NERDS 2024 : Program

Program

Day 1, Thursday, October 10th 2024 (Location: )

7:00 AM - 5:00 PM: Conference Registration
7:00 AM - 8:00 AM: Continental Breakfast
8:00 AM - 8:30 AM: Welcome and Opening Remarks
- Co-Chairs: HaiYing Wang (U. of Connecticut), Yingwen Dong (Sanofi)
- Opening Remarks
- NESS Representative: Shuangge Ma (Yale University) Slides
- Industry Representative: Xun Chen (Sanofi) Slides
8:30 AM - 10:00 AM: Keynote Session 1
- Session Chair: HaiYing Wang (U. of Connecticut)
- Innovative Statistical Approaches to Help Accelerate Rare Disease Drug Development: Bayes and Beyond - John Scott (FDA) Slides
- Advancing Rare Disease Drug Development through snSMART Design - Kelley Kidwell (U of Michigan) Slides
- Panel Discussion: Miganush Stepanians (Prometrika) Slides
10:00 AM - 10:30 AM: Refreshment and Networking Break
10:30 AM - 12:10 PM: Session 1: Novel Endpoint Development and Regulatory Use
- Session Chair: Cong Li (Takeda)
- S1.1: A simulation study to evaluate the statistical efficiency of different endpoint strategies for multi-system diseases888 - Kristine Rosenberger (Boehringer Ingelheim) Slides
- S1.2: EVIDENCE meta-analysis to support MRD as an endpoint for accelerated approval for multiple myeloma - Sean Devlin (Memorial Sloan Kettering Cancer Center) Slides
- S1.3: MRD-negative CR used as a novel endpoint to support the accelerated approval of Iclusig in newly diagnosed Ph+ ALL - Bingxia Wang (Takeda) Slides
- S1.4: From Rare to Reality: Leveraging Data to Optimize Endpoint and Clinical Development for Rare Diseases - Angela Qu (PARAXEL) Slides
12:10 PM - 1:10 PM: Networking Luncheon
1:10 PM - 3:15 PM: Session 2: Innovations in Statistical Design and Analysis
- Session Chair: Kristine Rosenberger (Boehringer Ingelheim)
- S2.1: Propensity-score regression with the sequential borrowing-by-parts power prior to efficiently borrow external control data in RCTs - Eric Baron (Servier)
- S2.2: Leveraging Real-World Data as External Controls for Single Arm Trials with Count-Type Endpoints in Rare Diseases: Utilizing Multiple Entries – a Simulation Study - Tianyu Sun (Moderna) Slides
- S2.3: The usage of external control data in rare disease drug development - Menghan Hu (Sarepta) Slides
- S2.4: Two stage adaptive design with treatment switching - Lingyun Liu (Vertex)
- S2.5: The use of a Bayesian approach to analyze outcomes in a Giant Axonal Neuropathy (ultra-rare) indication - Denis Rybin (Pfizer) Slides
3:15 PM - 3:45 PM: Refreshment and Networking Break
3:45 PM – 5:00 PM: Session 3: Innovation in Cell and Gene Therapy: Dose optimization, prediction and real-world evidence
- Session Chair: Chunpeng Fan (Insmed)
- S3.1: Challenges of underdosing control in dose-escalation studies for DNA-based gene therapies - Quang Nguyen (Regeneron)
- S3.2: A Bayesian Approach for Selecting Relevant External Data (BASE) to Study Long-term Outcomes In Gene Therapies - Xiang Zhang (CSL) Slides
- S3.3: Real-World External Control Arm Study in Supporting Cell Therapy Single-arm Trial for Pre-Marketing Regulatory Decision Making - Lihua Yue (Bristol Myers Squibb) Slides
5:00 PM - 6:00 PM: Networking Reception

Day 2, Friday, October 11th 2024 (Location: )

7:30 AM - 9:30 AM: Conference Registration
7:30 AM - 8:30 AM: Continental Breakfast
8:30 AM - 10:00 AM: Keynote Session 2
- Session Chair: Yingwen Dong (Sanofi)
- Clinical and Endpoint Considerations in SMA and ALS - Douglas Kerr (Dyne Therapeutics) Slides
- Causal inference options for a Phase 3 single-arm study with external control - Stephen Lake (Alexion, AstraZeneca Rare Disease) Slides
- Discussant: Lee-Jen Wei (Harvard) Slides
10:00 AM - 10:30 AM: Refreshment and Networking Break
10:30 AM - 12:10 PM: Session 4: Statistical methods and causal inference
- Session Chair: Ran Duan (Vertex)
- S4.1: The use of balancing methods in ALS real-world settings and beyond - Marie-Abèle Bind (MGH)
- S4.2: Use of Generalized Pairwise Comparison in Rare Diseases - Marc Buyse (Founder of Cluepoint, IDDI, One2Treat, Hasselt University) Slides
- S4.3: Causal Inference - Don Rubin (Harvard) Slides
- S4.4: Robust Inference of Treatment Effect in Platform Trials - Ting Ye (U of Washington) Slides
12:10 PM - 12:30 PM: Closing Remarks