Michael J. Rosenberg

The Agile Approach to Adaptive Research

Optimizing Efficiency in Clinical Development
Herausgegeben von Ekins, Sean

• Gebundenes Buch

Produktbeschreibung

Apply adaptive research to improve results in drug development

The pharmaceutical industry today faces a deepening crisis: inefficiency in its core business, the development of new drugs. The Agile Approach to Adaptive Research offers a solution. It outlines how adaptive research, using already-available tools and techniques, can enable the industry to streamline clinical trials and reach decision points faster and more efficiently.

With a wealth of real-world cases and examples, author Michael Rosenberg gives readers a practical overview of drug development, the problems inherent in current practices, and the advantages of adaptive research technology and methods. He explains the concepts, principles, and specific techniques of adaptive research, and demonstrates why it is an essential evolutionary step toward improving drug research and development.

Chapters explore such subjects as:
The adaptive concept
Design and operational adaptations
Sample-size reestimation
Agile clinical development
Safety and dose finding
Statistics in adaptive research, including frequentist and Bayesian approaches
Data management technologies
The future of clinical development

By combining centuries-old intellectual foundations, recent technological advances, and modern management techniques, adaptive research preserves the integrity and validity of clinical research but dramatically improves efficiency.

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Produktdetails

• Wiley Series on Technologies for the Pharmaceutical Industry
• Verlag: Wiley & Sons
• 1. Auflage
• Seitenzahl: 304
• Erscheinungstermin: März 2011
• Englisch
• Abmessung: 236mm x 155mm x 20mm
• Gewicht: 544g
• ISBN-13: 9780470247518
• ISBN-10: 0470247517
• Artikelnr.: 28776858

Autorenporträt

MICHAEL J. ROSENBERG, MD, MPH, is President and Chief Executive Officer of Health Decisions, Inc. Involved in the design and execution of pharmaceutical development programs for more than twenty-five years, Dr. Rosenberg was selected as Entrepreneur of the Year by Ernst & Young (Health Sector, SE). His company's innovative applications of technology have been recognized by the CIO Enterprise Value Award and the Cisco Growing with Technology Award. Health Decisions has also been named three times to the Deloitte & Touche Technology Fast 500 and the Inc. 500. Dr. Rosenberg is the author of more than 150 scientific articles and serves on advisory groups in business, technology, and medicine.

