A Patients and Families Support Group

A UK website about atypical Haemolytic Uraemic Syndrome (aHUS)

Answers for aHUS momentum returns

Answers for aHUS momentum returns

After a period of relative inactivity Answers for aHUS has regained momentum  this week.

Firstly it was invited to a meeting with NICE in London to give views on how NICE might be better at publishing its guidelines in a more readable and understandable format. This was how it announced the Eculizumab for aHUS decision click here.  A task group including Answers for aHUS will work on designs with the aim of completing its work in early 2018.

At the same meeting Andrew Dillon ,Chief Executive of NICE,   talked about the new evaluation processes that NICE will be adopting with a new range of cost effective thresholds ( expressed as “cost per QALY”)  . At the lower end below £10,000 per QALY , new therapies will not have go through as rigorous a process as those which exceed £100,000 per QALY.  Eculizumab exceeded the latter threshold as would most therapies for rare diseases requiring  highly specialised therapies ( HST) . Such therapies will have to go through a further decision making process i.e. via the NHS’ Clinical Priorities Advisory Group ( CPAG) . NHS England have also set an affordability limit for first year of implementation at £20m above which a phased implementation can be called for. Previously NHS England were given three months to implement new guidelines.

It will be a few more years before aHUS patients will be affected by a  further NICE evaluation and  decisions; no doubt there will be more process changes to come as there have been over the past five years.

Secondly, an update was received from Kidney Research UK about the research funds for aHUS. Tracy Murray has taken over as the Answers for aHUS community manager. She only began her job after Christmas and has been settling in since. Tracy informed Answers that our fundraising total  had reached £40,000 by 31 March this year. It may be recalled that our expectation was that it had reached £50,000, but a pledge of £10, 000 from a family which had raised funds to pay for eculizumab for  a family member has not materialised.

Nevertheless there has been some continuing fundraising by a family in Devon, the NHS aHUS Expert Centre Nurses and individuals through our Just Giving and Easy Fundraising accounts. However the telephone texting fundraising route has been closed through lack of use and should no longer be used.

KRUK continue to seek research proposals about aHUS  and a current round of calls is taking place. There should be news in the near future, but in the meantime  efforts for further funds to be raised  needs to be revived. Tracy will be letting us know about new ways of fundraising . If you have not already done so why not join in the Newcastle Bridges walk . This year it is on 11 June ,and it is 7 miles across Newcastle upon Tyne’s  iconic bridges.

Thirdly Answers has heard that funds for the Withdrawal of Eculizumab Study have been fully approved by the NIHR and it is expected that the research project will begin in Q3 this year.

The UK Study officially known as :

15/130/94 Multicentre, open label, prospective, single arm study of the safety and impact of Eculizumab withdrawal in patients with atypical haemolytic uraemic syndrome 

is essentially about stopping eculizumab treatment safely and is one of several such studies taking place around the word as reported in a recent aHUS alliance news item ( click here)

lastly Answers has heard from the Newcastle aHUS Expert Centre that a number of aHUS activities are likely to begin shortly ,watch out for more news about these.




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