NERDS 2025 : Agenda

Agenda

NERDS 2026 Workshop Agenda

October 8-9, 2026

Day 1: Thursday, October 8, 2026

Time	Program	Details
7:45 AM – 9:15 AM	Registration / Light Breakfast	Charles View Ballroom (16^th floor)
8:15 AM – 8:20 AM	Day 1 Welcome Address	Zhaoyang Teng, NERDS Chair.
8:20 AM – 8:30 AM	Opening Remarks	Sammi Tang, Astellas Pharma.
8:30 AM – 10:00 AM	Keynote Session Moderator: TBD	Li Wang, AbbVie: “Statistical Challenges and Opportunities in the AI Agentic Era” Tianxi Cai, Harvard University: “Solving the Diagnostic Odyssey: Bridging Macro-Level EHR Subphenotypes and Micro-Level Missense Variants” Ping Gao, Innovatiostat: “Control of Unconditional Type I Error with Dynamic Bayesian Design: A Hybrid Frequentist-Bayesian Approach”
10:00 AM – 10:30 AM	Coffee Break / Networking
10:30 AM – 12:10 PM	Invited Talks: Session 1 Innovative Analysis Methods in Rare Disease Research Moderator / Chair: Qi Zhang	Pengling Sun, Pfizer: “A Bayesian Dynamic Borrowing Approach to Support Regulatory Approval of Marstacimab in Pediatric Hemophilia” Tristan Massie, Insmed: “Comparison of Methods for Handling Death and Missing Data in Survivors in the Analysis of Functional Outcomes in Amyotrophic Lateral Sclerosis (ALS)” Christian Stock, Boehringer Ingelheim: “Bayesian Mixed Models for Repeated Measures with Informative Priors” Annie Wang, Astellas: “Bayesian Hierarchical Dose-Response Model Averaging in Small Clinical Trials for Decision-Making”
12:10 PM – 1:10 PM	Lunch	Charles View Ballroom (16^th floor)
1:10 PM – 3:00 PM	Invited Talks: Session 2 AI and Machine Learning in Rare Disease Research Moderator / Chair: Frank Fan	Ahrim Youn, Sanofi: “Sample Size Reduction by Applying ML-Based Causal Inference Methods” Yanwei Zhang, Takeda: “Leveraging AI to Enhance Study Design for a Pivotal Phase 3 Rare Disease Program” Ning Leng, AbbVie: “From Modeling and Simulation to Decision: Architecting Human-AI Collaboration for Trial Design” Jin (Veronica) Liu, Takeda: “Agents Statisticians Can Trust in Regulated Domains: Rare Disease Trial Design as a Stress Test”
3:00 PM – 3:30 PM	Coffee Break / Networking
3:30 PM – 5:10 PM	Invited Talks: Session 3 Emerging Topics and Case Studies in Rare Disease Development Moderator / Chair: TBD	Ran Duan or colleague, Vertex: Title TBD Howard Mackey, Roche/Genentech: “Opportunities for Innovation Are Hiding in Plain Sight, Let's Find Them” Kaushik Patra, Ultragenyx: “Designing a Pivotal Trial Where a Placebo Is Impossible: External Controls and a Surrogate-Endpoint Accelerated-Approval Strategy for Gene Therapy in Sanfilippo Syndrome Type A” Award Winner, TBD: Title TBD
5:10 PM – 6:00 PM	Poster Session	Mt. Auburn Ballroom (14^th floor)
5:30 PM – 7:00 PM	Reception	Mt. Auburn Ballroom (14^th floor)

Day 2: Friday, October 9, 2026

Time	Program	Details
7:45 AM – 9:15 AM	Registration / Light Breakfast	Charles View Ballroom (16^th floor)
8:15 AM – 8:20 AM	Day 2 Welcome Address	Mary Salvana, NERDS Co-chair.
8:30 AM – 10:00 AM	Keynote Session Moderator: TBD; Discussant: TBD	Kelley Kidwell, University of Michigan: “Interim Monitoring in snSMART Designs with Multiple Endpoints” C. Lee Cohen, FDA: “Advancing Rare Disease Drug Development: DRDMG Perspective”
10:00 AM – 10:30 AM	Coffee Break / Networking
10:30 AM – 12:10 PM	Invited Talks: Session 4 Innovative Clinical Trial Design in Rare Diseases Moderator / Chair: Cong Han	Bryan McComb, Pfizer: “Reframing BASIS as a Basket Trial Master Protocol: Lessons for Rare Disease Drug Development” Alex Sverdlov, Novartis: “A Quantitative Framework for the Design and Optimization of N-of-1 and N-of-Few Trials in Rare Neurological Diseases” Silvia Colicino, BMS: “From Dose Finding to Decision Making: Comparing 3+3, BOIN, and BF-BOIN Designs and Integrating Go/No-Go Criteria in Early-Phase Development” Yingdong Feng, Eli Lilly and Company: “Doing More with Fewer: What Hierarchical Composite Endpoints and Win Statistics Offer Rare Disease Trials, an ALS Illustration”
12:10 PM – 12:30 PM	Closing Remarks	Charles View Ballroom (16^th floor)