aHUSUK Welcomes and Accepts the Final Evaluation Determination Issued by NICE
The Final Evaluation Document (FED) issued by the National Institute for Health and Care Excellence (NICE) for Eculizumab to treat the ultra-rare atypical Haemolytic Uraemic Syndrome (aHUS) has confirmed the same recommendations of two previous separate decision-making evaluation processes starting in 2011; AGNSS (Advisory Group for NHS Specialised Services), CPAG (Clinical Priorities Advisory Group).
Each evaluation has come to the same conclusion, Eculizumab does the job of controlling an aHUS patient's compromised complement system and without it their own immune system would proceed to destroy major organs resulting in death or a much depleted quality of life. Eculizumab can prevent brain, heart and kidney damage as a result of aHUS. Moreover, it can enable those with chronic kidney damage caused by the disease to return to a normal life because the treatment also prevents aHUS destroying a transplanted kidney.
The NICE FED suggests it accepts that an effective complement inhibitor like eculizumab is worthwhile as treated patients can expect a normal life span, will be able to live fulfilling lives and make their own contributions to our society.
Commenting on the NICE FED and supporting guidelines, aHUSUK chairperson Emma Woodward said: "Of course we are delighted for every sufferer of aHUS and their families. But we also recognise something very important has just occurred. This is the first time NICE has evaluated a rare disease ultra-orphan drug. We are impressed with the committee's approach and believe that NICE has demonstrated a vision, willingness and capability to devise a process for evaluation of other such complex technologies to address the needs of those who seek equity of access to treatments of their severe rare diseases."
"The conditions that NICE has set for its recommendation in the FED for eculizumab are forward looking and far reaching. This evaluation places aHUS patients in England at the forefront of a movement which we hope means see that any aHUS patients anywhere in the world is neither neglected nor facing arbitrary nor financial caps limiting access to life-transforming treatments. In the UK we know that the NHS can deliver for patients and it will be important that clinical services can match treatment availability for all."
aHUSUK is confident that a safe, sustainable and affordable treatment for all aHUS patients will result from this decision; and is grateful that the matter is now being brought to a satisfactory conclusion.
For more information please contact [email protected]
Note to editors
aHUS is a rare disease that is genetic in origin, with abnormalities in the innate immune system called complement. These abnormalities lead to excessive activation of the complement system which can in turn lead to the development of blood clots in the filters of the kidney and in the capillaries of the heart and brain. Factors such as pregnancy or a viral infection may trigger an episode.
aHUSUK is a registered charity in England &
Wales and Scotland, run entirely by volunteers to provide support to aHUS patients and their families. More information about aHUSUK can be found at http://www.ahusuk.org
