NERDS 2023 : Program

Program

Day 1, Thursday, October 12th 2023 (Location: Derby Ballroom)

7:00 AM - 5:00 PM Conference Registration
7:00 AM - 8:00 AM Continental Breakfast
8:00 AM - 8:35 AM Welcome
- Co-Chairs: Kun Chen (U. of Connecticut), Richard Zhang (Pfizer)
- Opening Remarks
- NESS Representative: Rebecca Betensky (NYU)
- Industry Representative: Stephen Lake (Alexion)
8:35 AM - 10:25 AM Session 1: Keynote Presentation
- Session Chair: Ming Hui Chen (U. of Connecticut)
- S1.1: Strategies to Accelerate Rare Disease Drug Development - Janet Maynard (FDA). Slides
- S1.2: Innovative Thinking for Rare Disease Drug Development - Shein-Chung Chow (Duke University). Slides
- Panel Discussion: Lee-Jen Wei (Harvard). Slides
10:25 AM - 10:55 AM Refreshment and Networking Break
10:55 AM - 12:10 PM Session 2: Endpoint -Development, Selection and Validation
- Session Chair: Susan Wang (Boehringer Ingelheim)
- S2.1: Adaptive Endpoint Selection in Rare Disease Drug Development - Cong Chen (Merck & Co.). Slides
- S2.2: Evaluation of Biomarker Assessments in Conjunction with Clinical Endpoints for Early Decision Making in Rare Disease Drug Development - Carina Ittrich (Boehringer Ingelheim). Slides
- S2.3: Composite Endpoint in Cell/Gene Therapy Development - Yaohua Zhang and Bang Wang (Vertex Pharmaceuticals). Slides
12:10 PM - 1:10 PM Networking Luncheon (Location: Diamond Court AB)
1:10 PM - 2:25 PM Session 3: Trial Design - Challenges and Novel Approaches
- Session Chair: Lin Wang (Insmed)
- S3.1: Challenges of Mucopolysaccharidosis II (MPS II) drug development and path to acceleration of the pediatric rare disease drug development with novel endpoints - Yoonjin Cho (Regenxbio). Slides
- S3.2: Endpoints Selection and Efficacy Evaluation for Clinical Development in Rare Disease - Bo Huang (Pfizer). Slides
- S3.3: Design considerations in expanding lines of therapy for unmet medical need - Xin Wang (Bristol Myers Squibb). Slides
2:25 PM - 2:55 PM Refreshment and Networking Break
2:55 PM - 4:35 PM Session 4: RWE-Methods and Case Studies
- Session Chair: Sammi Tang (Servier)
- S4.1: SAM: Self-adapting Mixture Prior to Dynamically Borrow Information from Historical Data for Rare Disease Clinical Trials - Ying Yuan (MD Anderson). Slides
- S4.2: A Bayesian Nonparametric Model to Create Synthetic Matching Populations - Peter Mueller (U. of Texas at Austin). Slides
- S4.3: Leveraging Real-World Data and Real-World Evidence in Clinical Trial Design and Analysis for Rare Diseases - Chenguang Wang (Regeneron). Slides
- S4.4: An extended Bayesian divide-and-conquer analysis approach for hybrid control trials - Jian Zhu (Servier). Slides
5:00 PM - 6:00 PM Networking Reception (Location: Diamond Court AB)

Day 2, Friday, October 13th, 2023 (Location: Derby Ballroom)

7:30 AM - 9:30 AM Conference Registration
7:30 AM - 8:30 AM Continental Breakfast
8:30 AM - 10:10 AM Session 5: Statistical Inference
- Session Chair: Yang Song (Vertex)
- S5.1: A causal mediation model to evaluate the individual surrogacy of plasma neurofilament light chain (NfL) for SOD1 ALS using tofersen data - Peng Sun (Biogen). Slides
- S5.2: The use of balancing methods in ALS real-world settings and beyond - Marie-Abèle Bind (MGH)
- S5.3: Conditional and unconditional analyses for Bayesian historical data borrowing - Xiaodong Luo (Sanofi). Slides
- S5.4: Use of win statistics (win ratio, win odds and net benefit) for hierarchical analysis of clinical endpoints with low event rates and surrogate endpoints - Gaohong Dong (Beigene)
10:15 AM - 11:30 AM Session 6: Innovation
- Session Chair: Jeff Palmer (Pfizer)
- S6.1: An Exploration of Model-Based Dose Findings Algorithms for Use in Dose-Finding Gene Therapy Trials - Kevin Roberts (Pfizer). Slides
- S6.2: The Temporally-resolved Connectedness of Genetics, Imaging Biomarkers, and Clinical Endpoints in Alzheimer's disease - Ixavier A. Higgins (Eli Lilly)
- S6.3: AI/ML for drug discovery - Haoda Fu (Eli Lilly)
11:30 AM - 12:00 AM Refreshment and Networking Break
12:00 PM - 12:25 PM Closing Remark
- Co-Chairs: Kun Chen (U. of Connecticut), Richard Zhang (Pfizer)
12:25 PM - 12:25 PM Conference Adjourns