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.
The above statement can be found on the NICE website nice.org.uk
MPS Society Response
NICE has released the above statement with regards to re-evaluation process under the terms of the Managed Access Agreement(MAA) for elosulfase alfa for treating MPSIVa Morquio. The MPS Society is deeply concerned with this news and is actively engaging with all parties concerned to gain further clarity and the assurity of a fair process for our patient communities. We understand that this will be a time where our MPSIV community will understandably be worried and concerned and we will be giving 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.
MPS Society UK Reply: mpssociety.org.uk
My Way Access Response
As someone with MPS Morquio and has seen my life transformed since starting Vimizim in 2016. this is a very worrying development. It took years of campaigning for this drug to be made available in the UK and further campaigning before it was available in Ireland.
I will be putting my faith in the MPS Society UK to fight on our behalf to ensure that the differences between NICE and BioMarin Pharmaceutical are resolved before December 2020. This shouldn’t be about differences between organisations, as the real differences is faced by us who want and need continuous access to Vimizim.
Dermot Devlin’s Rare Disease Day 2017 talk