Access to life changing treatment
Our fight for Vimizim
Latest update from NICE - 16 November 2020
NICE, NHS England and NHS Improvement (NHSE&I) and BioMarin have reached a commercial agreement which will enable the NICE re-evaluation Vimzim to resume. While the re-evaluation continues, they have also agreed a commercial arrangement that will ensure patients can continue to access elosulfase alfa over the duration of the NICE re-evaluation (until December 2021). . Read the full update here.
23 June 2020
NICE have confirmed that NHSE are in discussions with Biomarin regarding the current MAA arrangements, including any additional extensions to the agreement should this be necessary for the re-evaluation of elosulfase alfa (Vimzim).
Statement from NICE - 30 March 2020
NICE has sought clinical expert advice in line with the wider NHS response to COVID-19 during these exceptional circumstances around the safe management of patients receiving treatment as part of a managed access agreement. The following statement aims to try and ensure consistency in approaches across managed access treatment centres and that all patients receive equivalent services across the country. Read the full statement here.
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How we got here
The fight to make Vimizim available was a massive undertaking for the MPS Society and all the families that fought with us. Download the timeline to find out about the journey we took.
26 March 2020
NICE have suspended all appraisals that are not related to COVID-19. This includes the Vimizim appraisal.
16 March 2020
We have been informed that due to mediation by the MPS Society a meeting took place today (16 March 2020) between NICE and the company. Our understanding is both parties had constructive dialogue and have agreed to meet again urgently. We hope that clarity is forthcoming and we will keep you informed of every development that happens.
12 March 2020
The MPS Society would like to reassure our community, that we are actively engaging with all parties (NICE, Pharmaceuticals, NHSE) and have had a number of sensitive discussions to resolve the current situation. We are unable to comment on these at the moment but will inform you of any developments and plans as we know more.
In the meantime the MPS Society and RDRP (MPS Commercial) have written and submitted their patient organisation report to NICE in the anticipation that the process will continue. Thank you to all of you who completed the survey and gave your personal accounts that has enabled us to put forward a compelling case from our patient community.
5 March 2020
We feel this unfortunate situation has been caused by a misunderstanding between NICE and the pharmaceutical company. The MPS Society are working with both parties to hold conversations and help facilitate a meeting in order to resolve this issue.
28 February 2020
NICE have today (28 February 2020) released the following statement in regards to the re-evaluation of Vimizim under the terms of the Managed Access Agreement (MAA).
We are writing to let you know that the re-evaluation of elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643] is now suspended.
This is because the company has not provided an evidence submission that is adequate for the committee to make a decision, and will not accept the terms of the charging procedure for highly specialised technologies. Whilst suspended, the terms of the Managed Access Agreement (MAA) for elosulfase alfa will remain the same. Access to elosulfase alfa will continue for new and existing patients, providing they meet the conditions for treatment. The re-evaluation of elosulfase alfa for treating mucopolysaccharidosis type IVa (re-evaluation of HST2) [ID1643] was originally scheduled so that the outcome would be known prior to the expiry date of the MAA in December 2020. NICE is following up with the company about its submission and options moving forward. We will write to you with any further updates in due course.
MPS Society response
NICE has released the above statement with regards to the re-evaluation process under the terms of the Managed Access Agreement (MAA) for elosulfase alfa for treating MPS IVA Morquio.
The MPS Society are deeply concerned with this news and we are actively engaging with all parties concerned to gain further clarity and assurance that there will be a fair process for our patient communities. We recognise that our members may be understandably concerned on hearing this news and we aim to give you regular updates on the situation as it develops.
Meetings have been scheduled over the next couple of weeks between the MPS Society and both the company and NICE to seek a resolution to this unacceptable situation and to ensure our patients have ongoing access to effective treatments and therapies both now and in the future.
What Vimizim means to our members
“Kamal’s only wish for Father Christmas is ‘to be a normal boy’, and by having Vimizim it gives him the chance to be as normal as can be.”
“…just being able to get on with life and enjoy my leisure time as everyone else does, without constantly feeling exhausted, has had a very big impact on my quality of life.”
“We can’t even bear to think about the fact that it may not be funded. It would feel like we had given Gracie the best chance, to have it snatched away from her."
"I have noticed my body really craves the enzyme replacement treatment, I’m very scared about it discontinuing and going back to that dark place.”