Monday, January 26, 2009

Sessions to be discussed on Jan 30

With the help of the steering committee, we have reduced the redundancy in the list of sessions suggested. The following 41 candidate sessions will be discussed at the Organizational meeting on Jan 30. Of the 41 sessions listed below, approximately 30 sessions will be selected based on a vote at the meeting.

A Pre-clinical/Tox
1 PK-PD Analysis
2 Toxicology

B Adaptive Designs
3 Adaptive Design - Theory and Methods
4 Adaptive Design - Applications and Examples
5 Adaptive Design - Other Issues (interactions between FDA/Industry)

C Safety
6 Future directions in planning safety analysis and risk management
7 Quantitative risk/benefit assessment
8 DMC and Safety Monitoring
9Thorough QTc study design and analysis

D Global Trials
10 Regulatory Issues in Global Harmonization
11 Statistical Issues in Global Harmonization

E Phase I Trials
12 Statistical and Design Issues in Phase I trial (cross over, blinding)

F Statistical Issues in Clinical Trials
13 Design Issues in Clinical Trials: Placebo/Historical Control/ Randomization
14 Interim Analysis/Futility analysis
15 Subgroup Analysis/Heterogeneity /Adjusting for Covariate
16 Survival Analysis
17 Statistical Modeling/ Simulation for Decision making

G Longitudinal Data Analysis and Missing Data
18 Longitudinal Analysis, Missing Data and Durability

H Bayesian
19 Bayesian Analysis and Designs

I Multiplicities/Multiple Comparison
20 Multiplicities/Multiple Comparison

J Meta Analysis
21 Meta Analysis

K Non-inferiority Trials
22 Non-Inferiority Trials - Designs and Issues

L Quality of Clinical Program
23 Quality of Clinical Programs- Designs/Methodological/Robustness

M Genomics/Biomarkers
24 Biomarker Evaluation
25 Genomics

N Technical/Computations/Graphics
26 Open-source software/global platform

O Therapeutic Area Specific Issues
27 Oncology I - Issues
28 Oncology II. - Designs and Analysis
29 Psychiatric Clinical Trials/ CNS/Insomnia

P Regulatory Issues
30 ICH-E9/ISS/ISE Analysis plans
31 Communication between FDA/Industry statisticians

Q Career Development
32 Statistical Consulting

R Patients Reported Outcome
33 Patients Reported Outcome

S Chemistry, Manufacturing and Control (CMC)
34 CMC I

T Food Safety
36 Food

U Veterinarian Medicine
37 Veterinary Medicines I - Design and Analysis
38 Veterinary Medicines II - Issues

V Devices
39 Devices I - Medical
40 Devices II - Diagnostics

W Vaccines
41 Vaccines

Saturday, January 24, 2009

Agenda of Friday January 30th Meeting

Organizational Pre-Meeting-
FDA/Industry Statistics Workshop 2009
The Universities at Shady Grove
January 30, 2009
8:00 a.m. to 4:30 p.m.

8:00 a.m. ...... Continental breakfast available
8:30 ............... Logistics of Shady Grove University
.......................... Introductions
9:15 ................ 2009 FDA/Industry Statistics Workshop Brief Update
9:45 ...................... 15 minute Break
10:00 .............. Workshop logistics
10:15 ............... Brief Discussion Short Course
10:45 ............... Wrap Up Short Course Discussion
11:00 ............... Brief Discussion Session topics
11:45 ............... Description of voting mechanism

12:00 noon ................ LUNCH & VOTE

1:15 p.m. ....... Presentation of Top Selection of Topics/Sessions
1:30 ............... Session logistics
1:45 ............... Review of Workshop themes and theme selection
2:00 ............... Creation of session committee’s (chairs/organizers)
2:30 .......................... 15 Minute Stretch Break
2:45 ................ Summary of Expectations:
..............................Reminder of Roles & Responsibilities
..............................Break out
3:45 ............... Summary of Meeting
................................Reminder of Tasks
4:00................ Depart

Meeting held at: The Universities at Shady Grove Conference Center
Address: 9630 Gudelsky Drive Rockville, MD 20850
Phone: 301.738.6059

Friday, January 16, 2009

Reminder to RSVP to the FDAIndustry Organizational Meeting

If you intend to attend the organizational meeting, please RSVP to

In this e-mail please indicate your meal preference (meat-chicken/vegetarian) as well as your phone number and professional affiliation (particularly if it not obvious).

We look forward to seeing you on Jan 30, 2009.

Additionally updates will be posted during the next 2 weeks, so please keep checking back on this blog!

Thursday, January 15, 2009

Jan 15 - Deadline for submission of courses/sessions

Thank you for your interesting suggestions for courses and sessions. We have received over 100 session and 20 course ideas!

The lists of suggested sessions and courses are posted below. If you have made a suggestion but do not see it posted, please drop Tammy/Carmen a note at In the coming weeks, to prepare for the January 30th organization meeting, we will be consulting with our steering committee to better organize the ideas.

Please come back and check often for updates.

