To: Jeremy Hunt, Secretary of State for Health

NHS England's scorecard Denies Access to Treatment for Ultra Rare Diseases

19/12/2014: Following the threat of a legal challenge by a 10 year old boy with Morquio, NHS England have announced that they are abandoning the scorecard and are going back to the drawing-board on their approval process. We are really pleased that the petition and threat of legal action has forced NHS England to develop a fair decision making process for ultra-rare diseases - thank you to everyone who signed.

NHS England's scorecard Denies Access to Treatment for Ultra Rare Diseases

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. Through dialogue with Jeremy Hunt and 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?

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. Today even though the European Medicines Agency gave Marketing Approval for an Enzyme Replacement Therapy for children and young adults with MPSIVA, Morquio disease in April 2014 and the health departments in France, Germany, Austria, Italy and even Turkey are paying for Morquio sufferers to receive Enzyme Replacement Therapy, 77 children and adults in England are denied Enzyme Replacement Therapy and to be treated at home.

Category


Reasons for signing

  • I have fought for Ultra Rare Disease Patients for the past 9years I have also set up the Ultra Rare Disease Disorders & Disabilities Foundation I know just what its like to have a child with an Ultra Rare Disease my grandson has EBVHLH and XLP he fight every single day and his Mum is the greatest I Knew Ultra Rare Disease would become a issue as it was not included in the rare disease plan and it should have been but we hope to put as much pressure on Westminister they will have to move on this
  • Sam & his family need justice & the murderers need prosecution & jail!
  • Everyone's health is important and if we can help with medication, we should.

Updates

2014-12-19 10:34:37 +0000
Success badge small

Petition is successful with 8,235 signatures

2014-12-17 21:26:36 +0000

Thank you to everybody who signed our petition. Following the threat of a legal challenge by a 10 year old boy with Morquio NHS England have announced that they are abandoning the scorecard and are going back to the drawing-board on their approval process. Whilst we are pleased that the petition and threat of legal action has forced NHS England to develop a fair decision making process for ultra-rare diseases we are disappointed that for those whose access to vital treatment is dependent on this process the wait continues.

We are grateful for your support and look forward to contacting you with positive news soon.

2014-11-16 15:38:25 +0000

5,000 signatures reached

2014-11-11 18:57:24 +0000

1,000 signatures reached

2014-11-11 17:01:47 +0000

500 signatures reached

2014-11-11 12:51:30 +0000

100 signatures reached

2014-11-11 12:07:48 +0000

50 signatures reached

2014-11-11 11:36:07 +0000

25 signatures reached

2014-11-11 11:16:34 +0000

10 signatures reached