Site, Sponsor, CRO: Understanding Roles & Expectations Creating a Team Approach Alan Genatossio, BSN, RN Seattle Children’s, Seattle, WA Colleen Dunn, RRT Stanford University, Palo Alto, CA

Presenter Disclosure Alan Genatossio, RN Colleen Dunn, RRT There are no relationships to disclose related to this presentation.

Momentum - A Century of Discovery Historical awareness – 18th Century German saying “A child whose forehead tastes like salt when kissed will soon die.” Delta F508 gene at least 50,000 years old, may confer a survival advantage to carriers -protect from cholera and other GI inflammatory conditions 1938 First comprehensive medical report by Dorothy Anderson, MD - life expectancy was 6 months, pancreatic enzymes replacement first used to treat affected children 1955 The Cystic Fibrosis Foundation forms and awards the first research grants to Dorothy Andersen, M.D., Paul di Sant'Agnese, M.D., and Harry Shwachman, M.D.

Momentum - A Century of Discovery 1962 30 Foundation accredited care centers are established – predicted median survival reaches 10 years 1966 Patient Registry launched to collect health information from care centers 1978 Number of accredited CF Care Centers reaches 100 1982 Research Development Program started 1989 Foundation supported scientists discover the defective CF gene 1993 FDA approves dornase alfa - the first drug developed specifically for CF.

Momentum - A Century of Discovery 1997 FDA approves inhaled tobramycin 1998 Therapeutics Development Network founded with 7 TDCs and the coordinating center 2000 Pseudomonas aeruginosa’s genetic structure mapped 2002 Research shows azithromycin improves CF lung health 2004 Hypertonic saline shown to improve CF lung health 2006 Ivacaftor enters clinical trials 2010 FDA approves aztreonam for inhalation

Momentum - A Century of Discovery 2012 FDA approves Kalydeco® 2015 FDA approves Orkambi® 2017 The Foundation maintains a robust pipeline targeting the disease from every angle.

TDN Timeline

Number of Studies in the TDN 2012 - 2018

Number of Protocols Reviewed

Where are we now? As an academic and hospital based research community we have: Experience – nearly a decade of scientific progress and 20 years of network facilitated multi-site clinical trials. Enthusiasm – the pipeline is aggressive, results are positive, hope has never been higher Structure – national clinical research infrastructure with 89 TDCs, Support – CFF funding, TDN Coordination, Mentoring Systems to help Turnover Stress

Family Sponsor IRB Site Hospital Vendors CRO