Greg Mulholland MP with six year-old Sam Brown, and parents Simon and Katy. 28/01/2015

Liberal Democrat MP for Leeds North West Greg Mulholland will today lead campaigners in a protest outside the Houses of Parliament to up the pressure on NHS England to fund much-needed drugs for those with ultra-rare diseases.

The protest will be 11am-12pm outside the St Stephen’s Entrance to the Houses of Parliament and is expected to be joined by a number of charities supporting those affected by the ultra-rare diseases in question, including MPS Society (supporting those with Morquio), Action Duchenne (supporting those with Duchenne Muscular Dystrophy) and others.

It follows as MP Mulholland will, for an unprecedented third time in just four months, raise the matter with David Cameron at Prime Minister’s Questions. 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 in the UK have these three ultra-rare diseases.

This is a long-running issue which, in December 2014, saw NHS England suspend their scorecard system for deciding which drugs to fund, after the threat of a legal challenge that it was discriminatory against drugs for ultra-rare diseases. The challenge was brought by ten year-old Kamal, who has Morquio Disease, a condition which only 88 people in the UK have.

Mr Mulholland‘s six year-old constituent Sam Brown also has Morquio. On Wednesday, 28th January, Sam travelled to 10 Downing St with his parents Katy and Simon to hand in a letter and petition calling on the Prime Minister to act urgently (photo attached).

However it remains uncertain when a new system 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 free as it is currently doing. BioMarin had been providing Vimizim on a goodwill basis to patients on a trial, including Sam. If Sam ceases to get the drug he needs and NHS England does not act, his condition will deteriorate and the damage will be irreversible.

In October, BioMarin had offered NHS England a capped price over five years for providing Vimizim but never heard back despite repeated follow-ups. Campaigners are demanding NHS England act and provide interim funding for Vimzim, Translarna and Everolimus until the new system is put in place for deciding which drugs to prioritise for funding.

Mr Mulholland has previously raised the matter at PMQs on 3rd December and 4th February. He has also met health minister George Freeman on 9th December and 18th December, along with campaigners, families and drugs companies. On 20th January he led a parliamentary debate on this issue, and has since tabled an Early Day Motion (EDM 819) which has been supported by 50 MPs in total so far. And just recently on 25th February, he questioned Secretary of State for Health Jeremy Hunt at a select committee meeting, demanding to know how Hunt was holding NHS England to account on this matter and if he would intervene personally.

Commenting, MP Mulholland said:

“These families and charities have had enough of being let down and fobbed off over the failure to approve funding for the life changing drugs these children need.

“They are we are consistently getting no real responses from NHS England or from Ministers, all of whom are hiding behind the ongoing consultation for a new system and even then, it is not clear when funding could be approved for the three drugs these children need and how long this will take.

“It was NHS England’s who had to scrap their process in December due to it being flawed and discriminatory, so the moral as well as legal responsibility is on them and the Department for Health to put in place interim funding now to pay for these until a they can establish a new, fit for purpose system for approving drugs longer term, that should have been there in the first place.

“So the Prime Minister must now personally intervene and with less than three weeks before Parliament dissolves, we need a decision on interim funding before then. People including children will soon lose access to the drugs they need and their conditions will deteriorate irreversibly as result and for that to happen due to a lack of leadership and accountability is appalling so we need an announcement on interim funding for these drugs now”.

Sam’s mother, Katy Brown, added:

“We need answers from NHS England and ministers now. We are sick of fobbed off and really need an interim solution now, otherwise Sam will stop getting his drugs after 11th May and his condition will worsen. The situation is simply not acceptable.”

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 12th 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:

“I am absolutely delighted MP Greg Mulholland is taking this issue to Prime Ministers Questions tomorrow, boys with Duchenne do not have time to waste. 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!”