NHS England & NICE’s Catalogue of events

NHS England & NICE’s Catalogue of Events

Why it’s essential that MPs and Ministers step in


• In 2013, Government abandoned AGNSS – the expert body that had previously advised on which specialist treatments and services should be made available for people living with rare diseases in this country.

• A twin-track system was created involving committees within both NHS England and NICE.

• NHS England’s new process for the evaluation of medicines for rare diseases had not been subject to any public consultation.

• On the 28th April 2014 after 5 years of clinical trials in children and young adults affected by Morquio disease (MPS IVA) the drug Elosulfase alfa, was licensed as Vimizim by the European Medicines Agency (EMA). This is the only treatment currently available for MPSIVA Morquio. The UK had 30% of the patient population enrolled on the clinical trial.

• The pharmaceutical company BioMarin have continued to provide Vimizim free of charge to all 34 UK patients enrolled on the clinical trial after the drug was licensed by the EMA in April 2014.

• 21st November 2014 a judicial review by a ten year old boy with Morquio supported by the MPS Society forced NHS England to halt all decision making by this new system after a letter before proceedings was sent alleging the NHS England scorecard was unlawful and discriminatory to people with ultra-rare diseases. The scorecard had not been published and there had not been any public consultation over its use. NHS England responded agreeing that the scorecard was unlawful and deferred the decision making process.

• 25th November 2014 Greg Mulholland MP raises the uncertainty about funding for a family in his constituency who live in a state of anxiety because they do not know whether the drug Vimizim will be approved for further use.

• 2nd December 2014 NHS England reveals it has scrapped key meetings on whether to fund a series of treatments for serious conditions following the threat of legal action.

• 3rd December 2014 Greg Mulholland MP raises the matter of the legal challenge and lack of process to David Cameron in Parliament.

• On the 9th December 2014 Life Sciences Minister George Freeman met with Greg Mulholland MP, three families of children with Morquio and Duchenne Muscular Dystrophy and the two patient organisations the Society for Mucopolysaccharide Diseases (MPS Society) and Muscular Dystrophy Campaign (MDC). NHS England in attendance.

• 15th December 2014 A CPAG (Clinical Priorities Advisory Group) decision on reimbursement was expected on this date but following the legal challenge and NHS England’s decision to scrap the scorecard system and postpone any decision making no announcement was made.

• 18th December 2014 Life Sciences Minister George Freeman met with Greg Mulholland MP, the two pharmaceutical companies BioMarin and PTC, and the two patient organisations MPS Society and MDC. George Freeman asked the pharmaceutical companies to submit proposals on treatment cost and delivery. To date we understand that neither of these proposals have even been acknowledged let alone considered. NHS England failed to attend

• 20th January 2015 Greg Mulholland led a debate in Westminster Hall about Drugs for Ultra-Rare Diseases.

• BioMarin and the MPS Society met with James Palmer, Richard Jeavons and Edmund Jessop at a stakeholder surgery where it was firmly explained that there would be no interim funding whilst NHS England continued to try to develop a fit-for purpose process. James Palmer stated that the process would include a 90 day stakeholder consultation followed by a CPAG meeting after the General Election and then a 90 day public consultation. This in reality takes us to mid-August 2015 and contrary to what the Prime Minister said in PMQ on 4th February 2015.

 23rd January 2015 BioMarin sent a letter to James Palmer and Richard Jeavons requesting further discussions on their financial offer and confirming compassionate use will be stopped on 12th May if no agreement reached by end of February 2015.

• 27th January 2015 NHS England launched a 90 day consultation on their reimbursement decision making process. No timescales are given for a decision.

• 4th February 2015 In PMQ David Cameron responded to a question by Greg Mulholland MP about interim funding of Vimizim for Morquio saying that, ‘The consultation is under way and will finish at the end of April. Following this, the NHS will make a decision as quickly as possible whether or not to routinely fund Translarna. I have discussed this with the Health Secretary and we will do everything we can do help.’ (Translarna is for Duchenne Muscular Dystrophy and not Morquio)

• 4th February 2015 At the Specialised Health Care Alliance James Palmer suggested that CPAG will sit in May/June and we can expect a decision two weeks after that.

