The aHUS Alliance continues its series ‘The Reluctant Advocate’, chronicling the UK’s journey to gain access to eculizumab for treating those with atypical HUS so that other people and nations can gain insight into the challenges of bring new aHUS drugs to market.
aHUSUK support for Rare Diseases had grown each year. By 2014 it had reached a pinnacle for such a small organisation.
We were present for the unveiling of Alexion’s “raise your hands ” artwork in London. aHUS patients and carers had participated in it.
We attended all the Home Nations’ Rare Disease Day events in their parliaments. I attended the one in Northern Ireland although sadly it was not to be held in Stormont, the home of the Northern Ireland Assembly.
Held in Queen’s University Belfast ,and with the theme “Joining together for Better Care” , on Rare Disease Day 28th February 2014, the conference was attended by several hundred rare disease patients and patient organisations from both the North and South of Ireland.
Early in the proceedings the Health Ministers from both administrations gave talks about their respective Rare Disease Implementation Plans.
Edwin Poots, MLA, ( see photo above) expressed determination to make things happen but was pragmatic and that it will be a matter of “incrementing progression” over time. Eculizumab would become available when it became policy in NHS England, and NHS Northern Ireland followed suit.
Alex White, TD, outlined some of the Rare Disease infrastructure being put in place, like the National Office for Rare Disease in Dublin. By the time it was opened in June 2015 he had been replaced as Health Minister by Leo Varardkar, and eculizumab had been approved for aHUS for Irish aHUS Patients the preceding February.
aHUSUK was by Rare Disease Day 2014 aware of a small number of aHUS patients in Northern Ireland as well as a number of aHUS families in England with familial links with areas in the South West of Ireland.
Before I left my hotel for the conference I was interviewed live over the telephone by Radio Wales about Rare Disease Day. This led to me talking about my family in a part of Swansea, a city in South Wales. The penetrance of aHUS in my family meant that they dominated the Welsh aHUS patient cohort. aHUS in my family had come from England. A recording of the interview can be heard here.
Both Irish Health Ministers saw cross border collaboration as very much needed to make a difference for those with rare diseases.
I could identify with that on Rare Disease Day 2014.
Earlier stories by the Reluctant Advocate can be read here.
Tell your story to raise aHUS and Rare Disease Awareness by supporting
the aHUS alliance Rare Disease Day Video 2019.
Click HERE for more information