NHS Delays Decision on Elosulfase Alfa

NHS Delays Decision on Vimizim

We are extremely concerned and disappointed that it took a legal challenge for NHS England to realise that a process which had taken many months to bring to fruition was so flawed and has resulted in this decision to go back to the drawing board. You will recall that back in the autumn of 2012 that the MPS Society and its members wrote to members of parliament to express concern that the new system being put into place from 1st April 2013 would fail children and young adults wanting access to Elosulfase Alfa. Everything the MPS Society said to Earl Howe and Jeremy Hunt, Secretary of State for Health, has sadly come to fruition. This further delay in the specialist commission process with the cancellation of the CPAG meeting is symptomatic of the fragile commissioning process put in place under the Health and Social Care Act.

Our CEO Christine Lavery said
“Whilst NHS England is now acknowledging that they have not got the robust processes in place to decide funding for treatments for rare and ultra-rare diseases the delay in the decision making process discriminates against these young children and adults with mucopolysaccaridosis IV Type A, some who were not part of the clinical trial, and are unable to receive the life changing treatment until the process is developed and the reimbursed funding is approved. There are also children not receiving compassionate drug at home and having to travel long distances to Manchester every week to receive elosulfase alfa, missing a vital time off school. Without treatment the mean life expectancy is 25 years in a progressive deteriorating condition. “

The MPS Society is seeking urgent action from the Secretary of State, Jeremy Hunt, to approve an interim funding and reimbursement through reintroducing the highly specialised service, which was disbanded on 1st April 2013. The MPS Society is working with all its members affected by Morquio disease and their families to achieve this. Many of you have already provided us with impact statements and we would encourage those who have not yet responded to do so immediately.

We encourage all our MPS individuals and their families to contact their MP, emphasising the urgent need for Morquio patients to be given access to Elosulfase Alfa through an interim process of highly specialised commissioning. You can find contact details for your MP here.

We would also encourage all members to sign the petition below which demands that the NHS fully funds Elosulfase Alfa. Please sign and share with your family, friends, colleagues and MP:


Please continue to follow our website and social media to alert as many people as possible to the plight of our Morquio children and adults as well as their families. We will bring you any further information as soon as it is available.