2. 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

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

4. 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.

5. Momentum - A Century of Discovery
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.

6. 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

7. 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.

9. TDN Timeline

10. Number of Studies in the TDN 2012 - 2018

11. Number of Protocols Reviewed

12. 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

13. Family
Sponsor
IRB
Site
Hospital
Vendors
CRO