aHUS Alliance aHUS Global Poll Featuring Analysis & Commentary by Dr. Christoph Licht 21 June 2016 WEBINAR Hosted by RareConnect In Collaboration with the aHUS Alliance aHUS Alliance Presenter: Linda Burke (USA), volunteer with The Atypical HUS Foundation

aHUS Alliance

The aHUS Alliance, an affiliation of aHUS patient groups around the world, includes among their goals:  supporting aHUS patient groups worldwide,  assisting national & global initiatives regarding information about medical and patient issues,  providing and promoting the Patient Voice, and  improving interactions among all stakeholders involved with aHUS treatment and research. “GOOD WILL COME TOGETHER”

aHUS Alliance aHUS Alliance - created in Feb 2013 in Barcelona. Advocates from aHUS patient organizations represented Belgium, France, Italy, Russia, Spain and the UK. aHUS Alliance – Engagement Outreach: Rare Disease Day Creation of aHUS Awareness Day, the annual 24 Sept Campaign (initiated 2015) Professional Conferences & aHUS Alliance Meetings Development of aHUS ASSETS & RESOURCES New Website launched May 2016– CONNECT INFORM COLLABORATE aHUS knows no borders. Common issues and concerns affect patients of all nations, ages, and cultures.

aHUS – an ‘Ultra Rare’ Disease

Adult aHUS Patient & Pedi Caregiver responses provided better understanding: INSIGHTS into aHUS diagnosis, treatment, and medical care Patient Voice – Identify NEEDS & ISSUES Data collected from 233 respondents from 23 countries. Poll offered in 6 languages: ENG, ES, FR, RUS, IT and JPN. Launched on Rare Disease Day, 29 February 2016 and open through 15 April aHUS RareConnect Poll - similar effort, 214 respondents from 17 nations RareConnect Webinar & Assets: aHUS Global Poll – OVERVIEW aHUS Alliance

aHUS Patient Voice – an Overview of Poll Participants Respondent Type: aHUS Adult patients - 52% Pediatric Caregivers - 48% Patient Gender: 66% Female 34% Male Most Common Age at Initial Onset (N=229) : 29% - 21 to 35 years old 20% < 3 years old Genetic Testing: 84% have or are awaiting Genetic Test Results National Response Rates (N=233): USA - 43% UK - 18% Canada - 11% Other 20 Nations - participation rate of less than 10% of survey responses The aHUS aHUS Alliance aHUS Info Sources: When seeking Information, MOST Check aHUS Patient Organizations– 37% Rely on their Doctor – 17% Utilize Search Engines – 26%

aHUS Alliance

aHUS Alliance MISSION STATEMENT The aHUS Alliance, through the collaboration of its affiliates, will promote global awareness of aHUS, will work with international aHUS researchers and, by supporting newly emerging national aHUS patient groups, will bring relief and support to those affected by aHUS to save, and improve the quality of, more lives. “GOOD WILL COME TOGETHER”

aHUS Alliance aHUS Alliance PROJECT INFORMATION SUPPORT RESOURCES

aHUS Alliance NEWS BLOG INFO CENTER PROJECTS CONNECT INFORM COLLABORATE INFORMATION Fact Sheets, Graphics, Clinical Videos Under Consideration: Guest Columnists Multiple Languages NEWS Research, Journal Articles Drug R & D, Clinical Trial Data Conferences, Advancements PROJECTS aHUS Awareness Day Rare Disease Day Country-Specific Projects BLOG Delving Deeper: Connections & Insights

aHUS Alliance DIALYSIS 2/3 of aHUS patients needed dialysis during or after initial onset, with only 32% reporting that no dialysis was needed. (N=233) 46% of poll respondents stated the most SIGNIFICANT DIALYSIS ISSUES was it interferes with normal routines. Other dialysis issues (Question 18) Impact on Other ORGANS 29% Negative affect on QUALITY at Work/School 28% Issues with ANXIETY or DEPRESSION 27% aHUS RESEARCH PARTICIPATION in aHUS Research: 50% of Respondents have already done so, and 36% more would like to: (Question 29) YES – 30% YES, and would AGAIN- 20% NO, But WOULD LIKE TO Know How – 36% Highest months for initial aHUS onset? Oct through Dec.

aHUS Alliance FURTHER EXPLORATION - COST Impacts aHUS Treatment  aHUS Treatment Options vary Greatly by Country  Cost Impacts aHUS Patient Care 7 out of 10 state their specialist or medical team mention COST of aHUS treatment in discussing patient care options. 16% state cost concerns affect their treatment options or medical care. (Question #41) Access to Treatment: 24% of respondents state aHUS medical care or treatment is limited by their National or Heath Ministry policies. (Question #43) 29% note that cost of medical care and treatment concern them and their family. Advancements in aHUS treatment or drug therapies: Factors or considerations (Question #45, N=224) Cost of new drugs would likely affect our usage - 33% Recommendation of our medical team - 28% Type of drug delivery/Ease & Convenience of New Treatment - 24%

aHUS Alliance Connect with the aHUS Alliance Emerging aHUS patient organizations – JOIN US Independent Advocates Affiliates: Renal, Genetic, Language-Specific, or national Rare Disease Groups PARTNER with the aHUS Alliance – Individuals & Groups The aHUS Alliance encourages stakeholders in every nation to connect and to collaborate aHUS Alliance. Individuals, organizations, industry, researchers and medical personnel are invited to participate with the 2016 September 24 campaign: Join the RareConnect aHUS Community – Connect Globally Rise above aHUS: 2016 Red Balloon aHUS aHUS Awareness Day Information & Resources:

aHUS Alliance A Special THANK YOU to aHUS Adult Patients and Pediatric Caregivers who participated in the Patient Voice with the 2016 aHUS Global Poll We appreciate the many aHUS researchers, physicians, organizations, and Individuals for their support of this project. Thanks to the RareConnect Team for hosting this 21 June 2016 aHUS Global Webinar and to Dr. Christoph Licht of Toronto’s Sick Children’s Hospital

aHUS Alliance CONNECT INFORM COLLABORATE US: aHUS Alliance : Be aHUS Aware – LIKE US on Facebook aHUS Alliance on Twitter: JOIN the Conversation – Network among aHUS Global Interests Follow the aHUS Awareness Day aHUS Alliance