Liberal Democrat MP for Leeds North West Greg Mulholland on Tuesday (24th March) raised six year-old Sam Brown’s case at Deputy Prime Minister‘s Questions and then led campaigners in a further protest outside the Department of Health to keep up the pressure on ministers to act.

Campaigners outside the Department of Health on March 24th. In front of the banner is six year-old Harvey Brown, who is not receiving the drugs he needs

Speaking in the House of Commons, Greg Mulholland urged the Deputy Prime Minister, Nick Clegg, to intervene to help get the drugs for the 180 patients in total across the UK affected by Morquio Disease, Duchenne Muscular Dystrophy and Tuberous Sclerosis.

Greg Mulholland went on to say:

“The Prime Minister said that there should be continuity of treatment, yet we have found out that that will not be delivered by the Department of Health. Katy Brown, the mother of my six-year-old constituent Sam Brown, has said that that is at best “misleading, at worst underhand”. This situation is disgraceful. We need to fund those drugs now on an interim basis.”

Greg Mulholland has also raised the matter three times in the last four months directly with David Cameron at Prime Minister’s Questions.

Responding, Nick Clegg began by paying tribute to Greg Mulholland “for the way he has sought to represent his constituent Sam Brown, and all the other children and their families“. Nick Clegg committed to “undertake to look urgently at the matter“.

Afterwards, Greg Mulholland led campaigners from the MPS Society, a charity supporting those with Morquio Disease, and the Tuberous Sclerosis Association, in a protest outside the Department of Health, with a ‘Keep Sam Smiling’ banner and photos of some of the 180 patients with one of the three ultra rare diseases in questions.

Speaking afterwards, Greg Mulholland said:

“After raising Sam Brown’s case with the Deputy Prime Minister today, I welcome his commitment to urgently look at the situation, but this has now been going on for far too long, Sam is one of 180 people around the country being let down by NHS England. It was NHS England whose process for commissioning drugs had to be scrapped, so the moral and potentially legal responsibility is on them to sort this out and put interim funding in place for the drugs these children need, yet they are hiding behind bureaucracy and not giving answers to the children and their families. This afternoon, campaigners and I protested outside the Department of Health to keep up the pressure. We are meeting the life sciences minister, George Freeman MP, this Thursday where we will push for the minister to make an announcement for interim funding as soon as possible.”

Sam Brown’s mother Katy Brown has also called out the Prime Minister for using “underhand tactics”. At PMQs on 11th March David Cameron said in response to Greg Mulholland‘s question that “I do not see any reason why there should not be continuity of care and continuity of drugs“. However in a letter to the mother of another Morquio patient, Cameron also says “…provided the drug company continue to supply the drug until NHS England is ready to make a decision“. This suggests NHS England might not make a decision by 12 May- the date from which drugs company BioMarin will cease supplying Sam’s drug to him for free- and will push for BioMarin to carry on supplying the drug at their own cost, with NHS England then blaming BioMarin if the latter refuses to do so. The words “continuity of care” are also highly significant since this strongly suggests those not currently receiving the drug for free will be excluded from a potential arrangement.

Sam Brown’s mother Katy Brown also said:

“NHS England have significantly failed in their duties. Two years on and there is still no process in place for ultra rare diseases. The Department of Health has a duty of care to step in NOW to prevent more lives being lost and destroyed. Their refusal to do so under the guise of the need for proper process is laughably ironic given a complete lack of proper process is the reason we are in this sorry mess. It is time to stop hiding behind vague words and face the truth. Sam deserves far better than this as does every single sufferer. I ask all involved to pause today and take a long hard look in the mirror. Look into the eyes of your children. Imagine it were them in this dreadful situation. Ask yourself if you as their mother or father had experienced failings of this magnitude, would you still be making misleading statements? Using stalling tactics in the hope that a problem will go away? Pushing blame elsewhere? Or would you be fighting hard for the treatment your children deserve? You can make this right.”

Charlotte Roberts, Communications Officer at the MPS Society and who also attended the protest, added:

“Thank you once again to Greg calling for answers to give children and young adults the chance of life. It is these affected individuals who are suffering the consequences of NHS England’s process failures which is deplorable and must be addressed. As if that wasn’t enough these individuals and devoted parents of affected children with Morquio have put their lives on hold to fight for treatment that will stop progression of the diseases and give these young people the chance to live into their 30s, 40s, 50s, 60s and 70s. An interim funding decision must be found.”

Jayne Spink, Chief Executive of the Tuberous Sclerosis Association (TSA), also at the protest, further said:

“Tuberous sclerosis physicians and the TSA have been battling for two years to get the NHS bureaucracy to review the highly effective and safe drug called Everolimus. Children and young adults, who if they lived in another developed country would be treated and restored to good health, are instead facing imminent danger of irreversible and severe decline in health or death. And this is entirely preventable. It’s high time that Ministers recognised the gravity of this situation and lived up to their responsibilities to these families. Time is not on the side of our young people; ministers have an ethical and moral responsibility to act now.”