Inhaltsangabe

Acknowledgements. Forward. 1 Opportunity for Efficiency. The Adaptive
Solution. An Industrial Success Story. Signs of Trouble Ahead. Converging
Challenges. The Struggle to Replace Lost Revenues. Clinical Research is the
Key. Behind the High Costs of Clinical Development. High Costs and
Increasing Prices. Growing Pressures Mandate Greater Efficiency. The High
Risk of Current Development Practices. Economic Consequences of Faster
Clinical Development. Thriving in a New Era. 2 Defining and Extending the
Adaptive Approach. The Adaptive Concept. Knowledge, Time and
Decision-Making. The Value of Early Knowledge. The Spectrum of Design and
Operational Adaptations. Maximizing the Adaptive Approach: Agile Clinical
Development. Measure Performance in Real-time. Metrics in Action. Right
Information to the Right Eyes at the Right Time. Make Timely Decisions.
Organize Work in Lean Processes. Rework in Clinical Studies. Backflow of
Patient Data. Match Technology with Tasks. Objections to Adaptive Methods.
Integrity and Validity. The Regulatory Environment. The Complexity of
Clinical Research. Conclusion. 3 Design Adaptations Part One: Finding the
Right Dose. Background. Types of Design Adaptations. Order of Discussion.
Dosing Nomenclature. etermining Maximum Safe Dose. Single Arm. Continual
Reassessment Method. Other Bayesian Dose-Finding Methods. Determining
Optimal Dose (Pruning). Multiple Arms. Improvements over Conventional
Approaches to Dose Finding. Dose Selection in Practice. Optimizing Dose
Selection. Minimizing Costs vs. Maximizing Information. Surrogate
Endpoints. Conclusion. 4 Design Adaptations Part Two: Additional Design
Changes. Sample-Size Reestimation. The Trouble with Planning Estimates. The
High Cost of "Underbuilt" Studies. The Benefits of Reestimation and
Rightsizing. Reestimation and Trial Stages. Rules to Restrict Reestimation.
Adjusting Sample Size for Nuisance Parameters. Seamless Designs: Combining
Multiple Phases. When to Consider Seamless Studies. Seamless Phase I/Phase
II Trials. Seamless Phase II/Phase III Trials. Planning Issues in Seamless
Trials. Phase I-II-III Designs. Adaptive Randomization. Response-Adaptive
Randomization. Other Forms of Adaptive Randomization. Other Types of Design
Adaptations. Noninferiority-to-Superiority Design. Adaptive Hypotheses and
Subpopulations. Treatment Switching. Conclusions. 5 Operational
Adaptations. Design and Operational Adaptations. The Nature and
Significance of Operational Adaptations. Implementing Operational
Adaptations. Enrollment and Other Site Issues. Data Quality. Monitoring.
Site Closeout and Database Lock. Supporting Functions for Efficient
Operations. The Bottom Line. 6 Agile Clinical Development. Benefits of
Agile Development. A Development Example. Program Planning. Safety Testing
(Phase I). Design. Planning. Operational Considerations. Putting the Plan
into Action. Benefits Realized. Transition to Dose-Finding Study. Dose
Finding (Phase II). Design. Planning. Operational Considerations. Putting
the Plan into Action. Benefits Realized. Transition to Confirmatory
Studies. Confirmatory Testing (Phase III). Design. Planning. Operational.
Putting the Plan into Action. Benefits Realized. Transition to Regulatory
Filings. Summing Up: The Power of Agile Development. 7 Planning Adaptive
Programs. Determining Design Adaptations and their Requirements.
Determining Operational Requirements to Support Design Adaptations.
Ensuring the Ability to Meet Operational Requirements. The Importance of
Programmatic Thinking. Looking Ahead. Advisory and Oversight Groups.
Optimizing the Planning Process. Regulatory Discussions. Contingency
Planning. Planning Tools and Techniques. Decision Trees. Simulation.
Uncertainties in Clinical Research. Simulation Tools. Simulation Step by
Step. The Limitations of Simulation. Conclusions. 8 Statistics and Decision
Making in Adaptive Research. The Frequentist Approach. What Frequentist
Results Say about the Results of a Clinical Study. Using Frequentist
Results to Make Decisions Based on Individual Trials. Other Issues with
Frequentist Methods. The Bayesian Approach. Prior Distributions. How
Bayesian Statistics Works. Bayesian Statistics in the Real World. Bayesian
Methods in Clinical Research. Comparing Bayesian and Frequentist Methods.\
Prior Distributions. The Pharma Context. Learning and Confirming. Ethical
and Operational Issues. Regulatory Considerations. Conclusions.
Frequentists, Bayesians, and Pragmatists. 9 The Agile Platform. Essential
Types of Data. Management Cycles in Clinical Studies. The Common Platform
for Design and Operational Adaptations. Data Capture. Data-Capture
Technologies. The First Generation. The Second Generation. The Third
Generation. Data Cleaning and Validation Technology. Data Analysis Tools.
Randomization. Site Management. Supply-Chain Management. Communications.
Conclusions. 10 The Future of Clinical Development. Is Restructuring an
Alternative to Improving Clinical Development? Greater Efficiency: Changes
that Everyone Can Applaud. Biotechs and Emerging Low-Cost Competitors.
Globalization, Offshoring, and Outsourcing. Managing More Complex Trials.
Individualized Medicine Demands Greater Efficiency. A More Important Role
for Postmarketing Studies. The Reward for Greater Efficiency. Financial
Implications of the Agile Approach. Financial Implications of the Agile
Approach for the Drug Industry. A Brighter Future for Clinical Development.
First Steps toward Agile Development. A Eight-Point Program for Embracing
the Adaptive Approach.

Rezensionen

"It is ideal for senior executives who want a strategic view of adaptive methods, managers looking for practical opportunities to significantly improve the efficiency of their operations, and statisticians interested in learning the broader ramifications of their arcane expertise." -- Journal of Clinical Research Best Practices, April 2010

"Rosenberg captures the current excitement surrounding adaptive clinical research, and provides a very readable introduction to the methodologies involved in implementing such research." -- Journal for Clinical Studies, March 2010

"This book certainly brings the relevant issues upfront." (Chemistry & Industry, June 2010)
"It is ideal for senior executives who want a strategic view of adaptive methods, managers looking for practical opportunities to significantly improve the efficiency of their operations, and statisticians interested in learning the broader ramifications of their arcane expertise." (Journal of Clinical Research Best Practices, April 2010)

"Rosenberg captures the current excitement surrounding adaptive clinical research, and provides a very readable introduction to the methodologies involved in implementing such research." (Journal for Clinical Studies, March 2010)