also like to thank our local community for the incredible and humbling support that we have received over the past year. We are now looking forward to a relaxing family Christmas, celebrating this victory and a much brighter future for Sam.”