11th March – Protest outside Parliament


11th March – Protest outside Parliament

The MPS Society, affected families and supporters, along with the Tuberous Sclerosis Association and other rare disease groups, stood in unison outside Parliament to remind David Cameron of the right to treatment that all people should have, regardless of the rarity of their condition. 


ITV News covered the protest and you can view their report here.

Below you can read the press release from Greg Mulholland’s office on this event:



Prime Minister must “fund our drugs NOW” say rare disease campaigners

Campaigners and families protested outside the Houses of Parliament today calling on the PM to stop letting NHS bureaucracy get in the way of funding for drugs needed by children with ultra-rare diseases.

The protest was led by a number of charities campaigning for access to the drugs these children desperately need. Charities included the MPS Society, Tuberous Sclerosis Association, Action Duchenne, Harrison’s Fund and others.

NHS England was forced to scrap its drugs commissioning process in December because of the threat of legal challenge that it was discriminatory. Until a new process is in place, campaigners are demanding NHS England provide interim funding to access the drugs Vimizim (to treat Morquio Disease), Translarna (to treat Duchenne Muscular Dystrophy) and Everolimus (to treat Tuberous Sclerosis). Just 180 children across the UK have these three ultra-rare diseases, some of them are seeing their conditions already worsening.

A number of MPs of all parties dropped by the protest to give their support- including Annette Brooke, Fiona MacTaggart, Russell Brown, Paul Farrelly, Alec Shelbrooke, Andrew Bingham, John McDonnell, Virendra Sharma, Mark Durkan, Sir Bob Russell, Dave Anderson and Vince Cable. 52 MPs in total have signed a parliamentary motion backing the campaign.

Greg Mulholland MP, who is leading the cross-party multi-organisation campaign, then questioned Prime Minister David Cameron for an unprecedented third time in just fourth months, and after PMQs took MPs to drop off a letter at 10 Downing Street to add further pressure on David Cameron. The letter was handed in by ten year-old William Reeve, who has Morquio Disease, and was accompanied by his mother Thelma.

It remains uncertain when a new commissioning process will be put in place. NHS England’s lack of response has already seen the drugs company BioMarin, which produces Vimizim, announce it will, as of 12th May, cease providing the drug for on a goodwill as it is currently doing. Patients on the drug now face no choice other than seeing their conditions worsen irreversibly after that date.

Speaking at Prime Minister’s Questions, Mulholland said:
“Mr Speaker this is the third time in four months I’ve raised this at Prime Minister’s Questions. NHS England letting down the 180 people with ultra-rare diseases, some of who are outside the House today. Failed by the flawed process, some of those children will lose access to their drugs from May and their conditions will deteriorate irreversibly. We have two Prime Minister’s Questions left. Can he tell me, in that time, will he announce when we will get interim funding for these drugs that the children need?”
Responding, Prime Minister David Cameron said:
“My honourable Friend is absolutely right to raise this issue because these are very rare and debilitating conditions and there are drugs that can help the children that have these conditions. My understanding, having looked at this, I know that the life sciences minister has looked very carefully, met with the families and with the drugs companies as well as with NHS England. My understanding is that NHS England is holding a review. The review will be completed by end of April and the companies are currently funding these drugs till the end of May. So I don’t see any reason why there shouldn’t be continuity of care and continuity of drugs. That is what I hope we can achieve.”
Commenting afterwards, Mulholland said:
“These 180 children and their families have been let down and fobbed off by NHS England for too long and just aren’t getting answers from NHS England. Campaigners and the children who were there today were heartened by the many MPs who gave their support today, and then joined me to hand in a letter at 10 Downing St. We will keep up the pressure until we get the interim funding these children desperately need. I urge the Prime Minister and ministers to act and ensure this interim funding decision is announced before Parliament dissolves at the end of March.”
Christine Lavery, Chief Executive of MPS Society, also added:
“NHS England, instead of recognising their failure to create a system to assess prioritisation of interim funding fair to ultra-orphan diseases continue to occupy their time misguidedly trying to deceive the very people they are empowered to serve ‘patients’. Patients include children and young adults with extremely rare diseases. We prevail upon that the Prime Minster, as the father of a son with a very rare disease, to act NOW to ensure interim funding is confirmed from the 12 May 2015 when free Vimizim is no longer available. Please put yourself in the shoes of these children, adults and their families.”
Paul Lenihan, Chief Executive of Action Duchenne, added:
“At the lobby event in January, I spoke about moral courage and I encouraged those in NHS England to have the courage to make a decision for our community. PTC Therapeutics have done this by making Translarna available to siblings of those who are already being treated by the EU- approved drug. We still await responses of our formal applications for NHS England to do the same: make a decision. That their decision was to delay the decision until May displays a lack of moral fibre by the key decision makers within NHS England as we already know that all the information required to make funding decisions is sitting within it.”
Jayne Spink, Chief Executive of the Tuberous Sclerosis Association, further added:
“It’s totally outrageous that the government seems content for bureaucratic delays to expose patients to avoidable harm, including exposing some to the risk of an avoidable death. In the case of Tuberous Sclerosis, we have a proven treatment that is widely available in other countries, sitting on shelves here in England. These are real people; children, parents, brothers, sisters. People who cannot and should not continue to wait for the medicine they so badly need.”
Emma Hallam, Founder and Director of the Alex’s Wish charity, added:
“Boys currently eligible may not be eligible much longer as their disease progression in inevitable. We hope David Cameron takes notice and makes a decision to provide interim finding for Translarna before time runs out!”