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17 October 2007, Wednesday

SESSION II: 
REGULATORY PERSPECTIVES AND LESSONS LEARNED FROM CONDUCTING STUDIES WITH ADAPTIVE TRIAL DESIGNS

8:30 Co-Chairperson’s Opening Remarks 
Judith A. Quinlan, Ph.D., Director Statistics, Biopharmaceuticals, GlaxoSmithKline

8:45 Regulatory Perspectives in Adaptive Trial Design
Dietlind Gardell, MD, Ph.D., Office of Clinical Trials, Therapeutic Products Directorate, Health Canada
In the current Canadian regulatory environment the emphasis on effective risk management across the therapeutic product life cycle encourages more individualized novel therapeutic approaches in clinical trials. Adaptive study designs are emerging as attractive options for study modifications in relation to accumulating data and interim data analyses. The potential for accelerated drug development and optimized resource allocation needs to be balanced with an increased focus on rigorous planning of flexible designs and on the regulatory standards for continued trial integrity, validity and patient safety based on the scientific evidence in the regulatory decision-making process.
9:15 Using Perturbed Unblinding for Sample Size Reassessment in Clinical Trials
Weichung Joe Shih, Ph.D., Professor and Chair of the Department of Biostatistics, UMDNJ-School of Public Health, and Director of the Biometrics Division, The Cancer Institute of New Jersey 
In this presentation I will review the current status of sample size re-estimation and point out the advantage of using un-blinded data and the need of masking the treatment group effect. The proposed ‘perturbed un-blinding’ method meets the above need and is simple to implement. In addition, I will also discuss the final test and address other concerns raised by the regulatory agency at The 2006 FDA and PhRMA conferences on the process issues regarding sample size re-estimation. The perturbed un-blinding method proposed in this paper should help statisticians in the pharmaceutical industry to better design clinical trials when facing uncertainty in the variability of the endpoint while addressing the regulatory concerns.
9:45 Adaptive Trial Design Infrastructure
Jerald S. Schindler, Dr.P.H., Vice President, Biostatistics and Research Decision Sciences, Late Stage Clinical Development Statistics, Merck Research Laboratories
In order to execute studies that utilize adaptive trial designs, important infrastructure must be put into place; this includes, but is not limited to, infrastructure to incorporate flexibility into the trial design and providing rapid access to ongoing trial information.

10:00 Exhibit Viewing, Networking and Refreshment Break

10:45 Implementing Adaptive Clinical Trials - Tools of the Trade 
Kathy Reinold, Project Manager, Adaptive Design Technology  
For nearly ten years, Tessella has worked behind-the-scenes to contribute the software for statistical models, randomization, and decision support on Bayesian-based adaptive clinical trials. From this experience, Tessella will present some innovative possibilities and insights into where things may go from here. 

11:00 Technology Spotlight
(Sponsorship Available. For more information, contact Arnie Wolfson at 781-972-5431 or awolfson@healthtech.com)

CASE STUDIES

Case Study

11:15 Operational Planning and Implementation with a Cardiovascular Trial Using an Adaptive Trial Design
Leslie P. Antonino, Clinical Protocol Manager, Cardiovascular and Metabolic Disease, Pfizer Inc.
There are many issues to be considered when planning for a trial that uses an adaptive design. This presentation will focus on; 1) operational considerations during study design, 2) planning and managing subject enrollment during a dose decision analysis and 3) lessons learned while conducting the trial. 
11:45 pm Luncheon Technology Workshop Adaptive EDC: Critical Steps for Managing Adaptive Trials
Khaled El Emam, Chief Scientist, TrialStat 
Electronic data capture software will play a pivotal role in enabling clinical researchers to use adaptive clinical trial designs. Success, however, largely depends on how adaptive EDC can be. Understanding a framework for evaluating EDC in an adaptive clinical trial context, therefore, becomes a critical first step.

Case Study

1:15 How to Build and Manage a Clinical Study Database with an Adaptive Design
Edie Owens, Program Data Manager, Pfizer Inc.

Case Study

1:45 Acute Migraine POC Using CRM - The Power of Team Work and a Can Do Attitude... Overcoming Logistical and Operational Hurdles
Judith Quinlan, Ph.D., Director, Statistics, Biopharmaceuticals, GlaxoSmithKline 
A great deal of theoretical debate exists around potential issues that may or may not exist when implementing adaptive designs. Concerns can range from possible introduction of biases, problems regarding confidentiality, maintaining the blind, etc. to concerns over the ease of implementation. Logistical challenges can often appear insurmountable or at least daunting obstacles. There is great potential to learn from early phase studies to increase our experience and understanding in regards to many of these issues. The case study to be presented is one such example. This was a phase II POC study in acute migraine, where patients needed to treat for migraine at early onset. In the face of existing processes and systems, the CRM method presented the team with many challenges. This presentation will examine the challenges and also the team’s solutions to implementation, and address how they handled the infamous, who saw what and when question!

2:15 Networking Refreshment Break

Case Study

2:45 Planning and Supplying Drug for Clinical Studies Using Flexible Designs
Micheline Marshall, R.Ph., MBA, Director of Clinical Pharmacy, Wyeth
This presentation will provide Clinical Supply Managers with an overview of adaptive trials as it relates to drug supply forecasting. A case study and lessons learned will provide real life experience with this new and exciting methodology. The focus will be on general requirements from a supply and technology perspective. It will highlight the challenge of managing global site inventories with an evolving randomization scheme. 

Case Study

3:15 Phase II Opioid Induced Constipation Dosing Study
Evan Tzanis, Associate Director, Medical Research, Wyeth
Opioids, are the mainstay of moderate to severe pain control for centuries. However opioids can cause a number of severe, and debilitating side effects as a result of the interaction with opioid receptors in the periphery. The most common of these peripheral side effects is constipation. Methylnaltrexone is a quaternary ammonium derivative of the opioid antagonist naltrexone. It is, therefore, possible to antagonize the undesirable peripheral side effects of opioids without affecting the desirable centrally mediated analgesic effects. Wyeth, in collaboration with our partner Progenics, is developing an oral formulation of methylnaltrexone for opioid-induced constipation. Key aspects of an adaptive design study that was conducted in patients with chronic non-malignant pain who also have opioid induced constipation will be discussed in this presentation.

Case Study

3:45 A Phase 2b Adaptive Dose-Finding Trial of a Novel Compound to Treat Acute Migraines
Xiaoyin Frank Fran, Ph.D., Senior Biometrician, Merck & Co., Inc.
Dose-finding is a critical step during drug development before the launch of the phase 3 program and where adaptive designs are commonly adopted. This case study of a novel migraine compound illustrates how we use a 2-stage adaptive design to go through multiple original doses, adjust sample sizes, and select the doses for later confirmatory trials. Theoretical and practical issues from the design, delivery, and decision-making perspective will be reviewed. The example demonstrates that with thoughtful planning upfront and appropriate monitoring during the trial, a well designed and executed adaptive trial can achieve objectives with great efficiency without weakening the results validity.

4:15 Panel Discussion with Speakers

4:45 Close of Putting Theory into Practice: Adaptive Clinical Trial Designs Conference

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