Clinical Trial Design

Missing Data in

Clinical Trials

Discover the most effective strategies to identify, reduce

and avoid the pitfalls of incomplete data in your clinical trials.

Missing DaTa MiTigaTiOn

exlevents.com/missingdata

March 16-17, 2015 • Loews Philadelphia Hotel • Philadelphia, PA

•

Missing data can seriously

compromise the outcome of a

clinical trial, possibly affecting

regulatory compliance

•

Recent studies have shown that

data reanalysis changes 35% of

trial conclusions

•

More robust clinical trials can

significantly improve your

drug’s safety and efficacy

Forum

Celestine Hicks, Associate Director, Clinical Data Management, asTRaZeneCa

Dr. Roderick Little, Richard D. Remington Distinguished University Professor of Biostatistics, UNIVERSITY OF MICHIGAN, SCHOOL OF PUBLIC HEALTH

Dr. Craig Mallinckrodt, Research Fellow, Strategy and Decision Sciences, eLi LiLLy

Maria Minasian, Principle Sample Strategy Analyst, BRISTOL-MYERS SQUIBB

Dr. Lori Post, Center for Medical Informatics,

YALE UNIVERSITY SCHOOL OF MEdICINE

Bill Potter, Senior Advisor to the Director, NIH, NATIONAL INSTITUTE OF MENTAL HEALTH

Patrick Zbyszweski, Senior Director, Clinical Data Management, ONCONOVA THERAPEUTICS

Terry Katz, Director, Global Management and Statistics,

MeRCK

eLi LiLLy anD

COMPany

Co–Chaired by:

FeaTuReD sPeaKeRs:

Overcome critical challenges to

ensure a successful clinical trial:

3

Minimize the impact of missing

data through better clinical

trial design

3

Monitor and reduce incomplete data

points during the conduct of your

study

3

Take proactive steps to

reduce

patient dropouts and optimize

protocols

3

Develop an effective pre-enrollment

plan for

collecting and cleaning

interim data

Learning Objectives:

3

Identify the impact that missing data

points have on study conclusions

and inferences

3

Learn how your trial results are

viewed within the current

regulatory climate

3

Employ trial-specific analytical

techniques to handle missing data

3

Explore best practices for increasing

participant retention and

compliance

statistical analys

is

Clin

Dear Colleague,

As you know, a successful clinical trial greatly depends on high-quality, accurate data. However, incomplete data points do occur. This can significantly compromise all areas of the research, from key inferences made to regulatory compliance. Regulatory bodies have provided relatively little guidance on how to deal with the setbacks caused by this problem, and many companies compensate by means of a single set of statistical analysis techniques for every study. This is usually not a comprehensive solution, and needs to be utilized in conjunction with other methods to ensure that a trial’s datasets are as robust as possible. The Missing Data in Clinical Trials Forum was designed in response to direct feedback from your colleagues to help you implement the most effective methods to mitigate the dangers of missing data in your company’s trials.

Over these two days, our key areas of focus will be:

• Identifying the impact of missing data on study results

• Minimizing the impact of missing data through better clinical trial design

• Employing the most appropriate analytical techniques specific to your study’s design • Learning best practices for increasing patient participation and compliance

This forum is the first of its kind in the clinical trial conference landscape. It is the only event to help you identify and address weak points during all stages of a study, including trial design, operations, monitoring and analysis. Our faculty represents an incredible spectrum of expertise. You are sure to leave this conference with innovative, readily employable techniques to vastly improve the quality of your trials.

We look forward to welcoming you to Philadelphia this March! Sincerely,

Stephen Grosso, M.S.

