Sub-teams

Modified

June 21, 2024

Real World Evidence/Real World Data (RWE/RWD)

Lead: Khadija Rantell (MHRA), Shihua Wen (Novartis)

Objective: The objective of this WG is to develop recommendations for generating robust RWE/RWD to support regulatory approval for CGT products. Specifically, the WG will review methods for direct and indirect comparisons using RWD and reflects on the opportunities and challenges of these approaches in the setting of CGT drug development using case studies as examples.

Firstname Lastname Institution
Zhenzhen Xu FDA
Khadija Rantell MHRA
Lina Yang Novartis
Shihua Wen Novartis
Yeonhee Kim NA

Estimand

Lead: Nicolas Ballarini (Novartis)

Objective: The objective of this WG is to promote the concept of estimands and establish a harmonized estimand framework for the clinical development of Cell and Gene Therapies (CGT). Specifically, the WG’s aim is to identify and address the unique challenges posed by intercurrent events in CGT development, and formulate appropriate strategies for their handling.

Firstname Lastname Institution
Yiran Hu Abbvie
Cong Han Astellas
Alessandro Previtali BMS
Khadija Rantell MHRA
Alex Sverdlov Novartis
Nicolas Ballarini Novartis
Shihua Wen Novartis
Avery McIntosh Pfizer

Dose Finding

Lead: Revathi Ananthakrishnan (BMS), Rong Liu (Regeneron)

Objective: To make the biopharmaceutical community aware of the dose finding methods and designs that CGT development can already adopt and identify improvements in these methods for the future as well as key challenges in dose finding in CGT development.

Firstname Lastname Institution
Cong Han Astellas
Daniel Li BMS
Revathi Ananthakrishnan BMS
Frank Shen BMS
Hao Sun BMS
Ally He Crisprtx
Alan Chiang Lyell
Andrew Lewandowski Novartis
Bambang Adiwijaya Novartis
Rong Liu Regeneron

Chemistry, Manufacturing, and Controls (CMC)

Lead: Sangwook Choi (BMS)

Objective: To identify key CMC challenges in CGT development and to serve as key opinion leaders in CGT CMC within biopharmaceutical communities

Firstname Lastname Institution
Sangwook Choi BMS
Bill Pikounis JNJ
Jose Ramirez Kitepharma
Alan Chiang Lyell
Lina Yang Novartis

Continuing Education and Outreach

Lead: Patricia Anderson (ICON plc)

Objective: To promote best practices in the design and analysis of cell and gene therapy trials through knowledge sharing of current global development in the field with focus on the following:

  1. Types of cell and gene therapies (what they are, how they work, what are the important considerations for statisticians)
  2. New and updated regulatory guidance
  3. New therapy approvals
  4. Industry hot topics or safety findings (eg, secondary malignancy investigations, new safety AEs)

In addition, the subteam supports requests for short courses and other presentations as well as general outreach.

Firstname Lastname Institution
Cong Han Astellas
Revathi Ananthakrishnan BMS
Patricia Anderson ICON plc
Alan Chiang Lyell
Khadija Rantell MHRA
Nicolas Ballarini Novartis
Shihua Wen Novartis

Trial Design

Lead: Weidong Zang (Sana)

Objective: The objective of this subgroup is to research and evaluate clinical trial design options in CGT development focusing on registrational trials. Specifically, we aim to explore new ideas and innovative approaches to provide insights, best practices, and recommendations to CGT drug development community on effective designs to accelerate drug approvals in various therapeutic areas including but not limited to oncology, autoimmune, and rare genetic disorders.

Firstname Lastname Institution
Daniel Li BMS
Revathi Ananthakrishnan BMS
Zhenzhen Xu FDA
Patricia Anderson ICON plc
Evgeny Degtyarev Novartis
Nicolas Ballarini Novartis

Long-Term Follow Up (LTFU)

Lead: Shihua Wen (Novartis)

Objective: Promote statistical thinking and best practices in the design and conduct of long-term follow-up studies for cell and gene therapy products through sound statistical methodology and innovative technology.

Firstname Lastname Institution
Patricia Anderson ICON plc
Alan Chiang Lyell
Alex Sverdlov Novartis
Shihua Wen Novartis

Data Science and Artificial Intelligence

Lead: Qinghua Song (Kitepharma), Md Shamsuzzaman (BMS)

Objective: Understand the opportunities and challenges associated Data Science and application of AI and Data Science in CGT (long term)

Serve as key opinion leaders in CGT Data Science & AI within biopharmaceutical communities (long term)​

Share presentation and publication (short term)

Firstname Lastname Institution
Daniel Li BMS
Md Shamsuzzaman BMS
Zhenzhen Xu FDA
James Whitmore Kitepharma
Qinghua Song Kitepharma
Alan Chiang Lyell