Science, Statistics and Decisions - The Right Questions and the Best Answers


General Session 1: Update on Critical Path with Special Attention to the Role of Statistical Science


In 2004 FDA announced a new initiative referred to as "a Critical Path Research Initiative" which had as its focus finding additional ways through applied scientific studies to develop better methods and new technologies that may improve the product development process. Since this announcement, statisticians have considered what their contribution to this process might be. This session will focus on the role of statistical science in enhancing more efficient, more effective and safer product development.

Powerpoint Format G1_Offen_CriticalPath
Powerpoint Format G1_Simon_CriticalPath
Powerpoint Format G1_Oneill_Critical Path
Powerpoint Format G1_Anello_Critical Path Intro

General Session 2: Bayesian Trial Designs - Case Studies


Bayesian clinical trial designs offer great opportunity to increase the efficiency of product development and regulatory review. This session will focus discussion on the potential benefits and current challenges in implementing these approaches. Bayesian designs in clinical trials for both drug and medical devices will be illustrated through case studies. Leaders in Bayesian trial design from academia, industry and regulatory agency will present their perspectives and possible solutions to the challenges encountered in implementing Bayesian designs. Regulators will share some practical experiences and the emerging issues that have arisen from these experiences. Statistical considerations, logistical issues, and regulatory agency feedback will be discussed.

Powerpoint Format G2_Mugglin_Bayesian
Powerpoint Format G2_Berry_Bayesian
Powerpoint Format G2_Panel Questions_Bayesian

General Session 3: Data Monitoring Committees: Practical Considerations and Useful Strategies


The 2001 FDA Draft Guidance on the Establishment and Operation of Clinical Trial Data Monitoring Committees (DMCs) provides useful insight and guidance regarding the role a DMC may play in a drug development program. Several practical issues, though, are likely to surface when implementing a DMC. This session will provide insight into the issues facing pharmaceutical companies and the FDA when a clinical trial involves a DMC -- issues such as the use of internal versus external DMCs, partial versus complete unblinding, information sharing across DMCs, and the advantages and disadvantages of having an independent statistician perform the interim analysis. Critical issues surrounding the statistical assessment of safety endpoints will also be examined. Strategies, both prospective and retrospective, to enhance safety signal detection in randomized clinical trials will be discussed.

Powerpoint Format G3_Omeara_DMCs
Powerpoint Format G3_Snapinn_DMCs
Powerpoint Format G3_Schwab_DMCIntro
Powerpoint Format G3_Wittes_DMC2

General Session 4: Pharmacogenomics


Technical and other issues abound when incorporating molecular and/or genomic biomarkers in clinical drug development. This is especially true in disease areas where the molecular pathways are not well established or when the mechanism of action of the pharmacologic effect is not well understood. These issues are also thorny in disease areas where the typical drug development program may involve the use of underpowered Phase II's, which is precisely that point in drug development when the exploration of candidate biomarkers would be most useful. This session will discuss these issues from both statistical and drug development strategy perspectives.

Powerpoint Format G4_Cabrera_Pharmacogenomics
Powerpoint Format G4_Ostland_ Pharmacogenomics
Powerpoint Format G4_Penello_Pharmacogenomics
PDF Format G4_Wang_Pharmacogenomics
Powerpoint Format G4_Bajamonde_Pharmacogenomics

General Session 5: Multiplicity Issues in Analysis of Clinical Trial Data


The session will cover important topics that arise in the analysis of clinical trials with multiple endpoints, including the generalized Familywise Error Rate (gFWER) concept and branching testing strategies such as gate-keeping. Prof. Hsu of Ohio State, Dr. Huque of FDA and Dr. Alex Dmitrienko of Eli Lilly and Co. will give their views on various issues and possible resolutions to these issues from the perspective of academia, FDA and industry, respectively.

