PS4 Back to the Future: What's New and Coming Next in Cell and Gene Therapy

Room: Salon E 

The rapid advancement of Cell and Gene Therapy (CGT) heralds an exciting new era for patients with devastating diseases around the world. While significant clinical responses to such therapies generate enthusiasm and more regulatory approvals by the day, the unique features of these therapies require collaborative education and pose new challenges in the design and analysis of such studies. Apart from the initial challenges of dose finding for CGT, as various additional types or versions of CGT products become available, further challenges arise. These include, but are not limited to, demonstration of the superiority of a new CGT product as CGTs emerge as standard of care, implementation of the estimand framework appropriate to CGT special considerations, use of real-world data and real-world evidence (RWD/RWE) to support or supplement CGT product registration or reimbursement, use of artificial intelligence/ machine learning (AI/ML) predicting patients who likely to respond to the therapy or experience toxicity, and design of decentralized trials for long-term follow up.

This session will highlight the recent innovations in statistical methodologies and application, to address the unique challenges facing the future of cell and gene therapies. After two shorter presentations, the session will feature a panel of speakers from various backgrounds to share their insights and stimulate discussion. This session is given on behalf of the American Statistical Association Cell and Gene Therapy Scientific Working Group.


1) Introduction to CGT Beyond CAR T-Cell Therapy for Statisticians and Data Scientists


CAR T-cell therapy is used as a motivating example in many cases. However, there are a multitude of other cell and gene therapies that bring their own considerations. In this session, we will briefly introduce other types of cell and gene therapies, for example NK cell therapies, gene therapy and gene editing technology, that may have different manufacturing and production, administration, persistence, toxicity profiles, and efficacy. Statisticians and data scientists should have keen awareness of the aspects of various therapies to be best equipped to serve and advise in our roles.


2) Lessons Learned and Statistical Challenges in the Future of CGTs


As cell and gene therapies are accepted in earlier lines of therapy and more CGT are available as approved treatments, the future may include comparison of new CGT to existing CGT products as well as standard of care. What designs could suitable in these spaces and what considerations are needed? For example, three Phase 3 cell therapy studies, ZUMA-7, TRANSFORM and BELINDA, are conducted to investigate similar anti-CD19 CAR-T therapy under same indication but lead to dramatic discrepant results. What are the methodological reasons behind the discrepancy? What are the lessons learned to inform the proper design of future cell therapy studies?


3) Panel Discussion, including applications of real-world evidence and AI to CGT

CGT therapy patients treated in the commercial setting need to be followed up in a registry. The use of such registry data or real-world evidence can be appealing to CGT studies to enhance efficacy, patient safety, and future study planning. How can we seize the application of the real-world evidence studies, potentially in combination with artificial intelligence, to apprehend this fast-moving field? Panelists will include members from the American Statistical Association Cell and Gene Therapy Scientific Working Group and other invited guests including regulators.

cell therapy

gene therapy

real world

artificial intelligence

innovation

data science 

Clinical Trial Design (e.g., Innovative/Complex Design, Estimands, Master Protocol)

ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop 2024

Presenting Author

Md Shamsuzzaman

Presenting Author

James Whitmore, Kite Pharma

Presenting Author

Bindu Kanapuru, FDA (OCE)

Presenting Author(s)

Josh Chen, Vertex
James Whitmore, Kite Pharma
Bindu Kanapuru, FDA (OCE)
Md Shamsuzzaman