The aHUS Alliance, an umbrella group of 15 nations with aHUS patient groups, launched a worldwide poll of aHUS patients and caregivers of pediatric patients to mark Rare Disease Day 2016 (29 February). Poll questions included 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. A similar effort was conducted in partnership with RareConnect in 2014, with 214 respondents from 17 nations.
With 233 respondents from 23 nations, the 2016 aHUS global poll remained open until 15 April to allow patients to access poll questions in 6 different languages: English, Spanish, French, Italian, Russian, and Japanese. The 2016 global poll questions were suggested from various sources, to include topics from the London 2015 aHUS Alliance multi-national meeting of patient organizations, online discussions across aHUS social media outlets, and interactions with aHUS researchers – all in conjunction with input from various interfaces within the aHUS arena. Additionally, some poll items were 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.
All nations within the aHUS Alliance were invited to participate on the 2016 survey subcommittee and were encouraged to be involved with multiple aspects of the 2016 aHUS global poll. The aHUS Alliance includes among their goals: supporting aHUS patient groups around the world, assisting national and global initiatives regarding information about medical and patient issues, providing and promoting the patient viewpoint, and improving interactions among all stakeholders involved with aHUS treatment and research.
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?
Providing this Patient Voice for aHUS patients from around the world, we invite you to delve deeply into the 2016 aHUS poll results. Whether you are an advocate considering the launch of an aHUS patient organization in your nation, or are a researcher or clinical trial investigator seeking to connect, we welcome your emails at: info@aHUSallianceAction.org.
POLL RESULTS & Additional Information:
DATA – Click to View:
2016 aHUS Poll Results (assets) – Webinar hosted by RareConnect, with commentary by Dr. Christoph Licht
aHUS Alliance “Patient Voice, 2016 Poll” – Our Website Blog
Mid-point Poll Results – RareConnect, blog
Earlier aHUS Poll Results (2014) – RareConnect, data & archived webinar, with Dr. T Goodship