Also, a quick reminder that all conference correspondences should be directed to our conference e-address:, instead of our work emails. Tammy and I have both been put in e-jail for the volume of emails we've received. This will also ensure that your emails reach us safely.

Submitted Session Proposals (as of Jan 15th)

A. Pre-clinical/Tox

  1. Individualized medicines? Role of PK-PD analysis
  2. Overview of Toxicology (Preclinical)
  3. Using PK-PD modeling to support selection of First-In-Human dose

B. Adaptive Designs

  1. Adaptive Designs - Implementing a Bayesian Outcome
  2. Recent Development in Adaptive Design
  3. Bayesian Adaptive Designs
  4. Unconditional use of Conditional Power
  5. Adaptive Group Sequential Designs
  6. Adaptive Design in practice - Successful implemetationof early/late stage AD
  7. Case Studies in Adaptive Design
  8. Adaptive Dose Ranging Studies
  9. Non-proportional Hazards, Adaptive Design, Group-Sequential Design

C. Analysis of Safety Data

  1. Safety Analysis and the Role of DSMB
  2. Analysis Approaches for Pre-marketing Safety Data
  3. FDA ISS Guidelines And SPERT Initiatives on Safety Analysis
  4. Benefit/Risk, Motivation of Methods
  5. Rare events monitoring
  6. Large Simple Trial for safety evaluation
  7. Risk-benefits
  8. Safety, PSAP and Risk Management
  9. Risk Management Plan: Risk Analysis and Management.
  10. Causal Analysis of Safety Data

D. Global Trials

  1. Global Harmonization
  2. Multi-Regional Clinical Trials
  3. Changing Landscapes - Statistical Issues in Global Clinical Development
  4. Post Marketing Research, Global Perspective

E. Phase I Trials

  1. Statistical challenges in Ph I Drug Development
  2. Phase 1 trials: Blinded or Unblinded at the Sponsor Level?

F. Statistical Issues in Clinical Trials

  1. Cross-Over Design with time-to-event endpoint
  2. Placebo Response and Mitigation Strategies in Clinical Trials
  3. Role of statistical modeling in early decision makings of drug development
  4. Statistical Issues in Identifying Early on-Tx Measures of Efficacy
  5. Statistical Issues in assessing Durability of Effects
  6. Forced Randomization in Clinical Trials
  7. Extracting information from blinded trials
  8. Futility Analysis
  9. Interim Analysis
  10. Subgroup Heterogeneity in Drug Effects
  11. Post Randomization Adjustment of Covariates
  12. Non-parametric Resampling techniques in Clinical Trials
  13. Statistical (Robustness) assessment of clinical development programs
  14. Analysis of Covariance in RCT
  15. Experience with Flexible Bayesian Plan in Phase II Biologic

G. Longitudinal Data/Analysis and Missing Data

  1. Longitudinal Analysis in dealing with missing values
  2. Missing Data/Multiple Imputation
  3. Sensitivity analysis for longitudinal studies with withdrawals in practice
  4. Joint Modeling of Longitudinal and Event Time Data
  5. Approaches in dealing with missing data

H. Bayesian

  1. Bayesian Methodologies
  2. Bayesian Interim Analysis
  3. Type I Error Rate Related Issues for Bayesian Trial Design in a Regulatory Environment

I. Multiplicities/Multiple Comparison

  1. Multiplicity in assessing multiple studies
  2. Multiple Comparisons
  3. Multiple Endpoints
  4. False Discovery Rates

J. Meta Analysis

  1. Analysis of subgroups across studies, in the meta-analytic framework
  2. Comparative effectiveness via indirect comparisons/historical control

K. Non-inferiority Trials

  1. Issues in Non-inferiority Trials, margins, population, historical control

L. Quality of Clinical Trials

  1. Methodological quality of clinical trials
  2. Assuring Data Quality in Clinical Trials

M. Vaccines

  1. Vaccines - Multiple Comparisons and Analysis in Vaccines Trials
  2. Sensitivity analysis for missing data in vaccine trials

N. Genomics/Biomarkers

  1. Prospective Calculation of Gene Identification power in analysis controlling the false discovery rate
  2. Analysis of Large Biomarker Data sets
  3. Defiing Performance Characteristics of Clinical Biomarker Assays
    > Variance Function based statistical method for optimal quality assessment for RT-QPCR replicate well performance
    > statistical metrics to quantify relative bias due to Rt-qPCR assay
  4. Integrating Evidence for Selecting Diagnostic Biomarkers for Drug Dev
  5. Enabling biomarker integration in the drug development
  6. Assessing Clinical Utility of Biomarkers developed by consortia/institutions in Genetics/Genomics
  7. Prospective calculation of gene identification power in analyzes controlling the false discovery rate
  8. Weibull Failure Time Mixture Models for evaluating Efficacy in the Presence of Biomarker
  9. Statistical Issues for Micro-array classifiers.

