Part I: Introduction to Biostatistics
Resources at NYULH
Huilin Li Ph.D. Judy Zhong Ph.D.
Associate Professor Professor
Director of NYUBR Director of CTSI BERD Program
Division of Biostatistics Division of Biostatistics
Department of Population Health Department of Population Health
June 16, 2022,
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Where is NYUBR?
Department
of Population • Chair: Marc N. Gourevitch, MD, MPH
Health
Division of • Director: Andrea B. Troxel, ScD
Biostatistics
NYU • Director: Huilin Li, PhD
Biostatistics • Associate Director:
Resource
Jiyuan Hu, PhD
Location: 180 Madison Ave, the second, fourth and fifth floors
Biostatistics-resource@[Link]
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NYUBR Research Team
• 7 Professors • 6 PhDs
• 6 Associate • 16 Masters
Professors
• 9 Assist.
Professors
Research
Faculty Scientists
Postdocto
Admin ral
Staff
Fellows
• 6 Postdocs
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Our Expertise:
• Study design
• Advanced analytical methods
• Data (clinical) coordinating center creation
and implementation
• Core leadership in center grants
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Our Expertise: Study Design
“TO CONSULT THE STATISTICIAN AFTER AN EXPERIMENT IS
FINISHED IS OFTEN MERELY TO ASK HIM TO CONDUCT A POST
MORTEM EXAMINATION. HE CAN PERHAPS SAY WHAT THE
EXPERIMENT DIED OF.”
Ronald A. Fisher (1890 – 1962)
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Our Expertise: Study Design
Reproducible
Research Needs
Good Design!
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Our Expertise: Study Design
Observational Experimental
Standard
Clinical
Cohort Adaptive
Trials
Pragmatic
Case-
control Multifactor
Laboratory
Longitudinal
Complex
survey
High
EHR throughput
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Our Expertise: Advanced Analytical
Methods
Hierarchical Survival Longitudinal
model fitting analysis analysis
Network Clustering, High-dimensional
analysis classification, omics data
and prediction analysis
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Our Subject Knowledge
Environmental
science Ophthalmology Orthopedics Addiction
Behavioral
Geriatric care economics Cancer Genetics
Early detection Chronic
and screening HIV Microbiome disease
Molecular
Palliative care Diabetes Drug use epidemiology
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Types of Biostatistics Collaborations
Short-term support Grant proposals Long-term support
• developing pilot • assisting in refining • proposal
study designs study questions and development
• performing statistical measurement • manuscript writing
analysis on a methods • statistical analysis
prepared dataset • developing study • presence at
• preparing abstracts and experimental departmental
or other designs research meetings
presentations • writing statistical • participation in
• preparing analysis plans journal clubs
manuscripts (either • computing precision, (methodological
initial or for a power, and sample review)
revision) sizes
• assistance with
research conference
presentations, and
K-award mentoring
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Core Resources
NYUBR
The Environmental Alzheimer’s
BERD at Disease
Biostatistics Health
Clinical and Center’s Data Other cores
Shared Statistics and
Translational Management
Resources at Bioinformatics
Science and Statistics
Perlmutter Facility Core
Institute (CTSI) Core
Cancer Center (EHSB)
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Biostatistics, Epidemiology and Research Design Program (BERD) in NYU-H+H
Clinical & Translational Science Institute (CTSI)
Translational research transforms basic science discoveries into practical applications
that can improve health, such as new diagnostic techniques, therapies, and
interventions.
T0
Define mechanisms
underlying health
problems or
disease
Yields knowledge about
defining mechanisms,
targets, or lead molecules
BERD provides biostatistical, epidemiological, and study design collaborations for projects
with translational research component
• Consultations
• Grant developments and data analyses (vouchers can be applied)
• Studios (multidisciplinary project reviews)
• IRB scientific reviews
• Trainings
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Self-service Tool Page and K-award Resource Repository
Biostatistics Tool K Application Resource Repository
Enable investigators independently implement relatively Enable efficient mentoring with shared resources on
straightforward tasks: • successful grant writing and summary statements
• Power calculators examples
• Sample size calculators • career development templates and examples
• Data analysis calculators (t-test, correlations, linear • biostatistics resources and training catalogs
regressions, ROCs etc.) • letter of support templates
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Biostatistics Shared Resource–Perlmutter Comprehensive Cancer Center (PCC)
To provide state-of-the-art statistical collaboration and consultation to members of the
Perlmutter Cancer Center (PCC) basic science, clinical and translational research
programs and Shared Resources.
BSR provides biostatistical, epidemiological, and study design collaborations provided by
dedicated cancer-focused faculty and staff of Biostatistics.
