AGENDA

9:30 AM: Networking Breakfast

10:00 AM: Opening Remarks

• Ellen Sigal, Friends of Cancer Research

10:10 AM: Session 1 – Seamless Trial Designs for Rare Cancer Drug Development 
Randomized controlled trials are often not feasible for rare diseases due to limited patient populations, ethical considerations, and time constraints. Seamless trial designs offer a promising path forward by enabling more flexible, adaptive approaches across development phases. Panelists examined how to design seamless trials that are both scientifically rigorous and operationally viable, what types of data can support regulatory decision-making in the absence of a control arm, and how thoughtful endpoint selection and design flexibility might help reduce patient burden while maintaining confidence in trial results. (Click to Watch the Session 1 Discussion Preview)

Session 1 Presentation Slides

Panel Discussion and Q&A 

• Allen Melemed, Chimerix, a Jazz Pharmaceuticals Company – Moderator  

• Harindra Abeysinghe, Johnson & Johnson
• Lola Fashoyin-Aje, Parexel

• Misha Mehta, Friends Advisory Advocate

• Chris Turner, Nuvalent, Inc.

• Ying Yuan, MD Anderson Cancer Center

11:40 AM: Session 2 – Trial Designs for Combination Drug Development 
Drugs in oncology are increasingly provided in combination, which require demonstration of the contribution of effect for each drug in the combination to support drug development and regulatory approval. This process can be challenging to implement due to resource and timing constraints. Panelists in this session discussed how to efficiently adapt trial designs and provide robust evidence for each agent’s contribution of effect, which statistical methods best isolate treatment effects, and how regulatory expectations could evolve to improve clarity and consistency in evaluating combination therapies. (Click to Watch the Session 2 Discussion Preview)

Session 2 Presentation Slides

Panel Discussion and Q&A

• Gideon Blumenthal, Merck & Co., Inc. – Moderator 

• Julie Bullock, GSK

• Gary Doherty, AstraZeneca

• Elad Sharon, Dana-Farber Cancer Institute

• Carol Vallett, Friends Advisory Advocate

12:45 PM: Lunch

1:20 PM:  Lunch Discussion – Future Policy Perspectives

• Jeff Allen, Friends of Cancer Research – Moderator 

• Anna Abram, Akin

• Remy Brim, BGR Group

• Roy Herbst, Yale Cancer Center

• Amy McKee, AstraZeneca

1:55 PM: Session 3 – Control Arm Selection for Multi-Regional Clinical Trials 
Control arm selection for multi-regional clinical trials (MRCTs) is increasingly complex due to regional differences in treatment availability, regulatory expectations, and evolving standards of care. This variability has the potential to undermine the interpretability and acceptability of trial results across regulatory agencies globally. This session explored the need for practical, science-based strategies to guide control arm decisions that are both regionally relevant and globally credible. Additionally, the utilization of transparent frameworks for supporting operational feasibility and data acceptability was explored. (Click to Watch the Session 3 Discussion Preview)

Session 3 Presentation Slides

Panel Discussion and Q&A

• Harpreet Singh, Precision for Medicine – Moderator

• Judd Englert, Amgen Inc.

• Sumithra Mandrekar, Mayo Clinic

• Kristin McJunkins, Friends Advisory Advocate 

• Raymond Osarogiagbon, Baptist Hospital Group

• Kathleen Winson, Genentech

3:00 PM: Meeting Adjournment