Current Campaigns: Our Fight for Vimizim
Over the last five years 34 MPSIVA patients have been part of a clinical trial for a treatment in the UK. This involved weekly travel to hospital for four hours of infusion. Hospitals were often hundreds of miles of home either resulting in a 12 hour day or an overnight stay in a nearby hotel. While the UK led on the clinical trial yet we are one of the last countries in Europe to have funding approved. The treatment Vimizim was licensed in April 2014 and over 20 countries have since agreed to fund the treatment.
Those who were part of the clinical trial are still compassionately receiving the treatment but it is uncertain how much longer this will continue. Unfortunately those were not involved in the clinical trial are being denied treatment until funding is approved.
A funding decision was expected mid-December but NHS England failed to develop a suitable decision making process for ultra-rare disease. Through supporting one of our ten year old members with Morquio to legally challenge the scorecard system, NHS England are now redeveloping their processes.
For all the news concerning our fight for Vimizim, please take a look at our 'Vimizim: news and information' page.
This decision is bittersweet as the ongoing delay means that a number of members who would benefit from treatment are denied access until a funding decision is made whilst others are continuing to travel hundreds of miles weekly to hospital for treatment which could be received at home.