Page 1 Page 2 Page 3 Page 4 Page 5 Page 6 Page 7 Page 8 Page 9 Page 10 Page 11 Page 12 Page 13 Page 14 Page 15 Page 16 Page 17 Page 18 Page 19 Page 20 Page 21 Page 22 Page 23 Page 24 Page 25 Page 26 Page 27 Page 28 Page 29 Page 30 Page 31 Page 32 Page 33 Page 34 Page 35 Page 36 Page 37 Page 38 Page 39 Page 40 Page 41 Page 42 Page 43 Page 44Big Data Emergin Alte Special Populations 26 MONDAY, SEPTEMBER 18, 2017 l Symposium 9 l 1:30 – 3:30 pm What to Do After a Pivotal Trial Has Failed Primary Endpoint Assessment: Totality of Evidence-based Drug Development Challenges & Opportunities APPLICATION TRACK CHAIR: Yan Xu, PhD, Associate Scientific Director, Global Clinical Pharmacology, Janssen: Pharmaceutical Co of Johnson & Johnson TARGET AUDIENCE: This Symposium will be useful for scientists from regulatory agencies, the pharmaceutical industry and academia to exchange their experience and views in totality of evidence for go/no-go decision and engage in active discussions, in particular what to do after a pivotal trial has failed primary endpoint assessment. GOALS AND OBJECTIVES: Following completion of this activity, the learner will be able to: 1. Apply totality of evidence to guide drug development decisions, eg considering the overall status of the pivotal trial, not just the primary endpoint; 2. Demonstrate a better understanding of evidence of effectiveness, which ought to be sought by establishing a body of evidence via multiple sources. A positive/negative p value by itself does not establish effectiveness or lack of evidence. No single index should substitute for scientific reasoning based on integrated knowledge; 3. List benefits and risks of sub-group analysis in drug development and personalized medicine. 1:30 – 1:35 pm Introduction Yan Xu, PhD, Associate Scientific Director, Global Clinical Pharmacology, Janssen: Pharmaceutical Co of Johnson & Johnson 1:35 – 2:05 pm Beyond a Failed Pivotal Trial: Scientific & Strategic Thinking Joga Gobburu, PhD, MBA, Professor of Pharmacy Practice & Science, Director, Ctr for Translational Medicine, Univ of Maryland School of Pharmacy 2:05 – 2:30 pm Drug Development Challenges & Opportunities Based on Totality of Evidence: An Industry Perspective Holly Kimko, PhD, Scientific Director/Fellow, Global Clinical Pharmacology, Janssen: Pharmaceutical Co of Johnson & Johnson 2:30 – 3:00 pm Challenges in Drug Approval Based on Total Evidence of Safety & Efficacy from a Positive Oncology Trial Sofia Paul, PhD, Senior Director, Biostatistics, Oncology, Novartis Pharmaceuticals Corp 3:00 – 3:30 pm Totality of Evidence in Regulatory Decision Making: Learning from Confirmative Trials Yaning Wang, PhD, Acting Director, Div of Pharmacometrics, Office of Clinical Pharmacology, Office of Translational Sciences, Ctr for Drug Evaluation & Research, US Food & Drug Administration Symposia