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A UK website about atypical Haemolytic Uraemic Syndrome (aHUS)



aHUSUK had been a participating stakeholder in the review of the process for approving new medicines in Scotland called for by the Scottish Government ( a few days  before eculizumab was evaluated by the SMC!).

Previous aHUSUK reports on progress of the review can seen by clicking here and here.

The Review was led by Dr Brian Montgomery ( featured image), and his report has been made public on the Scottish Government’s website , click here to read the whole report.

There are 28 recommendations for improving and re-engineering  the process and Recommendations  7 & 11 are of particular importance to aHUS patients.

Firstly a definition of what   a ” true” ultra orphan drug is , has been called for.

The report also calls for a new assessment and approval process for very rare disease

Although approvals for  IPTRs for rare and end of life disease  therapies  had increased over the past three years, SMC’s record for very rare diseases, like aHUS, showed only 1 in 7 drugs being approved.

Like aHUSUK, Answers for aHUS supports the development of a process to bring equity  to the  “true” ultra orphan medicines for very rare diseases like aHUS . NICE took over from AGNSS ,Advisory Group for National Specialised Services,  which did precisely that,but NICE were very uncomfortable about doing so.

But  now a separate process for very rare diseases and drugs like eculizumab is also what NICE and NHS England are proposing to do too.

Something like AGNSS within the NHS perhaps , albeit that the final decision is not being down to a politician to give.

aHUS patients in the UK now benefit from world class treatment, which is something which needs to be sustained and built on in the future.

Equitable processes for such decisions is to be welcomed by Answers for aHUS  for when the time comes again to provide the aHUS Patient Voice.






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