1. Develop new peer-reviewed grant applications
2. Develop research designs and protocols for investigator-initiated interventional
treatment clinical trials
3. Provide ongoing statistical collaboration for PRMC-approved, PCC investigator-
initiated interventional treatment clinical trials
4. Develop research designs and protocols for new PRMC-approved, investigator initiated
clinical trials, non-interventional, and observational studies
5. Training and mentoring of PCC members and staff in key statistical issues for study
design, analysis, and interpretation of cancer research studies
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NYUBR Collaboration Process
Scientific Cores &
Division of Shared Resources—
Biostatistics website Biostatistics
Resource website
Inquiry Form
Contact us:
Biostatistics-resource
@[Link]
NYUBR
Alzheimer
CTSI Cancer Center EHSB Others
Core
Designated fa culty and staff
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Thank you
NYU Biostatistics Resource email:
Biostatistics-resource@[Link]
Division of Biostatistics website:
[Link]
sections-centers/biostatistics/research
Scientific Cores & Shared Resources—Biostatistics Resource website:
[Link]
resource
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Part II: Tips for Preparing Grant
Proposals from Statistical Reviewers
Panelists: Drs. Andrea B. Troxel, Huilin Li, Judy Zhong,
Mengling Liu, Erinn M. Hade
Moderated by Dr. Jiyuan Hu
June 16, 2022,
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Perspectives from a Statistical Reviewer in
Study Sections and Grant Review Panels
• Study sections often have a statistical reviewer for applications involving
human subjects and basic science projects involving omics analysis
• Statistical review emphases:
• Significance
• Is the proposed hypothesis statistically testable?
• Investigators
• Does the team have statistical expertise to design, monitor and
analyze?
• Are the budgeted efforts sufficient to implement the scope of work?
• Innovation
• Innovative design of the trial and analysis of the observational data
is especially appreciated
• Approach
• Is the proposed strategy likely to produce unbiased and
interpretable results?
• Does the study provide adequate power, use an appropriate study
population?
• Does the application appropriately account for sex and relevant
18 biological variables, and potential confounders?
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General Tips
• Initial contact
• Translate clearly defined scientific questions into statistical hypotheses
• Ensure subject enrollment and sample collection address scientific question and enable data analysis
• Study design
• Driven by scientific questions: interventional vs. observational, retrospective vs. prospective, targeted
vs. untargeted, fixed vs. adaptive, controlled vs. pragmatic.
• Determined by projected effect sizes: preliminary studies or literature
• Constrained by resource: accessibility to study population, funding, compliance factors
• Preliminary results
• Generate hypothesis and provide rationale of the proposed investigation
• Provide supporting information to plan the project
• Sample Size and Power
• Sample size often determined for primary outcome; show sufficiency for secondary outcomes
• Projected effect size: clinical meaningful and well supported by preliminary results or literature
• Account for multiple testing and potential missing
• Simple models and assumptions for power analysis
• Statistical analysis plan
• Address the scientific questions: clarity is key with proper approaches (not over-complicated)
• Cover potential questions: missing data, adjusting confounders, nonlinear effects, dimensional
reduction
• Potential pitfalls and solutions
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Specific Tips for Large P or Center Grant
• Longer preparation time and larger team
• Multiple faculty biostatisticians to provide sufficient expertise
supports
• Biostatistics core
• Coordinate with the other administrative cores
• Collaborate with all research projects
• Statistical plans
• Summarize core activities across the entire the core document
• Specific analysis plans for each research project
• Show synergy/interactions across projects and cores
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Specific Tips for Clinical Trial Grant
• Clinical Trial
• Justification for choice of design
• Discussion of randomization/masking
• Clear statement of primary/secondary/exploratory objectives
• Consideration of interim analyses, monitoring, and DSMB
• Data & Safety Monitoring Board
• General consideration
• Align Board expertise with trial goals
• Budget considerations
• CTSI resource
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Specific Tips for Other Types of Research Grant
• Observational Study
• Validation cohort is important
• Ancillary study of the ongoing cohorts
• Secondary analysis of existing study
• Molecular Mechanism Study
• High dimensional data—multiple testing adjustment
• Association vs. Prediction
• Analysis approaches to reflect the updates in modern technology
• Batch effect
• Streamlined upstream and downstream data analysis
• Integrated analysis taking account of study design, omics data, and
clinical outcomes
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Role of Statisticians by Grant Mechanisms
• Large P or Center Grant – Core Director, Co-I, or statistical analyst
• Two-phase grant UG3/UH3 – MPI, Co-I, or statistical analyst
• Large research grant: R01/U01 – MPI, Co-I, or statistical analyst
• Small research grant: R03/R21 – Co-I
• Training grant – Mentor
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Case Study with the Suggested Timeline
4-6 weeks
Review/refi
ne study
design, 1-2 weeks
hypothesis,
preliminary Finalize the
8 weeks statistical
analysis
Contact analysis
statistician plan
6-8 weeks 2-4 weeks 0 week
Introduce Finalize the Submission
background, study
exchange design and
literature sample
size
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Thank you
NYU Biostatistics Resource email:
Biostatistics-resource@[Link]
Division of Biostatistics website:
[Link]
centers/biostatistics/research
Scientific Cores & Shared Resources—Biostatistics Resource website:
[Link]
CTSI Data & Safety Monitoring Board [Link]
institutes/clinical-translational-science/services-investigators/research-support/data-
safety-monitoring-board
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