Sub-teams
Real World Evidence/Real World Data (RWE/RWD)
Lead: Khadija Rantell (MHRA), Shihua Wen (Novartis)
Objective: NA
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 | Hang | 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 |
---|---|---|
Daniel | Li | BMS |
Revathi | Ananthakrishnan | BMS |
Ally | He | Crisprtx |
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:
- Types of cell and gene therapies (what they are, how they work, what are the important considerations for statisticians)
- New and updated regulatory guidance
- New therapy approvals
- 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 | Hang | 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: NA
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 |