Greg Mulholland MP secures debate on Funding for Drugs for Ultra-Rare Diseases

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Greg Mulholland MP secures debate on Funding for Drugs for Ultra-Rare Diseases

We welcome the debate lead by Greg Mulholland MP for six year old Sam Brown today at Westminster.

Following the legal challenge of a 10-year old boy with Morquio exposing the flawed processes of NHS England, there is currently no mechanism to assess licensed treatments for rare and ultra-rare diseases.

Morquio is a progressive condition affecting just 105 people in the UK. Patients with Morquio syndrome appear healthy at birth but, in the second or third year of life, severe symptoms start to become apparent – symptoms which seriously limit their life expectancy, including: heart disease, skeletal abnormalities, vision and hearing loss and difficulty breathing.

Vimizim, an Enzyme Replacement Therapy, and only treatment for the condition MPSIVA known as Morquio was licensed on 28th April 2014 by the European Medicines Agency (EMA) and is now available in over 20 other countries in Europe including France, Germany and the Czech Republic.

Despite the UK being the major player involved in the clinical development of Vimizim, it is still one of the last EU countries to get the drug funded following EMA approval. Patients on the clinical trial are still receiving the treatment compassionately but those who were not on the clinical trial remain untreated and their symptoms continue to get worse.

Clinical trial research into rare disease brings much needed investment to the NHS as hospitals are paid to carry out the research. Without an approved funding decision-making process in place, children and young adults who have spent years meeting the high demands of the clinical trial, often travelling the length and breadth of the country are left anxiously waiting to hear whether their new life-prolonging drugs will receive funding on the NHS.