Call to action USA aHUS Patients

In Article 462 notice was given of a study to help USA rare disease patients in general. and aHUS patients in particular, by gathering information about their experience of gaining funding for their treatment.

Although not an aHUS alliance Global Action study it is one we have agreed to support. It is being conducted by a Masters Degree student at the University of Nebraska, Michelle Willes. An appeal from Michelle for your help with this research follows:


My name is Michelle Willes and I am a genetic counselling student at the University of Nebraska Medical Center.

I am conducting a research study to better understand the experience of navigating payment and insurance coverage for medical costs associated with a rare disease diagnosis

I invite you to complete this anonymous survey which will ask about your rare disease diagnosis , your experience with health insurance coverage , and your awareness of financial resources for rare disease diagnosis.

The survey will take approximately 15 to 20 minutes to complete.

Thank you for your help!


The on line survey can be accessed at the following link HERE The Institutional Review Board approval reference number for this study is IRB # 0614-21-EX

The online questionnaire is available now until its new, extended deadline – closing date of this survey: 5 November 2021.

Michelle sent Global Action more information about the reason for her research. It stems from from a previous study “Burden of Rare Disease Study” which was published earlier this year which inspired her . It can be read at this link HERE

That study was of 379 rare diseases and with just over 1400 participants. aHUS was not included. It reported that the estimated direct and indirect costs of rare diseases in the USA is just short of $1 trillion. It concludes that the additional financial burden of those Irving with rare diseases is about $27,000 per year per patient. Of that sum, $ 3000 is due to prescription medicine cost.

Michelle is hoping that her research will work in support of this study to shine a greater light on the experience of navigating insurance coverage and payment rare disease community.

Michelle’s study findings will be published in 2022.

Article No. 463.

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