Conference Production Director

Missing Data in

Clinical Trials

Forum

This conference is designed

for professionals in the

pharmaceutical, medical device or

biotechnology industries involved

with the following:

• Clinical Operations

• Data/Document Management • Clinical Quality Management • Compliance

• Clinical Statistics • Clinical Trial Monitoring • Patient Recruitment • Clinical Data Science • Medical Research • Project Management • Data Analysis

• Strategic R&D Innovations

This event is also of interest to:

• Contract Research Organizations • Clinical Trial Management Software

Providers

• Data Concurrency Service Providers

Venue:

Loews Philadelphia Hotel

1200 Market Street Philadelphia, PA 19107

If you require overnight accommodations, please contact the hotel to book your room. ExL Pharma has reserved a block of rooms at a discounted rate for conference participants. We encourage participants to make reservations by February 23, 2015. To make reservations guests can call 888-575-6397 and request the negotiated rate for “ExL’s March Meetings.” Please book your room early as rooms available at this rate are limited.

Sponsorship and Exhibiting

Opportunities

Do you want to spread the word about your organization’s solutions and services to potential clients who attend this event? Take advantage of the opportunity to exhibit, present an educational session, host a networking event or distribute promotional items to attendees. ExL works closely with you to customize a package that suits all of your needs. To learn more about these opportunities, contact Andrew Ferguson, Business Development Manager, by calling 917-258-5150 or emailing aferguson@exlevents.com .

Monday, March 16, 2015

Day 1

8:00 Registration and Continental Breakfast

9:00 Conference Co-Chairpersons’ Opening Remarks Terry Katz, Director, Global Data Management and Statistics, MeRCK

Carol Robertson-Plouch, LRL Strategic R&D Innovations, eLi LiLLy anD COMPany

9:15 Overview of the Current state of Missing Data Mitigation and its impact on Life science Companies

• Review innovations in mitigation techniques

• Learn how incomplete data affects conclusions and inferences of trials

• Discover how statisticians can partner with clinical teams to achieve better analyses

Bill Potter, Senior Advisor to the Director, NIH, NATIONAL INSTITUTE OF MENTAL HEALTH

10:00 Develop an Operational strategy to ensure effective Trial Practices and Quality decision-Making

• Hear best practices regarding project management, budgeting, resources and developing SOPs

• Ensure that study teams meet all data quality deliverables and metrics

• Implement effective documentation and communication for improved outcomes

Colin Scott, Clinical Project Leader, nOvaRTis COnsuMeR HEALTH

10:45 Networking Break

11:15 Optimize Trial Design and Conduct to Minimize the Occurrence of Missing Data

• Explore study site selection criteria for a better trial • Discuss informed consent and distinguishing the difference

between withdrawing from the treatment and withdrawing from the study

• Utilize simulations to identify the probability of success Carol Robertson-Plouch, LRL Strategic R&D Innovations, eLi LiLLy anD COMPany

12:00 Best Practices for Efficient data Entry/Management to Mitigate the effects of Missing Data

• Learn how to tailor treatment selection appropriately for your trial

• Manage the challenges associated with eSource datasets • Learn how to adjust inclusion/exclusion criteria

Deborah Rittenhouse, Senior Manager, Clinical Data Management, CSL BEHRING

12:45 Luncheon

1:45 use emerging Technology to improve Clinical sample Tracking

• Identify the need to increase collaboration and transparency between all parties involved

• Learn how to streamline processes and take a proactive approach to clinical sample tracking

• Leverage technology to optimize clinical sample tracking Maria Minasian, Principle Sample Strategy Analyst, BRISTOL-MYERS SQUIBB

2:15 Best Practices for Improving the Quality of Safety data Reporting in your study

• Reduce dropout rates by improving awareness and training for study site personnel

• Optimize adverse event reporting and reduce follow-up questions for site and sponsor

• Improve reporting of safety events for more comprehensive datasets

Gerson Peltz, Senior Medical Director, Head of Clinical Trial Safety, BIOGEN IdEC

3:00 Networking Break

3:30 Proven insights to increase Patient Recruitment and Retention

• Identify populations most likely to remain in the study • Re-evaluate study endpoints so they are disease-specific • Learn how to effectively overhaul data collection methods • Discover how budget allocations for patients can increase data

completion

Dr. Lori Post, Associate Professor, Center for Medical Informatics, YALE UNIVERSITY SCHOOL OF MEdICINE

