In his campaign to get drugs funding for patients with ultra-rare diseases, after his meeting yesterday with life sciences minister George Freeman, Liberal Democrat MP for Leeds North West Greg Mulholland declared himself “disgusted” at the way NHS England have acted towards families, charities and by misleading ministers. He called on health ministers to use the powers explicitly available to them and intervene in the matter.

Greg Mulholland is campaigning for NHS England to announce interim funding for the just 180 people across the UK who are affected by the ultra-rare diseases Morquio disease, Duchenne muscular dystrophy and Tuberous sclerosis. This includes Greg Mulholland‘s own constituent, six year-old Sam Brown from Otley.

It is a matter Greg Mulholland has raised three times with David Cameron at Prime Minister’s Questions over the last four months, questioned the Secretary of State for Health Jeremy Hunt at a select committee meeting, and just this week raised it with Deputy Prime Minister Nick Clegg, who agreed to look urgently at the matter. He has also led protests- including families, charities and MPs of all parties- outside Parliament, Department of Health, and going to Downing Street.

Greg Mulholland secured yesterday afternoon’s meeting with life sciences minister George Freeman MP, and took along representatives from the charities MPS Society (supporting those with Morquio disease), Tuberous Sclerosis Association, Action Duchenne, Joining Jack, Duchenne Children’s Trust, Muscular Dystrophy UK, and drugs companies BioMarin (who produce Vimizim, the drug to treat Morquio) and PTC (who produce Translarna, for treating Duchenne muscular dystrophy).

However, despite receiving a face-to-face assurance on Tuesday from NHS England official Anthony Prudhoe that they would be present at the meeting, NHS England failed to show up. During a campaign in which ministers have repeatedly said the funding decision is for NHS England, and with NHS England failing to respond to correspondence on many occasions and failing to send representatives to meetings, Mulholland said he was “disgusted at NHS England cowards for failing to be answerable for their own mess.”

In early December, NHS England suspended their process for commissioning drugs for ultra-rare diseases because of legal action that it was discriminatory. While consulting on a new process, they have failed to put an interim process in place. Furthermore, in August 2014 drug company PTC made an offer to supply Translarna to NHS England, and in October BioMarin did the same for supplying Vimizim- NHS England have repeatedly failed to respond to either company’s offer. BioMarin are currently supplying the drug for free, on a goodwill basis, to patients on a clinical trial- with NHS England’s refusal to respond to the offer, BioMarin announced they would be withdrawing the free supply of Vimizim from 12th May onwards- to which NHS England replied threatening legal action despite failing to respond earlier.

During the meeting yesterday with the life sciences minister, it emerged that NHS England had clearly been misleading the minister, who was told by NHS England that they were in “active discussion” with BioMarin. The BioMarin representative at the meeting, UK/Ireland Country Director Nigel Nicholls, told the minister this was not the case.

The minister was also found knowing little about his powers to be able to intervene. MP Mulholland quoted at Mr Freeman directly from the Department of Health’s Framework Agreement with NHS England, paragraph 4.11.3, which says: “If the Secretary of State considers that NHS England is significantly failing in its duties and functions he is able to intervene and issue directions to NHS England“.

Greg Mulholland has also tabled a parliamentary motion, Early Day Motion 918, calling on Jeremy Hunt to use this power explicitly available to him and intervene. The cross-party motion, already signed by ten MPs, also refers to a select committee meeting of 25th February at which Mr Hunt, responding to a question from Greg Mulholland, says in reference to the Framework Agreement that “if NHS England is failing on that, we can hold them accountable on that.”

Commenting after the meeting, Greg Mulholland said:

“We have ministers saying the decision is not down to them, but rather NHS England. But NHS England are repeatedly failing to reply to letters or even turn up to meetings despite Anthony Prudhoe telling me they will attend, and ministers are wrongly claim they are unable to intervene when their power to do so is set out clearly in black and white. The whole affair is a total shambles, especially given we are here because NHS England’s own process was discriminatory and they were forced to scrap it. I am disgusted at the NHS England cowards for failing to be answerable for their own mess, and for completely letting down the 180 people who desperately need treatment. Ministers absolutely must intervene, and with people’s lives at stake, they must do so now.”

Action Duchenne CEO Paul Lenihan added:

“We are appalled at NHS England’s failure to attend yet another meeting with those charities whose members are most affected by the failure in the clinical commissioning process. The fact that the NHS were excused from the meeting by a Minister of State shows an atrocious lack of awareness, and that this Minister is being misled and misinformed by senior members of the NHS.”

Charlotte Roberts, Communications Officer at MPS Society, also added:

“Thank you to Greg for ensuring that a third meeting took place with the Minister George Freeman. We are hugely indebted for his tireless support both to his constituent Sam Brown and for all those affected by this debacle within the rare disease community. NHS England are misleading both the Minister and Prime Minister that they are consulting on a process but there is still no transparency on the assessment criteria they are going to use. They are also misrepresenting the notion of ‘active discussions’ with the pharmaceutical companies and need to be held to account for their shambolic approach to decision making. Whilst there is no process, no clarity, no transparency and no accountability it is the children and their families who are suffering as a consequence. We have four days left until Purdah and desperately hope that the Government demonstrate that when process fails they have a duty of care and intervene before it’s too late.”

Alex Johnson from Joining Jack and Emily Crossley from the Duchenne Children’s Trust said:

“It is disgraceful that – for the second time this year – NHS England failed to send a representative to meet with patients to discuss their review of Translarna. Their process of approving funding has been nothing short of a shambles, and this view is held not only by us but by the UK’s leading DMD clinician, Professor Kate Bushby. We urge the Life Sciences Minister George Freeman MP to address our grave concerns directly with NHS England for them to resolve this farcical situation.”