Platform Trial Designs in Drug Development: Umbrella Trials and Basket Trials, 1st Edition (Hardback) book cover

Platform Trial Designs in Drug Development

Umbrella Trials and Basket Trials, 1st Edition

Edited by Zoran Antonijevic, Robert A. Beckman

Chapman and Hall/CRC

304 pages

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Description

Platform trials test multiple therapies in one indication, one therapy for multiple indications, or both. These novel clinical trial designs can dramatically increase the cost-effectiveness of drug development, leading to life-altering medicines for people suffering from serious illnesses, possibly at lower cost. Currently, the cost of drug development is unsustainable. Furthermore, there are particular problems in rare diseases and small biomarker defined subsets in oncology, where the required sample sizes for traditional clinical trial designs may not be feasible.

The editors recruited the key innovators in this domain. The 20 articles discuss trial designs from perspectives as diverse as quantum computing, patient’s rights to information, and international health.

The book begins with an overview of platform trials from multiple perspectives. It then describes impacts of platform trials on the pharmaceutical industry’s key stakeholders: patients, regulators, and payers. Next it provides advanced statistical methods that address multiple aspects of platform trials, before concluding with a pharmaceutical executive’s perspective on platform trials. Except for the statistical methods section, only a basic qualitative knowledge of clinical trials is needed to appreciate the important concepts and novel ideas presented.

Table of Contents

Contents

Preface . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . ix

Acknowledgments . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . xiii

List of Contributors . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .xv

Part I Overview of Platform Clinical Trials . . . . . . . . . . 1

1 I-SPY2: Unlocking the Potential of the Platform Trial . . . . . . . . . . . . . .3

Laura Esserman, Nola Hylton, Smita Asare, Christina Yau,

Doug Yee, Angie Demichele, Jane Perlmutter, Fraser Symmans,

Laura van’t Veer, Jeff Matthews, Donald A. Berry, and Anna Barker

2 The Challenges with Multi-Arm Targeted Therapy Trials . . . . . . . . .23

Ryan J. Sullivan and Keith T. Flaherty

3 Basket Trials at the Confirmatory Stage . . . . . . . . . . . . . . . . . . . . . . . . .37

Robert A. Beckman and Cong Chen

4 Harnessing Real-World Data to Inform Platform

Trial Design . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .55

Daphne Guinn, Subha Madhavan, and Robert A. Beckman

5 Impact of Platform Trials on Pharmaceutical Frameworks . . . . . . . . .73

Zoran Antonijevic, Ed Mills, Jonas Häggström, and

Kristian Thorlund

Part II Stakeholders . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 83

6 Friends of Cancer Research Perspective on Platform Trials . . . . . . . .85

Jeffrey D. Allen, Madison Wempe, Ryan Hohman, and Ellen V. Sigal

7 Regulatory and Policy Aspects of Platform Trials . . . . . . . . . . . . . . . .97

Rasika Kalamegham, Ramzi Dagher, and Peter Honig

8 Multi-Arm, Multi-Drug Trials from a Reimbursement

Perspective . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .119

Anja Schiel and Olivier Collignon

9 Highly Efficient Clinical Trials: A Resource-Saving Solution

for Global Health . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .125

Edward J. Mills, Jonas Häggström, and Kristian Thorlund

10 Decision Analysis from the Perspectives of Single and

Multiple Stakeholders. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 141

Robert A. Beckman, Carl-Fredrik Burman, Cong Chen,

Sebastian Jobjörnsson, Franz König, Nigel Stallard, and

Martin Posch

11 Optimal Approach for Addressing Multiple Stakeholders’

Requirements in Drug Development . . . . . . . . . . . . . . . . . . . . . . . . . . 153

Zoran Antonijevic and Zhongshen Wang

Part III StatisticalMethodology. . . . . . . . . . . . . . . . . . . . 165

12 Primary Site Independent Clinical Trials in Oncology . . . . . . . . . . . 167

Richard M. Simon

13 Platform Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 181

Ben Saville and Scott Berry

14 Efficiencies of Platform Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 197

Satrajit Roychoudhury and Ohad Amit

15 Control of Type I Error for Confirmatory Basket Trials . . . . . . . . . . 211

Cong Chen and Robert A. Beckman

16 Benefit-Risk Assessment for Platform Trials. . . . . . . . . . . . . . . . . . . . 231

Chunlei Ke and Qi Jiang

17 Effect of Randomization Schemes in Umbrella Trials

When There Are Unknown Interactions between

Biomarkers. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 253

Janet J. Li, Shuai Sammy Yuan, and Robert A. Beckman

18 Combinatorial and Model-Based Methods in Structuring

and Optimizing Cluster Trials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 265

Valerii V. Fedorov and Sergei L. Leonov

Part IV Conclusions . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 287

19 An Executive’s View of Value of Platform Trials . . . . . . . . . . . . . . . . 289

David Reese and Phuong Khanh Morrow

Index. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .295

About the Editors

Zoran Antonijevic is Owner and Consultant of Z-Adaptive Design. He held executive positions in Pharmaceutical Companies and CROs and has designed more than 100 clinical trials in numerous therapeutic areas, many of which included adaptive designs. He has authored numerous papers and scientific presentations and edited Optimization of Pharmaceutical R&D Programs and Portfolios.

Robert Beckman is Professor of Oncology and of Biostatistics, Bioinformatics, and Biomathematics at the Lombardi Comprehensive Cancer Center and the Innovation Center of Biomedical Informatics, Georgetown University Medical Center. He has played significant leadership roles in developing new oncology clinical research groups at four pharmaceutical companies, while co-inventing several novel clinical and statistical development strategies. His versatile publication record comprises more than 250 contributions ranging from computational chemistry to clinical oncology.

The editors are past and current chairs of the Drug Information Association Adaptive Design Scientific Working Group, an international group of over 200 statisticians focused on novel clinical study designs and their role in bringing new medicines to patients.

About the Series

Chapman & Hall/CRC Biostatistics Series

Learn more…

Subject Categories

BISAC Subject Codes/Headings:
MAT029000
MATHEMATICS / Probability & Statistics / General
MED071000
MEDICAL / Pharmacology
MED090000
MEDICAL / Biostatistics