FIORD 2024 Agenda

Day 1 Schedule (November 7, 2024): 

Current Landscape of RWE

SpeakerDetails

Pallavi Mishra-Kalyani (Deputy Director, FDA)

Regulatory Considerations for Non-Randomized Studies in Drug Development

Pre-read: U.S. Department of Health and Human Services. (2024). Real-world evidence: Considerations regarding non-interventional studies for drug and biological products

Stephen Duffield (Associate Director, RWE Methods, National Institute of Health Care Excellence (NICE))

 Novel Analytics at The National Institute of Health Care Excellence. What Matters Most?

Weili He (Head of Medical Affairs & Health Technology Assessment Statistics, AbbVie)

Current Landscape of RWE - An Industry Perspective

Focused Sessions Day 1

Advanced Methods for Covariate Adjustment to Improve Precision in RCTs

SpeakerDetails

Daniel Rubin (Statistician, Division of Biometrics IV, Office of Biostatistics, OTS, CDER, FDA)

Adjusting for Covariates in Randomized Clinical Trials for Drugs and Biological Products

Pre-read: U.S. Department of Health and Human Services. (2024). Adjusting for covariates in randomized clinical trials for drugs and biological products.

Kelly Van Lancker (Postdoctoral Researcher, Applied Mathematics, Computer Science & Statistics, Ghent University)

Automated, Efficient and Model-Free Inference for RCTs via Data-Driven Covariate Adjustment

Pre-read: Van Lancker K, Díaz I, Vansteelandt S. (2024). Automated, efficient and model-free inference for randomized clinical trials via data-driven covariate adjustment. arXiv preprint arXiv:2404.11150.

Laura Balzer (Associate Professor, Biostatistics, UC Berkeley)

Collaborative TMLE to Maximize Empirical Efficiency in Randomized Trials

Pre-read: Balzer LB, Cai E, Godoy Garraza L, Amaranath P. (2024). Adaptive selection of the optimal strategy to improve precision and power in randomized trials. Biometrics

AI/ML Tools in Regulatory Submissions

SpeakerDetails

Hana Lee (Senior Statistical Reviewer, OB, OTS, CDER, FDA)

Artificial Intelligence and Machine Learning for Medical Product Development and Evaluation

Pre-read: U.S. Food and Drug Administration. (2024). Artificial Intelligence and Medical Products: How CBER, CDER, CDRH, and OCP are Working Together.

Gilmer Valdes (Director of Clinical AI and Associate Professor, Moffitt Cancer Center)

Interpretable AI/ML: Improving Generalizability and Evaluation of Untestable Assumptions

Pre-read: Gennatas ED et al. (2020). Expert-augmented machine learning. Proceedings of the National Academy of Sciences

Gene Pennello (Senior Statistician, DIDSR, CDRH, FDA)

Clinical Development of AI-Enabled Medical Devices: Challenges and Opportunities from Statistical and Regulatory Perspectives

Pre-read: Petrick N et al. (2023). Regulatory considerations for medical imaging AI/ML devices in the United States: concepts and challenges. Journal of Medical Imaging

Day 2 Schedule (November 8, 2024): 

Focused Sessions Day 2

Complex Innovative Trial Designs

SpeakerDetails

Ramin Ebrahimi (Senior Director in Biostatistics, Gilead Sciences)

Innovative Trial Designs in HIV Research at Gilead

Veronica Miller (Director, Forum for Collaborative Research)

Forum perspective on innovative trial designs in HIV research

James Peng (PhD Student, Biostatistics, University of Washington)

Surrogate Endpoint-Based Provisional Approval Causal Roadmap

Pre-read: Gilbert PB, Peng J, Han L, Lange T, Lu Y, Nie L, Shih MC, Waddy SP, Wiley K, Yann M, Zafari Z. A Surrogate Endpoint Based Provisional Approval Causal Roadmap. arXiv preprint arXiv:2407.06350

Lei Nei (Director, Division of Biometrics IV, Office of Biostatistics, CDER, FDA)

Borrowing Information from Non-Concurrent Controls to Enhance Efficiency in Platform Trials

Real-World Evidence Case Studies

SpeakerDetails

Rachael V. Phillips (Biostatistician, CTML, UC Berkeley)

Causal Targeted Machine Learning for Real-World Evidence Studies in Radiotherapy Oncology

Edward Bein (Statistician, Division of Biometrics VII, Office of Biostatistics, OTS, CDER, FDA)

Study 747-405: A Real-World Data Observational Study of the Efficacy and Safety of Ocaliva (OCA) for the Treatment of Primary Biliary Cholangitis (PBC)

Romain Neugebauer (Research Scientist, Kaiser Permanente Northern California)

Comparison of Type 2 Diabetes Pharmacotherapy Regimens using Targeted Learning and EHR Data: Approach and Results from the ONTARGET Diabetes Melitus Study

Data Integration

SpeakerDetails

Hector Izurieta (Associate Director for Novel Clinical Investigations, OVRR, CBER, FDA)

Real-World Evidence Study Methods that May Help Address Unmeasured Confounding

Pre-read: Izurieta HS, Wu X, Lu Y, Chillarige Y, Wernecke M, Lindaas A, Pratt D, MaCurdy TE, Chu S, Kelman J, Forshee R. (2019). Zostavax vaccine effectiveness among US elderly using real‐world evidence: Addressing unmeasured confounders by using multiple imputation after linking beneficiary surveys with Medicare claims. Pharmacoepidemiology and Drug Safety

Kara Rudolph (Associate Professor, Epidemiology, Columbia University)

How Measured Mediators can Help Achieve Identification for Transported Effects and Improve Understanding of Why Effects Differ Across Populations

Pre-read: Rudolph KE, Laan MJ. (2017). Robust estimation of encouragement design intervention effects transported across sites. Journal of the Royal Statistical Society Series B: Statistical Methodology

Henrik Ravn & Sky Qiu (Senior Statistical Director, Novo Nordisk & PhD Candidate, Biostatistics, UC Berkeley)

Comparison of Type 2 Diabetes Pharmacotherapy Regimens using Targeted Learning and EHR Data: Approach and Results from the ONTARGET Diabetes Melitus Study