***England Update Vimizim***


Elosulfase alfa, Vimizim is recommended within its marketing authorisation, as an option for treating mucopolysaccharidosis type IVa (MPS IVa) only in the context of a managed access agreement.

This is the first time NICE have looked to a more flexible approach to agree a decision on treatment reimbursement and the MPS Society welcomes this opportunity. Without this Managed Access Agreement those with MPS IVA would be facing a ‘no’ decision.

We have been working incredibly closely with the expert clinicians, NHS England and BioMarin to create this managed access agreement which is nearly complete. If and only if NICE deem this agreement to be robust enough will they make their final decision a positive.

All the stakeholders involved in creating this document want the decision to be positive and are committed to giving every English patient (Scotland, Wales and Northern Ireland each make their own decision), regardless of age or whether an ex-clinical trial patient or not, fair access to treatment (if NICE agree to the Managed Access Agreement).

This is a unique opportunity and if we get this right may lead the way for how future drugs are reimbursed.

Our deadline for completing this will be the same as the overall consultation deadline. NICE will then be consulting on this document. Due to this additional requirement from NICE we have now had confirmation that their final decision on funding Vimizim will be January 2016. This is hugely disappointing especially for those not on treatment.

We would welcome as many people as possible responding to the NICE consultation welcoming the unique Managed Access Agreement, as mentioned above without which we would definitely be facing a ‘no’. We would though suggest that the timescales are challenged, especially with the knowledge that so many are being denied treatment.

The link again for the NICE consultation can be found here. And the deadline is the 23rd September. If you have problems with the website please let us know and we will contact NICE.