aHUS Alliance – Sharing Through Blogs
Sharing Patient Group Experience Through Blogs – aHUS Alliance
Working with patient groups for decades, it’s always striking how tough and resilient the individuals at their heart have to be. The challenges and pressures seem to have intensified in that time, and getting noticed in the noise of the internet and social media, ever harder.
Following on, in this sea of chatter, one blog series really stands out. The title captures its purpose:
“aHUS Advocacy in the UK – The Reluctant Advocate & the story of the battle of how eculizumab treatment became available to those people diagnosed with atypical HUS in the UK.”
Tracking the lifecycle of a rare disease group
The blog series was written by Len Woodward. It looks back over nearly a decade of experience. He was a founding Trustee of the patient group aHUSUK and a member of the global patient organisation the aHUS Alliance.
Moving forward, the Alliance has archived his series of `Reluctant Advocate’ blogs on its website. This gives a personal, direct, and open insight into the lifecycle of a rare disease group.
Forming the group
Additionally, the early blogs in the series reflect on the formation of the UK rare disease group. As so often with patient group founders, life was the trigger when his daughter was finally diagnosed with atypical Haemolytic Uraemic Syndrome, or aHUS. This is a very rare, chronic and life-threatening genetic condition affecting the immune system.
A leading UK specialist in the field recognised the need for a patient group, and called together families at a meeting in London.
Reflecting back, Len Woodward summarises the dilemma faced by many groups at formation:
“Now we were in the room with the families of another six aHUS patients who had answered Prof. Goodship’s call and had been challenged to become a formal charity with “objectives, rules and a constitution”. To say that there was a reluctance by all to do so would be an understatement…
…As the meeting progressed it had become clear to us that such a group was essential to meet the National Health Service’s demands to be able to provide the patient voice when its committee met to evaluate the case for eculizumab to be used for aHUS in England.” – Len Woodward, aHUS Alliance
Fighting for care
Furthermore, the series of blogs follow the small group’s long, challenging and confusing journey of building the case for treatment. It climaxes with a meeting where the rare disease committee of the UK National Health Service, allocates 5 minutes for the patient case.
Len reflects back on the pressure of getting this `5 minutes’ right:
“aHUSUK’s job was to show how debilitating and life threatening aHUS was and that Eculizumab offered benefits “beyond price”. We had our Patient Voice Report but we also had five minutes to get the point across too. It was important that every second of the 5 minutes was used and no more. Every word had to count.
Three trustees and their families met and spent 8 hours designing and developing the talk and its supporting visuals. In run through after run through words were changed and times were cut until the optimum was reached. A five-minute talk emerged which said all that needed to be said and said it plainly enough.” – Len Woodward, aHUS Alliance
It’s a rite-of-passage for many patient groups. Just yesterday, I was talking with a US group about the emotional impact on patients making face to face submissions to the US FDA (Food and Drug Administration).
It’s tough to do, and tough to get right. Sometimes I’ve seen the driven `I’m not a victim’ `Can do’ mentality of patient submissions undermine the impact of a condition or symptoms.
Dealing with setbacks
Following on, the patient group’s blog series documents many setbacks on the journey. Critically, the rare disease committee the group presented to had recommended the target treatment. However, the UK Department of Health changed the decision-making process and structures. This shifted the responsibility for the decision to the main agency NICE (now the UK National Institute for Health and Care Excellence).
Len recalls in his blog breaking the news to his wife:
“It’s a ‘no decision’. I said having read through an incoming e mail that morning.
“They said ‘no’.” my wife replied incomprehensively.
“No ,they said ‘yes’.” I replied confusingly.
“They said ‘yes’?” she returned.
“Yes” I confirmed, “but the Minister cannot make a decision now as he wants it all done again, but this time by NICE, so it is a ‘no decision’ until then.
– Len Woodward, aHUS Alliance
Advocating to the full
At this point, as Len puts it, the group had to `change gear’ to fight for the original committee’s recommendation. It had a huge impact on the small group:
“What started as a four hours a month task at the start had rapidly passed four hours a week and was now four hours a day for most aHUSUK trustees and would now move to 2 to 3 times that for some.” – Len Woodward, aHUS Alliance
In addition, the group focused on the UK political process, using written and online petitions to gain the attention of Members of Parliament and the UK Prime Minister.
Using media and social media, both petitions were hugely successful, in drawing attention to the issue:
“A small number of aHUSUK members had created considerable noise, all of which was being noted at the Department of Health.”
Reflecting and sharing
Although the climax of Len’s story is five years ago in 2013, the key message remains one of hope. No matter how rare the disease, no matter how high the challenges, small patient groups worldwide keep fighting.
NICE did ultimately create a treatment guideline for atypical Haemolytic Uraemia Syndrome. They did this by accepting evidence from the patient group, and clinical evidence from Professor Goodship, who had first brought the families together to start the patient group.
Len’s blog also talks about the formation of the global patient group for the rare disease, now with patient groups in more than 30 countries.