• 5th February 2015 ITV calendar questioned David Cameron about access to Vimizim for six-year old Sam Brown and David Cameron quotes April for a decision and that the drug company will fund it until May so there is room for a decision to be made in time. (No official timescales available)

• 17th March 2015 First NICE Evaluation Meeting Manchester. Input from clinical experts, patient experts, NHS England and BioMarin. Concerns were raised with one clinical expert’s nonexistent experience with patients with Morquio.

• 8th April 2015 MPS Society Chief Executive raises serious concern relating to process that we perceive may be detrimental to the NICE evaluation of Elosulfase Alfa and as a consequence prejudice the outcome of the NICE evaluation.

• MPS Society responds to NHS England’s consultation raising concerns about the lack of transparency and its failure to provide any information about the detailed processes and criteria behind these principles. It is currently impossible as a patient, parent or carer to know or understand or trust NHS England that they will arrive at a decision through a fair and transparent process that reflects the needs of all health service users including those with ultra-rare diseases.

• 25th March 2015 Third meeting secured with the Minister for Life Sciences George Freeman which was attended by pharmaceutical company representatives from BioMarin and PTC, a number of Duchenne Muscular Dystrophy Charities, Tuberous Sclerosis Association and MPS Society. A ten page letter was written to George Freeman highlighting the catalogue of errors from NHS England and how they are failing individuals who are desperate for treatment. NHS England failed to attend

• April Fools Day Protest Over 50 children, mums, dads, aunties, uncles and grandparents from across the UK gathered outside Downing Street with huge banners and placards.

• Following our protest on 1st April 2015 BBC Point West asked Secretary of State for Health, Jeremy Hunt on access to Vimizim and on camera he said that he would follow this up if the Conservatives were elected and he was in the position of Secretary of State for Health.

• 27th April 2015 The 90 day NHS England consultation process formally ends. Concerns were raised as process still lacked detail and methodology.

• 28th April 2015 One year Anniversary of the European Medicines Agency licensing Vimizim comes and goes and the wait for treatment continues. Those not on the clinical trial are still being denied treatment.

• Mum Katy Brown met with Prime Minister David Cameron during his election campaign. David Cameron said he would write to NHS England.

 12th May 2015 The date BioMarin were due to cease supply of free drug. Following months of no engagement from NHS England at the eleventh hour NHS England verbally provided timescales of when a decision will be made. BioMarin agreed to continue funding compassionate treatment to those on the clinical trial until the verbally agreed decision date -25th June 2015. The letter committing to dates and promised by James Palmer was never signed by Simon Stevens and hence not received by BioMarin

• Those who were not on the clinical trial are still being denied treatment.

 4th June 2015 NICE’s indication from first evaluation confirms that they would currently not recommend Vimizim for treating MPS IVA (Morquio) and would like further explanation of the benefits it provides to people with the condition. Final recommendation due at the end of the year.


Patients and their families with Morquio have had 13 months of process failures leading to delays for a decision on when the life changing drug clinically trialed in the UK will be made available.

NHS England who make the interim decision on access to treatment whilst NICE complete their assessment had to go back to the drawing board on their process which still lacks detail and methodology.

NICE’s committee need to acknowledge the significance of clinical expertise in their process and address their current expertise shortfalls.

BioMarin will stop providing free drug to those who were on the clinical trial on the 25th June when NHS England have verbally committed that an interim decision will be made.

A decision on the only life changing treatment available for the ultra-rare condition is always going to be emotional but months of uncertainty caused by process failures is unacceptable.

Our members are hugely grateful to their MPs who have supported them by writing to NHS England and Jeremy Hunt. Prime Minister David Cameron is aware of our campaign yet children and young adults are continuing to be let down by process failures and the recognition that people with ultra-rare diseases deserve a chance at life just as those with common disease do.

We will continue to fight for the only licensed treatment available for those with Morquio and need all the support we can get.

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