1,000 signatures reached
To: JEREMY HUNT, SECRETARY OF STATE FOR HEALTH
NHS ENGLAND'S FAILED APPROVAL SYSTEM DELAYS ACCESS TO TREATMENT
We want interim funding for treatments caught up in NHS England's failed approval system.
The failure to reinstate the Highly Specialised Services and ultra-orphan drug appraisal process for children and adults with ultra-rare diseases that NHS dismantled on 1 April 2013 is catastrophic for the 88 children and young people in England with Morquio Disease, MPSIVA. Following dialogue with Jeremy Hunt and many patient's constituency MPs we advised the NHS England reorganisation would fail people in England with with ultra-rare diseases and sadly how right we are.
Why is this important?
The UK was at the forefront of the research on Vimizim with 7 of the 8 designated Lysosomal Storage Disease (LSD) centres involved in the study. This brings much needed investment into the NHS as the hospitals are paid to undertake this research.
Almost 30 percent of the global patient population enrolled in the clinical trial were from England. The commitment from families to meet the demands of the clinical trials is huge, often travelling hundreds of miles on a weekly basis for treatment. To then face uncertainty and not have a clear process for drug approval is very demoralising. England has lead on the trials but is behind 21 other countries who are paying for Morquio sufferers to receive Enzyme Replacement Therapy, denied to sufferers in England.
Whilst new therapies are expensive for children and adults with ultra-orphan diseases the number affected in England are usually in the tens and never total more than 500 affected individuals. These ultra rare diseases affect many organs of the body and usually result in death in childhood or early adulthood.