ROUNDTABLE Pediatric Patients – How to Measure the Risk/Benefit?
Saturday, June 3, 2023 10:15 AM - 11:15 AM
Room 242B
Clinic & the Lab
Roundtable
How does patient assessment change in the pediatric space?
Moderators
Susan Prockop, MD, Boston Children’s Hospital, USA
Chrystal Louis, MD, MPH, Zentalis Pharmaceuticals
Panelist
Yona Geffen, PhD, Vice President R&D, Gamida Cell
Marina Cavazzana, MD, PhD, Director of the Department of Biotherapy at Necker Hospital, Paris Cité University, France
Michael Schuster, MS, MBA, Head of Business Development and Project Management, Mesoblast, USA
Kevin Bosse PhD, RAC-US, CABP(H), Director of Regulatory Affairs, Nationwide Children’s Hospital, USA
Session Description
In this session the current FDA and EMA guidance for integration of pediatric populations into early phase trials will be reviewed. The session will then focus on assessing the risk/benefit ratio, access to patients and path(s) to approval of novel cellular therapeutics in pediatrics. The discussion will include evaluation of different trial designs and outcome measures such as sequential or simultaneous inclusion of pediatric patients, indications where the intervention is primarily in children, and metrics to assess long-term safety and efficacy. The session will conclude with brainstorming ways for academic, industry and patient advocacy groups to enhance the breath and depth of novel cellular therapy studies conducted in pediatric patients.
Learning Objectives
Moderators
Susan Prockop, MD, Boston Children’s Hospital, USA
Chrystal Louis, MD, MPH, Zentalis Pharmaceuticals
Panelist
Yona Geffen, PhD, Vice President R&D, Gamida Cell
Marina Cavazzana, MD, PhD, Director of the Department of Biotherapy at Necker Hospital, Paris Cité University, France
Michael Schuster, MS, MBA, Head of Business Development and Project Management, Mesoblast, USA
Kevin Bosse PhD, RAC-US, CABP(H), Director of Regulatory Affairs, Nationwide Children’s Hospital, USA
Session Description
In this session the current FDA and EMA guidance for integration of pediatric populations into early phase trials will be reviewed. The session will then focus on assessing the risk/benefit ratio, access to patients and path(s) to approval of novel cellular therapeutics in pediatrics. The discussion will include evaluation of different trial designs and outcome measures such as sequential or simultaneous inclusion of pediatric patients, indications where the intervention is primarily in children, and metrics to assess long-term safety and efficacy. The session will conclude with brainstorming ways for academic, industry and patient advocacy groups to enhance the breath and depth of novel cellular therapy studies conducted in pediatric patients.
Learning Objectives
- Understand current FDA and EMA guidance for integration of pediatric subjects into phase trials of novel cellular therapies (PIP requirement).
- Appreciate how the underlying disease, patient numbers, and the presence or absence of alternative therapies informs both registration trial design and hurdles for approval.
- Appreciate the burden of long-term follow-up in pediatrics.
Kevin Bosse PhD, RAC-US, CABP(H),
Director of Regulatory Affairs
Nationwide Children’s Hospital
Panelist
Director of Regulatory Affairs
Nationwide Children’s Hospital
Panelist
Marina Cavazzana MD, PhD
Director of the Department of Biotherapy at Necker Hospital
Paris Descartes University
Panelist
Director of the Department of Biotherapy at Necker Hospital
Paris Descartes University
Panelist