O. Technical/Computations/Graphics

  1. Use of Open-source software for regulated clinical Trial research
  2. Global platform for global clinical trials
  3. Strategies for Statistical Software Development for Drug and Diagnostics Research

P. Therapeutic Area Specific Issues

  1. Specific Issues in Oncology Trials
  2. CNS Issue - Diary Data
  3. CNS Risk - suicidal ideation
  4. Cost Effective Go/No Go Decision in Oncology
  5. Endpoint Issues in Oncology Clinical Trials
  6. Censoring Issues in Survival Analysis
  7. Adequacy of patient follow-up and sensitivity analyses in the presence ofmissing data for PFS in oncology trials
  8. Statistical Issues in Design and Analysis of Psychiatric Clinical Trials

Q. Regulatory Issues

  1. Changing Political landscape in Drug Development - Discussions of Avandia and other blockbuster drugs that came under dark clouds
  2. ICH-E9 Update - what works and what doesn't
  3. Communicating to FDA statisticians unambiguously using CDISC ADaM
  4. Issues regarding generation of a SAP for ISE/ISS
  5. Unifying Data in FDA and its use in drug review

R. Career Development

  1. Effective communication with clinicians
  2. Statistical Consulting
  3. Working Session on Improving communications between project statisticians and FDA statistical reviewers
  4. A statistical Career - beyond power calculation and p-values

S. Patient Reported Outcome

  1. PROMIS, the NIH Roadmap for a Patient Reported Outcomes Measurement Information System.
  2. Patient Reported Outcomes
  3. Determining validity of patient reported outcomes (PROs) as endpoints in clinical research settings
  4. A review of the impact of the draft PRO Guidance document on the statistical analysis of PRO data.

T. QTc Study

  1. Thorough QTc study design and analysis
  2. Statistical Issues in Thorough QTc study

U. Chemistry, Manufacturing and Control (CMC)

  1. ICH-Q8 Design Space: Statistical Strategies and Case Studies
  2. Issues in Chemistry, Manufacturing, and Control (CMC)
  3. Increasing Objectivity in Risk-based Approach for Biopharmaceutical Development, Manufacturing, and Control
  4. The role of multivariate projection methods in Quality by Design

V. Food Safety

  1. Food Safety-Collection and analysis of safety data of food borne pathogens

W. Veterinarian Medicine

  1. Important Statistical Concepts in the Planning, Analysis and Monitoring of Veterinary Clinical Trials
  2. Assay Validation in Veterinary Biologics and Pharmaceuticals
  3. Monitoring Assay Stability in Veterinary Biologics and Pharmaceuticals
  4. Measuring the Effect of Intervention in Veterinary Biologics and Pharmaceuticals
  5. Study Design Issues in Veterinary Biologics and Pharmaceuticals

X. Devices

  1. Drug Device Co-Development Considerations
  2. Approaches and Problems with Allergy Device Perfromance Validation
  3. Regulatory and Statistical Issues for Companion Diagnostics in Drug-Device combination products

Tuesday, January 6, 2009

January 30th Organizational Meeting

The January 30th Organizational Meeting will be held at the Universities at Shady Grove Conference Center from 8 a.m. - 4 p.m.

The Universities at Shady Grove is located off Shady Grove Road in Rockville, MD. There are several hotels located near the Washington Rio which is within 1 mile of this meeting location.

If you are driving, the Shady Grove University (and Washington Rio Hotels) is conveniently located off the major Interstate (I-370). If you are using the train, the meeting location is about a 10 minute taxi-ride from the Metro.

More details will follow including an Agenda.

If you would like to RSVP to attend this meeting please contact Carmen/Tammy at *** Please also indicate whether you'd like chicken or vegetarian lunch. ***

Friday, January 2, 2009

Proposed Short Courses (to date as of Jan. 15)

  1. Adaptive Designs
  2. Data Safety Monitoring Committee's
  3. Working Discussions on Statistical Consulting (interactive small course for <30> pariticpants)
  4. Statistical Graphics
  5. Roles and Strategies for Covariate Adjustment in RCTs
  6. Introduction to Clinical trials (FULL DAY appropriate for statisticians with 5 years experience)
  7. Examination of Risk/Benefits
  8. Missing Data Analysis
  9. Dose Finding
  10. Longitudinal Analysis
  11. Survival Analysis
  12. Safety Analysis
  13. Bayesian Analysis
  14. Meta Analysis
  15. Likelihood Methods in RCT
  16. Resampling Techniques in Medical Research
  17. Biomarkers (selecting, identifying, assay issues)
  18. Assessing and Improving Trial Quality
  19. Interim Analyses and unblinding
  20. Non-proportional Hazards, Adaptive Design, Group-Sequential Design

Happy New Year and Brief Update on Workshop details!

Happy New Year!

Carmen and I are excited about the upcoming FDA/Industry Workshop we are co-chairing in Fall 2009.

The conference location/hotel has been arranged: the Capitol Hilton right next to the White House.

The conference date has also now been confirmed: Wed.-Fri. Sept. 23-25th.

We look forward to a productive organizational meeting on January 30, 2009 at the Shady Grove University. If you are interested in participating please contact us at:

We wish you a Safe and Happy New Year and look forward to seeing you in Sept 2009 or perhaps at the organizational meeting in late January!