4:15 Monitor and Reduce Missing Data During the Conduct of your Clinical Trial

• Utilize remote EDC monitoring methods

• Harness information extracted from electronic health records • Learn how to take a proactive approach to minimize or reduce

data being excluded from analyses at the point of collection Terry Katz, Director, Global Data Management and Statistics, MeRCK

5:00 Conference Co-Chairpersons’ Day One Remarks Terry Katz, Director, Global Data Management and Statistics, MeRCK

Carol Robertson-Plouch, LRL Strategic R&D Innovations, eLi LiLLy anD COMPany

tuesday, March 17, 2015

Day 2

9:00 Conference Co-Chairpersons’ Recap of Day One Terry Katz, Director, Global Data Management and Statistics, MeRCK

Carol Robertson-Plouch, LRL Strategic R&D Innovations, eLi LiLLy anD COMPany

9:15 Overview of the Current Regulatory Landscape and anticipating the Future

• Learn how incomplete data affects regulatory approval or denial • Compile and present trial documentation to increase

compliance

• Discuss clinical trial design from a regulatory viewpoint Celestine Hicks, Associate Director, Clinical Data Management, asTRaZeneCa

10:00 Develop an effective Pre-enrollment Plan for Collecting and Cleaning interim Data

• Create a cross-functional quality-by-design approach to ensure that all critical data is prioritized and accounted for

• Discuss tools that can be used to aid in the process and highlight missing or unclean data

• Identify potential pitfalls or missteps due to CTMS, CRF or matrix designs

Patrick Zbyszewski, Senior Director, Clinical Data Management, ONCONOVA THERAPEUTICS

10:45 Networking Break

11:15 Learn How to Utilize the Best Imputation Method for your application

• Explore techniques, including last observation carried forward (LCOF), baseline observation carried forward (BCOF), responder analysis and survival analysis, in order to accurately process your trial’s datasets

• Learn how to utilize generalized estimating equations

• Discover how to use all available data to increase trial accuracy Raj Bandaru, Informatics Science Director, asTRaZeneCa 12:00 Characterize Missing Data Mechanisms to Optimize

Primary analyses

• Learn how to characterize missing data points as either missing completely at random (MCAR), missing at random (MAR) or missing not at random (MNAR)”

• Identify the patterns of patient dropouts

• Discover acceptable and unacceptable analytic methods for each analysis type

Dr. Craig Mallinckrodt, Research Fellow, Strategy and Decision Sciences, eLi LiLLy anD COMPany

12:45 Luncheon

1:45 Modern Developments in sensitivity analyses

• Hear an overview of the different types of sensitivity analyses and learn how to use the most appropriate model for examining the robustness of your trial’s conclusions

• Learn how to report the findings of a sensitivity analysis to add credibility to your trials

• Discuss emerging solutions that can enhance analyses Dr. Roderick Little, Richard D. Remington Distinguished University Professor of Biostatistics, univeRsiTy OF MICHIGAN, SCHOOL OF PUBLIC HEALTH

2:30 Missing Data Mitigation Techniques from a CRO’s Perspective

• Hear examples of how multiple imputation can minimize data loss

• Learn about control-based multiple imputation for primary and sensitivity analyses

• Use mixed models and learn how to view the generated results 3:15 Conference Co-Chairpersons’ Closing Remarks

Terry Katz, Director, Global Data Management and Statistics, MeRCK

Carol Robertson-Plouch, LRL Strategic R&D Innovations, eLi LiLLy anD COMPany

3:30 Conclusion of Conference CASE S t UD y

“

”

“

_”

Great overview and sessions on key

new areas for recruitment and risk

management!

- Executive Director, Clinical Operations, Acorda Therapeutics

Surpassed my expectations –

Excellent conference!

- Associate Director, Global Inspection Lead, Merck & Company

Very useful to get together with others

working in the same area and to see

the vendors/services available.

- Sr. Clinical Research Associate, Momenta Pharmaceuticals

“

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www.missingurl.com

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