Bridging Patient Needs with Regulatory Flexibility in Rare Disease Drug Development
Bilal AbuAsal, PhD, US Food and Drug Administration, Silver Springs, MD

Approval of Eteplirsen: Patient Advocate Perspective
Katherine Lambertson, BS, BA,  Genetic Alliance, Washington, DC

Industry Perspective on Rare Disease  Drug Development
Keith M. Gottesdiener, MD, Rhythm Pharmaceuticals, New York, NY

Drug Development in Rare Diseases (Amyloidosis): Consortium Perspective 
Kristen Hsu, BS, Amyloidosis Research Consortium, Newton, MA

SYMPOSIUM: Demonstrating Biosimilarity with Clinical PK and PD Data in Lieu of Comparative Efficacy
The Role of PK and PD in the Regulatory Framework for Biosimilars Approval 
Yow-Ming Wang, PhD, US Food and Drug Administration, Silver Spring, MD

The Role of Mechanistic PKPD Modeling in Explaining Variability in Efficacy Outcomes for Biosimilars
Wojciech Krzyzanski, PhD, University at Buffalo, SUNY, Buffalo, NY

ORAL ABSTRACT SESSION III: Drug Transporters and Pharmacogenomics
OIII-001 INTERACTIONS OF AZO DYES COMMONLY USED IN ORAL DRUG PRODUCTS WITH THE ORGANIC ANION TRANSPORTING PEPTIDE 2B1 AND HUMAN GUT BACTERIA.
Presenter: Ling Zou, PhD, University of California, San Francisco

OIII-002 CHARACTERIZING THE ONTOGENY OF TEN RENAL TRANSPORTERS IN AFRICAN AMERICANS USING QUANTITATIVE PROTEOMICS, GENE EXPRESSION ANALYSIS AND CLINICAL DATA. 
Presenter: Kit Wun Kathy Cheung, PharmD, University of California, San Francisco

OIII-003 PRECLINICAL EVALUATION OF  NUDT15 GENOTYPE-GUIDED THIOPURINE DOSE INDIVIDUALIZATION USING CRISPR-CAS9 MOUSE MODEL. 
Presenter: Rina Nishii, MS, St. Jude Children’s Research Hospital

OIII-004 TARGETED SEQUENCING IDENTIFIES MISSENSE VARIANT IN THE BEST3 GENE ASSOCIATED WITH ANTIHYPERTENSIVE RESPONSE TO THIAZIDE DIURETICS.
Presenter: Sonal Singh, PhD, University of Florida

WORKSHOP: Substrate-Dependent Polymorphic Effects in CYP-Mediated Drug Metabolism and Challenges for Pharmacogenetics Implementation
Substrate-Specific Metabolism of CYP2D6: How can CYP2D6 Phenotype Prediction  be Improved?
Andrea Gaedigk, PhD, Children's Mercy, Kansas City, MO

Accommodating Substrate-Dependence in CYP Genotype to Activity Phenotype Translation for Pharmacogenetic Implementation
Kelly Caudle, PharmD, PhD, St. Jude Children's Research Hospital, Memphis, TN

SYMPOSIUM: Pragmatic Approaches to Improvements in Pediatric Drug Therapy
Building Expertise in Pediatric Formulations: How to Improve Expertise of and Access to Pediatric Formulations 
Catherine Litalien, MD, CHU Ste-Justine, Montreal, QC, Canada

Drug Safety in Pediatrics: Shifting from Catching up to Moving Forward 
Michael Rieder, MD, PhD, University of Western Ontario, London, ON, Canada

Novel Approaches to Clinical Trials: How Smart Design Can Improve Yield 
Saskia N. de Wildt, MD, PhD, Radboud University, Nijmegen, The Netherlands

SYMPOSIUM: Innovation in Clinical Dose Selection and Trial Optimization using Bayesian Approaches: Steps Toward Accelerated Patient Care

Chairs: Indrajeet Singh, PhD, Janssen Pharmaceuticals, Spring House, PA and Ying Ou, PhD, Amgen, South San Francisco, CA

Bayesian Statistics and its Implications for Drug Development
Stephen Ruberg, PhD, Eli Lilly, Indianapolis, IN

Bayesian Adaptive Trials in Oncology Drug Development - Maximizing the Synergy Between Statisticians and Clinical Pharmacologists
Stuart Bailey, PhD, Novartis, Cambridge, MA

Challenges and Opportunities of Bayesian Adaptive Trials: Regulatory and Pharmacometrics Perspectives
Yaning Wang, PhD, US Food and Drug Administration, Silver Spring, MD

Challenges and Opportunities of Bayesian Adaptive Trials: Where Do We Go From Here?
Carl C. Peck, MD, University of California, San Francisco Center for Drug Development Science, San Luis Obispo, CA