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Trk 13: CER/Glob Health Econ [clear filter]
Tuesday, June 28
 

4:00pm

#271: The Things Kids Say: Clinical Outcome Assessments in Pediatric Clinical Trials
Limited Capacity seats available

Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-570-L01-P; CME 1.25; IACET 1.25; RN 1.25

Patient perspectives in clinical trials are a regulatory focus. Challenges exist when considering pediatric patients. This forum will further explore FDA guidance, challenges, and solutions regarding clinical outcome assessments/patient-reported outcomes in pediatric clinical trials.

Learning Objectives

Define clinical outcome assessments (COAs), including patient-reported outcomes (PROs), with special attention to the FDA PRO guidance document; Describe possible challenges when implementing pediatric COAs, as exemplified in case studies involving pediatric functional constipation and sickle cell disease; Describe best practice recommendations for the development, selection, and use of COAs in pediatric trials.

Chair

Gina Calarco

Speaker

Overview of Regulatory Perspective of Developing and Utilizing COA/PRO in Pediatric Clinical Trials
Andrew E. Mulberg, MD

Case Studies for the Development of COA/PRO Tools for Use in Pediatric Clinical Trials
Diane Turner-Bowker, PhD



Chair
avatar for Gina Calarco

Gina Calarco

Associate Director, Pediatric Center of Excellence, Quintiles
Gina Calarco is an Associate Director of Project Management and the Deputy Head of the Pediatric Center of Excellence (PCoE) at Quintiles. Ms Calarco completed her BSN at Saint Luke’s College and MPH from the University of Kansas. She is an affiliate member of the American Academy... Read More →

Speakers
avatar for Andrew Mulberg

Andrew Mulberg

Deputy Division Director, Gastroenterology and Inborn Errors Products, OND, CDER, FDA
Andrew E. Mulberg, MD, FAAP, CPI, is Associate Professor of Pediatrics University of Pennsylvania and Professor of Pediatrics at Unviersity of Maryland. He has principally edited a book entitled Pediatric Drug Development: Concepts and Applications published in 2009 and 2013 with... Read More →
avatar for Diane Turner-Bowker

Diane Turner-Bowker

Director, Patient-Centered Outcomes, Adelphi Values
As Director of Patient-Centered Outcomes at Adelphi Values, Diane Turner-Bowker provides senior scientific leadership on the development of COAs for use in medical product development to support regulatory, payer, and communication strategies, as well as for use in clinical practice... Read More →


Tuesday June 28, 2016 4:00pm - 5:15pm
103A Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
 
Wednesday, June 29
 

10:30am

#322: Real-World Evidence in Drug Development: Creating the Right Environment for Enhanced Pre-Launch Evidence
Limited Capacity seats available

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-16-571-L01-P; CME 1.00; IACET 1.00; RN 1.00

Ideally, health technology assessment and other decisions at launch are informed by an understanding of the (added) value of treatments for patients. In this session, we will explore the barriers and enablers for using real-world data in generating effectiveness evidence in R&D.

Learning Objectives

Identify the positive and negative incentives for generating effectiveness evidence in the current environment; Distinguish different study designs and forms of evidence synthesis using real-world data that can provide input to decision makers; Identify potential solutions for addressing current challenges for generating effectiveness evidence in the pre-launch environment.

Chair

Pieter Stolk, PharmD, PhD

Speaker

The Environment for Enhanced Pre-Launch Evidence: An EU Perspective
Chris Chinn, MSc

The Environment for Enhanced Pre-Launch Evidence: A US Perspective
Gregory Daniel



Chair
PS

Pieter Stolk

Project Manager, University Medical Centre Utrecht
Pieter Stolk, PhD, was trained as a pharmacist with a PhD from Utrecht University. Pieter is interested in all aspects of medicines regulation and policy (with a focus on marketing authorisation and HTA of medicines). He has been involved in several EU public private partnerships... Read More →

Speakers
CC

Chris Chinn

Head of Real World Data Strategy and Partnerships, Sanofi
Chris graduated from Oxford University with a degree in Biochemistry. He qualified as a chartered accountant with Ernst & Young in London and completed a MSc in Health Economics at City University, London. He has led health outcomes research teams at Eli Lilly and GSK, and in his... Read More →
avatar for Gregory Daniel

Gregory Daniel

Deputy Director and Clinical Professor, Duke-Margolis Center For Health Policy
Gregory Daniel, PhD, MPH is the Deputy Director of the Duke-Robert J. Margolis, MD Center for Health Policy and a Clinical Professor in Duke's Fuqua School of Business. He directs the DC-based office of the Center leading its pharmaceutical and medical device policy portfolio, and... Read More →
avatar for Gregory Daniel

Gregory Daniel

Deputy Director and Clinical Professor, Duke-Margolis Center For Health Policy
Gregory Daniel, PhD, MPH is the Deputy Director of the Duke-Robert J. Margolis, MD Center for Health Policy and a Clinical Professor in Duke's Fuqua School of Business. He directs the DC-based office of the Center leading its pharmaceutical and medical device policy portfolio, and... Read More →
PS

Pieter Stolk

Program Manager, Escher, the Lygature Platform For Regulatory Innovation
Pieter Stolk, PhD, was trained as a pharmacist with a PhD from Utrecht University. Pieter is interested in all aspects of medicines regulation and policy (with a focus on marketing authorisation and HTA of medicines). He has been involved in several EU public private partnerships... Read More →


Wednesday June 29, 2016 10:30am - 11:30am
107AB Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA