October 31 – November 1 | Beijing Xinjiang Plaza, ChinaThe first DIA “quantitative science forum” in 2013 was a successful event that provided a multi-functional forum within China to discuss quantitative science topics in pharmaceutical product development. DIA and China Clinical Trial Statistics (CCTS) Working Group will hold the second “quantitative science forum” in 2014. With an emerging need to evaluate drug both in pre-licensing and post licensing settings, there is no clear local guidance and consensus how to do it, scientifically and operationally. Therefore, the 2014 forum is set to share knowledge and discuss quantitative issues in safety, efficacy, risk-benefit, and potentially cost-effective utilization. We will also have dedicated session featuring consensus papers from the key statistics opinion leaders and experts from CCTS.

Unit A1618, Tower A, Top Electronics City, No.3 Hai Dian Avenue, Hai Dian District, Beijing 100080, China Tel. +86 400 996 1966 | Fax. +86 10 5704 2651 | www.diachina.org | DIA@diachina.org

2014 Quantitative Science Forum: Quantitative Drug Evaluation in and Beyond Clinical Trials

LEARNING objEctIvEs

X Highlight quantitative science’s critical role in medical product evaluation in clinical trials and in real world

X Discuss different ways to evaluate pharmaceutical products from multiple angles in in the regulatory environment

X Discuss the importance of guidance, standards, and scientific validity in medical product development

FEAtuREd topIcs

X Post-marketing safety (re-)evaluation: surveillance or study?

X Statistical issues in oncology drug development

X Statistical review and ADaM implementation

X Vaccine effectiveness in real world: experiences and challenges

X Quantitative evaluation on risk benefit

X Bayesian methods and applications in drug development

X Meta-analysis: theory, application and caveats

X Subgroup analysis

X Issues on developing biosimilar products

X Observation studies and health economical evaluation on medical products

X Selected topics with consensus by domestic experts

Program Co-Chairs

tARGEtEd AudIENcE

X Statisticians

X Clinicians

X Data Management Professionals

X Regulatory Scientists

X Quantitative and Clinical Scientists in Academia

X Quantitative and Clinical Scientists in Regulatory Agencies

William WaNg, PhDExecutive Director, Biostatistics and Research Decision Sciences (BARDS), Merck Research Laboratories / Merck& Co., Inc.

ChEN Feng, PhD, Professor Dean of School of Public Health, Nanjing Medical University, Chair of China Association of Biostatistics (CABS) , Chair of China Clinical Trial Statistics (CCTS) Working Group

co-spoNsoRs

pARtNERs

中国临床试验生物统计学组China Clinical Trail Statistics (CCTS)

International Chinese Statistical Association

International Society for Biopharmaceutical Statistics

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William WANG, PhDExecutive Director, Biostatistics and Research Decision Sciences (BARDS), Merck Research Laboratories / Merck& Co., Inc.

Roger QU, PhD Head of Clinical Statistics, Pfizer (China) R&D Center

CHEN Feng, PhD, Professor Dean of School of Public Health, Nanjing Medical University, Chair of China Association of Biostatistics (CABS), Chair of China Clinical Trial Statistics (CCTS) Working Group

SONG Yang, PhD Senior Principal Scientist, Biostatistics, Late Development Statistics, Biostatistics and Research Decision Sciences - Asia Pacific, Merck Research Laboratories / MSD R&D (China) Co., Ltd.

DAI Luyan, PhD Associate Director Biostatistics, Biometrics and Data Management at Boehringer-Ingelheim, Asia/META

YAO Chen, Professor Vice Director, Peking University Clinical Research Institute, Head of Department of Biostatistics, Peking University First Hospital

TANG Dejun, PhD Site Head, Biometrics & Statistical Sciences and Statistical Methodology, Novartis Pharma Co., Ltd.

Victor WU, PhDAssociate Director, Biostatistics and Programming, PPD

NING Yi, Sc.D, MDHead of Epidemiology GlaxoSmithKline (Shanghai)

HUANG Qin, PhDDeputy Director and Senior Reviewer, Office of Biostatistics, Center for Drug Evaluation (CDE), CFDA

CHEN Gang, PhD Senior Director, Head of China Biometrics Center, Johnson & Johnson

Bob YAN, PhDHead of Biostatistics and Programming, Sanofi China

PROGRAM COMMITTEE

XIA Jielai, Professor Professor, Department of Statistics, The Fourth Military Medical University

Nicole F. LI, PhD Asia Pacific Site Head of Biostatistics, Roche (China) Holding Co., Ltd.

WANG Ouhong, PhDDirector Biostatistics at Amgen China

WANG Yanping, PhD Director, Asia Pacific Statistical Science, Eli Lilly and Company

YI Bingming, PhD Head of Statistics, Epidemiology, and Data Management, GlaxoSmithKline R&D (Shanghai)

CHEN Jie, PhD Director, Global Biostatistics, Merck Serono (Beijing) Pharmaceutical R&D Co., Ltd.

WANG Ke, PhDSenior Research Consultant, Health Economics and Outcomes Research Eli Lilly and Company

ZHOU Wei, MD, PhDRegional Director, Epidemiology Asia Pacific, Merck Research Laboratories / MSD R&D (China) Co., Ltd.

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DAy 1 | FRIDAy, OCTOBER 31

8:30 – 12:00 OPENING PLENARY SESSION

Session Co-Chairs

Feng CHEN, PhD, Professor Dean, School of Public Health, Nanjing Medical University, Chair of China Association of Biostatistics (CABS), Chair of China Clinical Trial Statistics (CCTS) Working Group

William WANG, PhD Executive Director, Biostatistics and Research Decision Sciences (BARDS), Merck Research Laboratories / Merck& Co., Inc.

8:30 – 8:45 WELCOME REMARKS

DONG Haijun, PhDManaging Director, DIA China

William WANG, PhD Executive Director, Biostatistics and Research Decision Sciences (BARDS), Merck Research Laboratories / Merck& Co., Inc.

Feng CHEN, PhD, Professor Dean, School of Public Health, Nanjing Medical University, Chair of China Association of Biostatistics (CABS), Chair of China Clinical Trial Statistics (CCTS) Working Group

8:45 – 10:10 KEYNOTE SPEECH

Pharmaceutical R&D is an accumulative and iterative process, spanning from discovery to develop, from learning to confirming, from pre-marketing to post-marketing. This is particularly illustrated in the safety evaluation during the drug development process. This session will invite experts from regulatory agencies, academic institutions and industry to share their view and experiences.

A Regulatory Overview of Quantitative Drug Safety EvaluationAloka CHAKRAVARTY, PhDDirector, Division of Biometrics VII, Office of Biostatistics, Office of Translational Sciences, CDER, FDA

Post-authorisation Safety and Post-authorisation Efficacy EvaluationStella BLACKBURN, PhDVice President of Quintiles Innovation, Former EMA Risk Management Development and Scientific Lead

10:10 – 10:40 COFFEE BREAK

10:40 – 12:00 PLENARY SESSION (CONTINUED)

Statistical Methods for Signal Detection and its ApplicationXIA Jielai, ProfessorProfessor, Department of Statistics, The Fourth Military Medical University

Quantitative Knowledge Management in the Pharmaceutical Development LifecycleTom DOBBINS, PhDExecutive Director, Biostatistics and Research Decision Sciences, Merck & Co., Inc.

12:00 – 13:30 LUNCH

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15:00 – 15:30 COFFEE BREAK

DAy 1 | FRIDAy, OCTOBER 31

13:30 – 15:00

SESSION 1: POST-MARKETING SAFETY (RE-) EVALUATION: SURVEILLANCE OR STUDY?

Post-marketing surveillance and studies are important tools to monitor and evaluate the safety of medicines, either newly approved or have been on the market for a long time, in the real world setting, including in large populations and among special populations. There are different guidelines on post-marketing safety surveillance or studies in different countries, including the recent draft guidance from China Center for Drug Revaluation. Researchers around the world have been using different data sources including spontaneous report from routine pharmacovigilance, electronic medical record or claim databases, and primary data collection studies to evaluate the safety. In this session, experts from US, Japan, and China will discuss on the status and progress of post marketing surveillance or studies, share their experience and view of this field, discuss on the strength and limitations of different data sources, and explore the collaborations between different regions in monitoring and evaluating the safety of drugs or vaccines.

Session OrganizerBingming YI, PhD Head of Statistics, Epidemiology, and Data Management, GlaxoSmithKline R&D (Shanghai)

ZHOU Wei, MD, PhDRegional Director, Epidemiology Asia Pacific, Merck Research Laboratories / MSD R&D (China) Co., Ltd.

Post Authorization Safety Studies: Experience from US and EUNancy SANTANELLO, MDVice President, Merck Research Laboratories / Merck& Co., Inc.