PDF Format G5_Dmitrienko_Multiplicity
Powerpoint Format
Powerpoint Format G5_Hsu_FDA_industry

Parallel Session 1 - Post Market Adverse Events: FDA Reporting Systems and Surveillance
Powerpoint Format
Parallel Session 01Intro, Powerpoint Format P01_Duggirala_PostMarketSurv, Powerpoint Format P01_Woo_PostMarketAdverseEvents,
Powerpoint Format
P01_Levine_ PostMarketAdverseEvents

Parallel Session 2: Development and Use of Patient-centered Questionnaires in Clinical Research

Powerpoint Format P02_Kammerman_PatQuestionnaires, Powerpoint Format P02_Sloan_PatQuestionnaires, Powerpoint Format P02_Rodenberg_PatQuest

Parallel Session 3: Bridging Studies - Concept, Issues, and Their Applications in International Simultaneous Drug Development
Powerpoint Format
P3_Chow_Bridging, Powerpoint Format P03_Takeuchi_Bridging

Parallel Session 4: Statistics in Preclinical Studies

Powerpoint Format P04_Menton_PreClinical, Powerpoint Format P04_Moon_Preclinical, P04_Crimin_PreClinical

Parallel Session 5: Topics in Drug Safety

Powerpoint Format P05_Bull_TopicsDrugSafety, Powerpoint Format P05_ Dong _TopicsDrugSafety, Powerpoint Format P05_Kulldorff_DrugSafety

Parallel Session 6: Vaccine Trials

Powerpoint Format ParallelSession06PanelDisc.ppt

Parallel Session 7: CDISC Initiatives: Impact on Electronic Data Submission
Powerpoint Format
P07_Christiansen_CDISC, PDF Format P07_Nessly_CDISC, Powerpoint Format P07_Wilson&Liu_CDISC

Parallel Session 8: Non-inferiority Studies
PDF Format
P08_Tsong_Noninferiority, Powerpoint Format P08_Dagostino_Noninferiority

Parallel Session 9: Contemporary Challenges in Statistical Evaluation of Diagnostic Products
Powerpoint Format
P09_ Baker_ DiagnosticProducts, Powerpoint Format P09_ Kondratovich_ DiagnosticProducts, Powerpoint Format P09_ShiDiagnosticProducts

Parallel Session 10: Integrated Statistical Computing Environments for Efficiency and Quality

Powerpoint Format P10_Schumack_Integrated Comp, PDF Format P10_Hopkins_IntegratedComp, Powerpoint Format P10_Wilson_Integrated StatComputing

Parallel Session 11: Flexible Design - Are We Ready Yet?

PDF Format P11_Posch_FlexibleDesigns, Powerpoint Format P11_ Dragalin _FlexibleDesign, Powerpoint Format P11_ Hung _FlexibleDesign,
Powerpoint Format P11_ Krams _FlexibleDesign

Parallel Session 12: Statistical Methods for Nonrandomized Trials and Subverted Randomized Trials
Powerpoint Format
P12_Berger_NonRandomized, Powerpoint Format P12_White_NonRandomizedStudies, Powerpoint Format P12_Yue_NonRandomizedStudies

Parallel Session 13: Combination Products
Powerpoint Format
P13_JHung_CombProducts, Powerpoint Format P13_Peterson_CombProducts, Powerpoint Format P13_ChanAhnn_CombProducts

Parallel Session 15: Statistical Issues in Medical Device Trials (Birds-of-a-Feather Session)

Powerpoint Format FDA-Ind_med_devtitle_05.ppt, Powerpoint Format drugsvsdevices1.ppt, Powerpoint Format Devicevsdrug2.ppt, Powerpoint Format CerkvenikNRStudies1.ppt,
Powerpoint Format FDA-Indmeddev05.ppt, Powerpoint Format FutureDESChallengesSept162005.ppt

Parallel Session 16

Powerpoint Format P16_Sethurman_QT, Powerpoint Format P16_Zhang_QT

2005 Workshop Registrant Directory
MS Word 2005RegistrantList.rtf