Graybill Conference 2024

Hosted by Colorado State University in Fort Collins, CO

June 9 – 12, 2024

“Rare diseases are rare, but rare disease patients are numerous.” – Orphanet.

Finding cures for pediatric and rare disease is now a global health priority. According to Orphanet (https://www.orpha.net/consor/cgi-bin/index.php), a 37-country network, reported 6172 unique rare diseases, affecting 300 million people worldwide, about 72% of which are genetic and about 70% of which are exclusively pediatric onset. More than 90% of rare diseases are without an FDA-approved treatment.

Rare diseases are numerous, heterogenous in nature, and geographically disparate. Developing a treatment for pediatric and rare diseases presents unique challenges. Various public policy, regulatory initiatives have been implemented in US and rest of the world to help reduce the barriers. Given its limited patient pool, the success of advancing patient care and treatment options hinges on multi-stakeholders and interdisciplinary collaboration. The conference is organized to dive deep in each of those areas with the goal to bring more effective and safe treatments to patients diagnosed with rare diseases worldwide.

Travel Information

Conference Center Address

Lory Student Center

1101 Center Ave Mall

Fort Collins, CO 80521

Air Travel

If your trip to the conference involves air travel, the closest major airport is Denver International Airport (DEN). Denver International Airport is served by numerous major international and national airlines, and offers connections to many other U.S. and international hubs.

Getting to Fort Collins from Denver International Airport
Rental Car

If traveling by rental car, we recommend using a Maps App, such as Google Maps, Apple Maps, etc. The venue address is 1101 Center Ave Mall, Fort Collins, CO 80521. The drive to Fort Collins from Denver takes approximately 1 hour, but depends on traffic. This drive may have tolls, depending on the route you choose in your maps app.

Taxis

Taxi cabs and mobile taxi services (Uber/Lyft) are also available, but are fairly expensive ($120-350).

Airport Shuttle (Recommended)

Groome Transportation offers an airport shuttle that will take you from the airport directly to Colorado State University’s Lory Student Center. The ride, which takes about 1 hour, 20 minutes will cost $30-40. You must purchase this option in advance by visiting the Groome Transportation website.

When booking online, choose “FC – CSU Transit Center” and “Denver International Airport” as your destinations.

Train / Bus

For the Train / Bus option, you will follow signs in Denver International Airport to the A-line. Once you purchase your ticket ($5-10), get on the A-line headed toward Union Station and depart the train at Union Station.

From Union Station, you will exit the train terminal onto Wewatta Street, which is where the bus picks up. Purchase your ticket ($10) and board the NORTH line toward Fort Collins and depart at Downtown Transit Center in Fort Collins.

From the transit center, you will purchase your ticket (free at this time) and board the MAX line bus to Laurel Street Station and depart at the Colorado State University Campus. From here, follow signs for the Lory Student Center.

Accommodations

Best Western University Inn

914 S. College Ave, Fort Collins, CO 80524

(970) 484-2984

Website

Hilton Fort Collins

425 W Prospect Rd, Fort Collins, CO 80526

(970) 482-2626

Website

Registration

Registration will open soon.

Information about registration can be found on the WNAR/IMS/Graybill website.

Early bird registration ending date is TBD.

Important Dates

November 1, 2023

Deadline for submission of 2024 WNAR/IBS Outstanding Impact Award and Lectureship

January 10, 2024

Deadline for submission of invited session proposals (submit here)

TBD

Deadline for abstracts of Invited Oral Presentations

February 15, 2024

Deadline for Contributed Oral abstract submissions

February 15, 2024

Deadline for Student Paper abstracts

TBD

End of early bird registration

April 8, 2024

Deadline for full paper submissions of students participating in Student paper competition

TBD

End of online registration

Program

Short Courses

Innovative Methods for Incorporating Real-World and Historical Data into Clinical Trials

Chair & Organizer: Tianjian Zhou, Colorado State University

  • Dehua Bi, University of Chicago. PAM-HC: a Bayesian nonparametric construction of hybrid control for randomized clinical trials using external data
  • Meizi Liu, Takeda. Propensity-score based meta-analytic predictive prior for incorporating real world data
  • Noirrit K. Chandra, University of Texas at Dallas. Bayesian nonparametric common atoms regression for generating synthetic controls in clinical trials
  • Hongfei Li, Incyte. An informative prior for borrowing historical data under the mixture cure rate model
Design and Analysis in Clinical Trials

Chair & Organizer: Zhezhen Jin, Columbia University

  • Shikun Wang, Columbia University. A general backward joint model of longitudinal and survival data with application to dynamic prediction
  • Toshimitsu Hamasaki, George Washington University. Risk assessment in clinical trials
  • Ying Lu, Stanford University. The trade-off between bias and efficiency in borrowing RWD in hybrid designed trials
  • Shangwu Samuel Wu, University of Florida. Pick-the-winner sequential parallel comparison designs
Bridging the Knowledge Gap: Advances in Pediatric Extrapolation