Post-Marketing Surveillances in Japan – Lessons Learned and Best Practices David T. YOSHIIAsia Pacific Regional Director, Global Data Operations, PAREXEL International

Post-Marketing Safety Evaluation in ChinaHE Jia, ProfessorProfessor and Director, Department of Biostatistics, Second Military Hospital

13:30 – 15:00

SESSION 2: STATISTICAL ISSUES IN ONCOLOGY CLINICAL DEVELOPMENT

This session will focus on specific statistical challenges in oncology clinical development and discuss the methodologies used to address these issues.

Session OrganizerNicole F. LI, PhD Asia Pacific Site Head of Biostatistics Roche (China) Holding Co., Ltd.

Quantitative Solutions in Oncology Clinical TrialsWANG Xin, PhDSenior Manager Statistician of Eli Lilly and Company

Informative Censoring in Survival Analysis and Association between PFS and OSJulie CONG, PhDSenior Associate Director Statistics Boehringer Ingelheim Pharmaceuticals Inc.

Statistical Considerations in Early Stage Oncology Trial DesignSHEN Shuyi, PhD Associate Director Roche Co., Ltd.

Issues to be Explored in Oncology Clinical TrialsZHAO Naiqing, ProfessorProfessor of Biostatistics, Fudan University

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15:30 – 17:00

SESSION 4: VACCINE REAL-WORLD EVALUATION: EXPERIENCES AND CHALLENGES

Because of various limitations in vaccine evaluation at clinical trial phases, it is important to know vaccine’s post-marketing effectiveness and safety in real world. In this session, experts will introduce the methodology of vaccine real-world evaluation and share their experiences on study design and data analysis and interpretation.

Session OrganizersBob YAN, PhDHead of Biostatistics and Programming, Sanofi China

LIU Shusen Associate Principle Scientist, Epidemiology Merck Research Laboratories / MSD R&D (China) Co., Ltd.

Methodology of Vaccine Post-marketing Safety EvaluationWANG Xuanyi, ProfessorProfessor of Public Health Clinical Center Affiliated to Fudan University

The GARDASILTM Vaccine Impact on Population (VIP) Study in Nordic CountriesLIAW Kai-Li, PhDSenior Principle Scientist Merck Research Laboratories / Merck& Co., Inc.

Statistical Methods and Challenges in Vaccine Clinical TrialsCHEN Xuan, PhDManager of Biostatistics, Sanofi China

15:30 – 17:00

SESSION 3: STATISTICAL REVIEW AND ADAM IMPLEMENTATION

ADaM is part of CDISC for submission, which is content standard about analysis datasets. It will be a disaster to do statistical review with non-standardized analysis datasets. In this session, we are going to discuss the regulatory expectation to analysis datasets and demonstrate how to create and do statistical review with ADaM datasets.

Session OrganizersVictor WU, PhDAssociate Director, Biostatistics and Programming, PPD

SHI Changhong, PhD Associate Principle Scientist MSD R&D (China) Co., Ltd.

Regulatory Expectation to Analysis Datasets and Adam Overview Tineke CALLANTBiostatistical Coordinator, SGS Belgium NV – Life Science Services/Certified ADaM Instructor, CDISC

Statistical Review with ADaM Datasets BAO Wenjun, PhDChief Scientist, R&D Manager, JMP Life Sciences, SAS Institute Inc., USA

An Effective General Process to Build Adam DatasetsSHI Changhong, PhD Associate Principle Scientist MSD R&D (China) Co., Ltd.

CDISC ADaM TAs Standards Program OverviewLI HuadanManager, Biostatistical Programming, MSD R&D (China) Co., Ltd.

Panel discussion

UPCOMING OFFERINGS

UNITED STATES

NOV 6-7 WASHINGTON, DCPediatric Research Conference 2014Join key stakeholders within the global pediatric research community to discuss the challenges faced today and on the horizon for constructing and conducting global pediatric research programs.

SWITZERLAND

NOV 6-7 BASELISPE/DIA Workshop on Computer Systems Compliance “Maintain Data Integrity to Reduce Risk for the Patient”This 2-day conference will provide a forum for information, discussion on conceptual and practical questions and experience exchange through lectures, panel discussions, and interactive workshop sessions. It will focus on the work being conducted by two leading organizations within the life sciences sector who approach the subject from differing directions but with the same clear objective of ensuring data integrity to reduce the risk to the patient.

SPAIN

NOV 10-11 BARCELONAJoint DIA/AEMPS Statistics WorkshopThe meeting aims to discuss the latest developments in statistical methodology and decision making in exploratory development, confirmatory development, licensing decisions and management of benefits and risks post-licensing.

