NICE provides positive feedback

Following the recent news that Elosulfase alfa (Vimizim) has been recommended by NICE (National Institute for Health and Care Excellence) for treatment MPS IVA Morquio in England and Wales we have received some excellent feedback from the committee about our input, which we are pleased to share with you below.

Our contribution to the re-evaluation formed a written submission that was presented to the committee. This document was our final opportunity to represent the patients and their perspective, as well as the views of our in-house experts. With the decision on this treatment in the balance, we knew the importance of this document. The feedback we have since received from the committee and NICE has told how our input:

"Provided great context and clarity to the patient experience and their views of their outcomes over the MAA period"

"Helped the committee to understand what the numbers meant to real people"

Together with Rare Disease Research Partners (RDRP) we have worked for many years to support the patients and collect patient outcome data for this treatment under the Managed Access Agreement. We are immensely proud to have been entrusted with representing the patient voice and to hear that our submission met all the requirements of the committee:

"The patient submissions were very thorough and of the highest standard the committee has seen so far and the committee were not sure they could have asked for more"

We continue to serve the rare disease community and thank everyone that has been involved in this effort.