Access to life changing treatment

Our fight for Vimizim

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).

Latest 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.

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.

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About MPS IV Morquio

Helpline

 

Our helpline is open 9-5pm

Monday-Friday: 0345 389 9901

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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. 

Fighting for Vimizim timeline.jpg

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).

NICE statement​

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.

​If you are concerned or want to talk to our support and advocacy team about how you or a family member may be affected please call on 0345 389 9901 or email Sophie Thomas, our Head of Patient Services.

We are the only registered charity providing professional support to individuals and families affected by MPS, Fabry or a related disease in the UK.

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