GERMANy

DEC 1-3 BERLIN15th Conference on European ElectronicDocument Management (eDM) and ExhibitionCome and learn about the benefits and constraints associated with the various topic-related processes and contribute to answering the question whether standardization is a blessing or a curse.

DEC 2-3 BERLINBiosimilars ConferenceThe conference will give an overview of the current regulatory, scientific and market perspectives for biosimilars, including discussion on a number of Hot Topics in the field.

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DAy 2 I SATURDAy, NOVEMBER 1

8:30 – 10:00

SESSION 6: BENEFIT RISK EVALUATION IN PHARMACEUTICAL DEVELOPMENT

Ensuring the safety and effectiveness of pharmaceutical products is an increasingly complicated task, requiring a balanced consideration of a multitude of factors. Over the past several years, structured approaches to benefit-risk assessment in drug development and regulatory decision-making have been developed. This session will present various benefit-risk evaluation approaches used by different regulatory bodies and their implementation in practice.

Session OrganizersWilliam WANG, PhD Executive Director, Biostatistics andResearch Decision Sciences (BARDS), Merck Research Laboratories / Merck& Co., Inc.

JIANG Qi, PhDExecutive Director, Global Biostatistical Science, Amgen Inc.

Considerations for Benefit:Risk Assessment in Pharmaceutical Drug DevelopmentJIANG Qi, PhDExecutive Director, Global Biostatistical Science, Amgen Inc.

Overview of EMA Benefit Risk FrameworkStella BLACKBURN, PhDVice President of Quintiles Innovation, Former EMA Risk Management Development and Scientific Lead

10:10 – 10:30 COFFEE BREAK

10:30 – 12:00

SESSION 8: META-ANALYSIS: THEORY, APPLICATION AND CAVEATS

Meta-analysis has been widely used to analyze and interpret data for medical products. This session will provide an overview of meta-analysis methods, with examples and discussion on their merits, limitations and pitfalls, and their specific applications in safety evaluation with a regulatory perspective. This session will also cover an introduction of network meta-analysis, which is an essential tool in the increasingly important area of health technology assessment.

Session OrganizerSONG Yang, PhD Senior Principal Scientist, Biostatistics, Late Development Statistics, Biostatistics and Research Decision Sciences - Asia Pacific, MSD R&D (China) Co., Ltd.

Meta-analysis and its ProblemsNING Yi, Sc.D, MDHead of Epidemiology GlaxoSmithKline

Meta-analysis in Safety Evaluation: Regulatory IssuesAloka CHAKRAVARTY, PhDDirector, Division of Biometrics VII, Office of Biostatistics, Office of Translational Sciences, CDER, FDA

Introduction to Network Meta-analysis and its ApplicationCHENG Gang, PhDSenior Biostatistician, Biostatistics/Biometrics and Data Management, Asia/META, Boehringer Ingelheim (Shanghai) Pharmaceuticals

12:00 – 13:30 LUNCH

8:30 – 10:00

SESSION 5: BAYESIAN METHODS AND APPLICATIONS IN DRUG DEVELOPMENT

Bayesian analysis has been widely applied to the drug development. Recently more and more clinical trial related data analyses have utilized Bayesian methods to gain scientific conclusions in more efficient ways. This session will present some applications related to Bayesian methods regarding some challenges in clinical trials regarding subgroup and non-inferiority trials.

Session OrganizerTANG Dejun, PhD Site Head, Biometrics & Statistical Sciences and Statistical Methodology, Novartis Pharma Co., Ltd.

Bayesian Meta-analysis Using Hierarchical Models YIN Guosheng, PhDProfessor of Department of Statistics and Actuarial Science the University of Hong Kong

Bayesian Decision Making in the Drug Development ProcessCHEN Fei, PhDAssociate Director, Johnson & Johnson, USA

Bayesian Hierarchical Models for Safety AnalysisXU Cong, PhDSenior Biometrician Integrated Information Sciences, Novartis China

10:30 – 12:00

SESSION 7: SUBGROUP ANALYSIS IN CLINICAL TRIALS

Subgroup analysis is commonly performed and reported in clinical research. This session will discuss key considerations in subgroup analysis, including statistical methods as well as regulatory requirements.