Chair & Organizer: Vickie (Yuanye) Zhang, Servier BioInnovation

  • Hengrui Sun, FDA. Regulator’s view on pediatric extrapolation, ICH E11A and other guidelines
  • Vickie (Yuanye) Zhang, Servier BioInnovation. Mechanism of action-based Bayesian extrapolation strategy to accelerate pediatric drug development
  • Mayadah Shabbout, GSK. Extrapolation analysis plan for an integrated pharmacokinetic, safety, and efficacy open-label trial for the treatment of anemia associated with chronic kidney disease in male and female children and adolescents aged 3 months to under 18 years requiring dialysis
  • Antara Majumdar, GSK. Discussion
Endpoint Development and Analysis Considerations for Rare Disease Clinical Trials

Chair & Organizer: Rebecca Chiu, FDA

  • Monica Morrell, FDA. Applications of patient-focused drug development in rare disease programs
  • Mitch Thomann, Boehringer Ingelheim. Developing a clinical endpoint model for hidradenitis supperativa
  • Satrajit Roychoudhury, Pfizer. Dynamic enrichment of Bayesian small sample, sequential, multiple assignment randomized trial (snSMART) design using natural history data: a case study from Duchenne muscular dystrophy
  • Yared Gurmu, FDA. Global tests for multiple endpoints in rare disease trials
Utilizing External Data to Inform Drug Development in Pediatric Oncology

Chair & Organizer: Pallavi Mishra-Kalyani, FDA

  • Lindsay Renfro, Children’s Oncology Group. Considerations for external controls in pediatric oncology
  • Arup Sinha, FDA. Regulatory considerations for and examples of external controls in pediatric oncology drug development
  • Haitao Pan, St. Jude’s Hospital. Evaluating the type I/II error rates in trials incorporating external control data
  • Satrajit Roychoudhury, Pfizer. Discussion
Opportunities, Gaps and Promises Brought by Digital Health Technology

Chair & Organizer: Yaohua Zhang, Vertex Pharmaceuticals

  • Vincent Tan, Vertex Pharmaceuticals. New kids on the block: analyzing data from state-of-the-art diabetes devices
  • Maria Matilde Kam, FDA. Regulatory perspective of applying digital health technologies in clinical trials
  • Vadim Zipunnikov, Johns Hopkins University. Developing more sensitive endpoints by leveraging novel statistical methods for Digital Health Technologies (DHTs) data
  • Nishit Agarwal, Medidata. Redefining patient assessment in clinical trials with digital technologies
Bayesian Methods in Clinical Drug Development

Chair & Organizer: Antara Majumdar, GSK

  • Ming-Hui Chen, University of Connecticut. Bayesian re-design of a new pediatric trial via borrowing information from concurrent adult trials and historical pediatric and adult trials with similar mechanism of action
  • James Rogers, Metrum Research Group. Evaluating conditional exchangeability assumptions for Bayesian borrowing, with application to pediatric extrapolation
  • Illana Trumble, GSK. Building a hybrid external control arm using a fusion of Bayesian borrowing and causal inference
  • Hengrui Sun, FDA. Discussion
Statistical Methods for Environmental Epidemiology

Chair & Organizer: Tianjian Zhou, Colorado State University

  • Kayleigh Keller, Colorado State University. Mitigating measurement error and confounding in cohort studies of air pollution and cognitive aging
  • Ander Wilson, Colorado State University. Heterogeneous distributed lag models to estimate personalized effects of maternal exposures to air pollution
  • Glen McGee, University of Waterloo. Testing for non-additive interaction in flexible Bayesian models for multi-pollutant mixtures
  • Shanshan Zhao, National Institute of Environmental Health Sciences. Accommodating limit of detection of multiple exposures in environmental mixture analyses: an overview of statistical approaches
Statistical Models for Rare Disease Studies

Chair & Organizer: Yuan Ji, University of Chicago

  • Joseph Ibrahim, University of North Carolina at Chapel Hill. The scale transformed power prior for time-to-event data
  • Tianjian Zhou, Colorado State University. RoBoT: a robust Bayesian hypothesis testing method for basket trials
  • Alexander Kaizer, University of Colorado Anschutz Medical Campus. Increasing the statistical efficiency of rare disease research studies through master protocol designs
  • Yuan Ji, University of Chicago. Discussion
Subgroup and Small Sample Analysis Issues

Chair & Organizer: Brian Wiens, ACELYRIN

  • Yao Chen, Novartis. Workflow to assess treatment effect heterogeneity in clinical drug development
  • Peter Thall, MD Anderson Cancer Center. Bayesian treatment screening and selection using subgroup-specific utilities of response and toxicity
  • David Shera, MTSS Group. Employing mixed models for longitudinal data and small sample sizes: evaluating gene therapy for Canavan’s disease
Panel Discussion: Regulatory Innovations and Collaborations to Promote Rare Disease Development

Chair & Organizer: Erik Bloomquist, Merck
Moderator: Brian Wiens, ACELYRIN

  • Dionne Price, FDA
  • Guei-Feng (Cindy) Tsai, Taiwan CDE
  • Andrew Thompson, EMA
  • Frank Pitavy, EMA

Organizing Committee

Program Committee

BeiGene (Chair)
Columbia University
ACELYRIN
Colorado State University

Local Organizing Committee

Colorado State University (Chair)
Colorado State University
Colorado State University

History

The Graybill Conference has been hosted annually by the Department of Statistics at Colorado State University. Dr. Franklin A. Graybill came to Colorado in 1960 and was involved in the development of the statistics program, first as part of mathematics and then in its own department. He founded the Graybill Statistical Laboratory in 1961, which recently celebrated its 50th year in operation.

Read more about the history here.