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.
Visit our page on access to life changing treatment to find out more about our ongoing campaign.