Session OrganizerWANG Yanping, PhD Director, Asia Pacific Statistical Science, Eli Lilly and Company

Statistical Considerations for Subgroup AnalysisFeng CHEN, PhD, Professor Dean, School of Public Health, Nanjing Medical University, Chair of China Association of Biostatistics (CABS), Chair of China Clinical Trial Statistics (CCTS) Working Group

Statistical Subgroup Analysis in Clinical TrialsIlya LIPKOVICH, PhDSenior Director Center for Statistics in Drug Development, Quintiles Innovation

Panel DiscussionXIA Jielai, ProfessorProfessor, Department of Statistics, The Fourth Military Medical UniversityTANG Dejun, PhDSite Head, Biometrics & Statistical Sciences and Statistical Methodology, Novartis Pharma Co., Ltd.Nicole F. LI, PhDAsia Pacific Site Head of Biostatistics Roche (China) Holding Co., Ltd.

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13:30 – 15:00

SESSION 10: ISSUES ON DEVELOPING BIOSIMILAR PRODUCTS

China is currently riding on the wave of global emergence of biosimilar products. A biosimilar is a biological product that is similar to an already approved biological product, notwithstanding minor differences in clinically inactive components. The clinical study designs will be critical to provide the evidence that there are no clinically meaningful differences between the biosimilar and the approved biological product in terms of the safety, purity, and potency. The session will cover the topics including phase I pharmacokinetics bio-equivalence design and design considerations in phase III for the equivalence testing, study population shift and other practical considerations in developing biosimilars in China market.

Session OrganizersDAI Luyan, PhD Associate Director Biostatistics, Biometrics and Data Management at Boehringer-Ingelheim, Asia/META

Bruce XUE, PhDSenior Biostatistical Manager, Head of Biostatistics – Development, Janssen R&D, J&J, China

Consideration of Study Population Shift in Phase III Study of Biosimilar Product DevelopmentLIAO Shanmei, PhDSenior Manager, Pfizer (China) R&D Center

An Overview of Testing For Equivalence of Clinical Efficacy and Practical Considerations in Biosimilar Development in ChinaZHANG Tao, PhDSenior Biostatistician, Boehringer-Ingelheim, China

Bioequivalence Trials in Drug Development – Study Design and Data Analysis ConsiderationsJames JIAO, PhDAssociate Director, Johnson & Johnson, USA

15:00 – 15:30 COFFEE BREAK

15:30 – 17:00

SESSION 11: SELECTED TOPICS WITH CONSENSUS BY DOMESTIC EXPERTS

This session will focus on the introduction of Chinese Clinical Trials Statistics (CCTS) Working Group, including its mission, member and achievement. Besides a presentation given by CCTS chair for an overall introduction of the group, another two speakers will give introduction for two recently published CCTS consensus.

Session OrganizersYAO Chen, ProfessorVice Director, Peking University Clinical Research Institute, Head of Department of Biostatistics, Peking University First Hospital

Tony GUO, PhD Director, Biostatistics, MSD R&D (China) Co., Ltd.

CCTS: Mission, Vision and Potential ImpactFeng CHEN, PhD, ProfessorDean, School of Public Health, Nanjing Medical University, Chair of China Association of Biostatistics (CABS), Chair of China Clinical Trial Statistics (CCTS) Working Group

Introduction to CCTS Consensus of Non-Inferiority TrialsXIA Jielai, ProfessorProfessor, Department of Statistics, The Fourth Military Medical University

Introduction to CCTS Consensus of MultiplicityWANG Tong, ProfessorProfessor, School of Public Health Shanxi Medical University

DAy 2 I SATURDAy, NOVEMBER 1

13:30 – 15:00

SESSION 9: HEALTH ECONOMICS AND OBSERVATIONAL STUDY

Real world evidence from health economics and outcomes research (HEOR) and observational studies is crucial to drug development and pharmacoeconomic evaluations. Findings from a well-designed HEOR or observational study can answer questions a single randomized controlled trial cannot answer. Availability of real world data broadens the study population and the scope of health outcome. Related statistical approaches are also constantly evolving.

Session OrganizersWANG Ke, PhDSenior Research Consultant, Health Economics and Outcomes Research Eli Lilly’ and Company

NING Yi, Sc.D, MDHead of Epidemiology GlaxoSmithKline (Shanghai)

Indirect ComparisonHAN Simeng,PhDChina Director Analysis Group Inc.

The Front Sheet of Medical Record and ApplicationMA Xiemin, ProfessorAssociate Director of Hospital Management Research Center Peking University Application of observational study in drug developmentGUO Na, PhD Epidemiologist Roche (China) Holding Co., Ltd.