The aHUS Alliance, an umbrella group of 15 nations with aHUS patient groups, launched a worldwide survey of aHUS patients and caregivers to mark Rare Disease Day 2016 (29 February). A similar effort was conducted in partnership with RareConnect in 2014, with 214 respondents from 17 nations and responding to outreach in 6 different languages. The 2014 poll included questions on common themes familiar to patients with rare diseases and echoing broadly on Rare Disease Day: barriers to diagnosis, access to treatment, availability of genetic testing, need for increased research efforts, participation in registries and clinical trials, and better/more information about their rare disease.
Rare Disease Day, created in 2008 by EURORDIS, helps the world to better understand the challenges and support needs faced by rare disease patients and their families. This special day serves to underscore the need to share knowledge about all rare disease issues and information, and also champions the call for development of new research for diagnostics and treatment. Rare Disease Day historically has included momentum for advancement of national plans and policies for rare diseases in a number of countries as well. To honor the intent of the day with an action of collaborative advocacy, advocates within the aHUS Alliance chose Rare Disease Day to launch its 2016 aHUS global poll.
In 2014, the global aHUS survey brought some interesting facts to light and we expect similar value in the 2016 survey results. After only two days, the 2016 aHUS global poll garnered responses from 34 aHUS patients and caregivers in 8 nations, expanding in the first week for a total of 78 survey respondents from 10 countries. This year’s poll will be open during all of March 2016, with survey results to expected to be available in both global data and country-specific formats.
From the aHUS Alliance commentary on 2014 aHUS poll results:
“Of the 214 aHUS patients included in the Survey almost two thirds (64%) were reported as being female with just 36% being male (Q2). There appeared to be no gender difference in respondents up to 18 years (41 to 41) but it is in the adult group that females vastly outnumber males (95 to 37). Only in the preteens group do boys slightly outnumber girls (36 to 32). Children up to 10 years of age were the largest reported group (28%) and the next largest group (21%) were aged 30 to 40 years (Q3). The average age of all respondents was 27 years.
The majority of patients (54%) reported that they had their first severe episode of aHUS before their 21st birthday and for most of those it was even before they were 10 years old. Very few became ill (4%) after their 50th birthday so most of the remaining patients became ill in early adult hood to middle age (Q5). The average time respondents have been living with the illness is 8 years, making 19, the average age of onset.
To read the rest of the aHUS Alliance 2014 poll commentary, click HERE.
To access 2014 RareConnect aHUS global poll charts and graphs, click HERE.
To view Dr. Tim Goodship’s webinar on interpreting 2014 RareConnect aHUS global. click HERE to view Dr. Goodship’s webinar. (with links to slides and poll assets)
So why launch an aHUS global poll in 2016? Certain questions and topics have repeatedly appeared within patient communities, aHUS social media outlets, and within meetings such as the aHUS global conference of nations in London 28 June 2015. Atypical HUS patient groups within the aHUS Alliance network to connect information, opportunities, and stakeholders within the aHUS arena. In the orphan drug arena, the landscape is rapidly changing. Though people with atypical HUS face the threat of a chronic disease with life-threatening health crises, most nations have limited access to full treatment options. Clinical trials, diagnosis advancements, research updates, and development of complement inhibitors all have tremendous impact on patient lives. But is there a way to provide researchers, clinicians, and industry insight into how best to interface with patients and patient organizations to the benefit of all stakeholders? Is there a way to involve aHUS patients and caregivers worldwide as valued partners in patient care, to include even those isolated patients currently not represent by an aHUS patient organization? Can patient experiences regarding aHUS diagnosis and treatment issues provide valuable insights for policy makers and the healthcare community?
Members within the aHUS Alliance think meaningful information from the 2016 aHUS global poll can provide researchers, clinical trial investigators and industry a better perspective of how to reach physicians and their patients. The survey also was designed to help patient groups better understand the needs of their members and to explore the differences in care, diagnosis and treatment access around the world. We believe the data from the 2016 aHUS global poll additionally will provide aHUS patient organizations data to present to their government’s policy makers, healthcare industry, and Health Ministers concerning options in their nations for continuation or expansion of patient care and treatment options.
We encourage all individuals – all nations – all aHUS patient organization to encourage aHUS patients and caregivers around the world to participate in the 2016 aHUS global survey.
Click HERE to view a blog regarding 2016 possibilities for potential new aHUS treatments.
Click HERE for the 2016 aHUS global survey.
Survey Results will be posted on www.ahusallianceaction.org
E: Info@aHUSallianceaction.org Twitter: @aHUSallianceact
Blog about the survey, from patient organization Atypical HUS India
March 16, 2016, UPDATE: Click the link to read the