Isabelle Lousada What a Patient Driven Multi-Stakeholder Organisation Can Do and Achieve. The Case of the ARC

Current Landscape Complex, debilitating, progressive disease with no cure Late diagnosis Few treatment option Scarce resources No obvious research blue print Stakeholders not aligned; working and thinking in isolation As a consequence……. With the status quo  - advances of the magnitude needed will not be made

Scarce Resources Unlimited wants and needs Limited Scarce Resources The economic problem is to match limited Resources to unlimited wants and needs

ARC’s Role FDA/EMA Enhance Regulatory decision making Nominating opportunities presented by science Academia Amyloidosis Research Consortium Improve patient care HMO’s ARC Expedite medical product development process Industry Patient informed drug development Patients

Value of ARC Your texte here Formal Governance Structure Programs Clear mission statement Your texte here Targeted deliverables Timeframe of deliverables Scientific/Translational Research Milestone driven work streams ARC Defined decision making roles Clinical Trials Defined opportunities for giving input Transparency Health Service Research Accountability and trust Mechanism for broad access to consortium output

ARC’s Collaborative Network Europe Australasia USA Barbara Ann Karmanos Cancer Institute Boston University Amyloidosis Center City of Hope National Cancer Center Cleveland Clinic Columbia University Medical Center Mayo Clinic Medical College of Wisconsin Memorial Sloan Kettering Cancer Center  Penn Amyloidosis Program, Stanford Healthcare Sylvester Cancer Center, University of Miami Tufts Medical Center  Vanderbilt University Medical Center National Referral Center of Amyloidosis, Limoges France French Reference Center For Familial Amyloid Polyneuropathy France Amyloidosis Center Heidelberg Fondazione IRCCS Policlinico San Matteo Italy Groningen Unit for Amyloidosis Research & Development Netherlands Hospital Clínic of Barcelona Spain National Center for Amyloidosis  UK Princess Alexandra Hospital Australia Victorian & Tasmanian Amyloidosis Service Australia Westmead Hospital, NSW. Australia Kumamoto Amyloidosis Research Center Japan Samsung Medical Center, Seoul Korea

Online Amyloidosis App Clinical Trial Finder Speaker Engagements Industry Medical meetings Government Publications Patient Experience NT-ProBNP Posters Guidelines Educational ACC Round table ASH/HFSA satellite symposium AAHFN Advocacy Congressional meetings Rare disease caucus briefing FDA Publications Patient Voice AL Guidance ATTR Guidance Industry Advisory Council Expert Panel Program Patient Physician FDA engagement Round table PFDD Biomarker development Rare Disease think tank Improving Clinical Trials Natural History partnership Collaborative Network 25 Centers 3 studies identified

“Facilitating collaborative partnerships among government, academia, industry and patients groups is arguable the most important role that we can play in supporting advancement of drug development and regulation.” Janet Woodcock Director, FDA