Something different has been tried this year to raise awareness of aHUS. It involved the Food & Drugs Administration , the FDA, in the USA.
An advocacy partnership, called the USA aHUS Patient Advisory Partnership , or PAP, was formed by the atypical HUS Foundation, aHUS Network Action , Global Families and aHUS alliance Global Action organisations. .
It was set up to undertake an FDA Patient Listening Session*. Many other disease communities have taken this opportunity in the past but this was a first for the US aHUS patient community.
It was held today ,21 September, when six aHUS patient and carer representatives spoke to an audience of FDA employees online with the aim of :
- Telling them about aHUS and what having and living with it is like
- Highlighting the challenges and opportunities for patients for the remainder of the 2020s
So for the first time this was the aHUS community telling this very influential body, not just in the USA but globally too, about what the patient community thinks is important for them to know.
All important stuff that relates directly to FDA employees what ever their role in the influential work done by their organisation.
- trials of new aHUS treatment technologies ,
- regulatory approval of new treatments
- and post marketing monitoring of real world evidence of a continued balance of “benefits versus risks” of any of the treatments FDA approves
The Partnership was represented by Taylor Coffman , Alyssa Deffenbaugh , Dave Deffenbaugh , James Shadringer, Heather Young and Len Woodward.
Over one and half hours they told the FDA, and answered questions, about their experience of having aHUS and its treatment now and in the future.
Collectively they conveyed the disease spectrum from being on dialysis to on complement inhibitors treatment with or without a kidney transplant. The talks were illustrated throughout with images of the speakers before, during and after their illness.
Len bookended the talks with a simple introduction to aHUS and a summary of issues that matter to aHUS patients.
All of which would also impact on the work of the FDA in the coming years.
This session was not about regulatory approval of any particular complement inhibitor existing or in development. Of course there may be several to come in the next few years which FDA will be asked to approve. And of course there is on going post marketing monitoring of existing complement inhibitors adverse events.
Key messages given can be summed up as:
Today the job of raising aHUS awareness was ably achieved by the members of this USA aHUS PAP.
Though this is not the end of it.
A full written report about what was said and done at the Listening Session will, once it is approved by the FDA, be published on their website sometime later this year.
As we head into aHUS Awareness Day on this Sunday 24th September, the USA aHUS PAP has made its contribution to global aHUS awareness.
More information about FDA Patient Listening Sessions can be seen on FDA’s website.
Article No. 603