Re-Engineering Clinical Trials

Best Practices for Streamlining the Development Process

1st Edition - December 16, 2014
Editors: Peter Schueler, Brendan Buckley
Language: English
Hardback ISBN:
9 7 8 - 0 - 1 2 - 4 2 0 2 4 6 - 7
eBook ISBN:
9 7 8 - 0 - 1 2 - 8 0 0 7 9 0 - 7

The pharmaceutical industry is currently operating under a business model that is not sustainable for the future. Given the high costs associated with drug development, there is a… Read more

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The pharmaceutical industry is currently operating under a business model that is not sustainable for the future. Given the high costs associated with drug development, there is a vital need to reform this process in order to provide safe and effective drugs while still securing a profit. Re-Engineering Clinical Trials evaluates the trends and challenges associated with the current drug development process and presents solutions that integrate the use of modern communication technologies, innovations and novel enrichment designs. This book focuses on the need to simplify drug development and offers you well-established methodologies and best practices based on real-world experiences from expert authors across industry and academia. Written for all those involved in clinical research, development and clinical trial design, this book provides a unique and valuable resource for streamlining the process, containing costs and increasing drug safety and effectiveness.

List of Contributors
Introduction
Section 1. Why Does the Industry Need a Change?
- Chapter 1. Why Is the Pharmaceutical and Biotechnology Industry Struggling?
  - 1. Introduction
- Chapter 2. What Are Current Main Obstacles to Reach Drug Approval?
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 3. Japan: An Opportunity to Learn?
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Take Home Message
  - 6. Concluding Remarks
- Chapter 4. The “Clinical Trial App”
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Data Protection
  - 6. Take Home Message
Section 2. What Does Our Industry and What Do Others Do
- Chapter 5. Re-Engineering Clinical Trials: Best Practices for Streamlining the Development Process
  - 1. The Need
  - 2. The Solution
  - 3. Re-engineer Trials with New Technologies and Innovations
  - 4. New Technologies
  - 5. Existing and Unseen Challenges
  - 6. SWOT
  - 7. Applicable Regulations
  - 8. Take Home Message
- Chapter 6. How Can the Innovative Medicines Initiative Help to Make Medicines Development More Efficient?
  - 1. The Need
  - 2. Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 7. 2E: Experiences with Lean and Shop Floor Management in R&D in Other Non-Pharmaceutical Branches
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 8. Failure Mode and Effects Analysis (FMEA): Well-Known Methodologies, But Not in Our World
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Take Home Message
  - 5. Applicable Regulations
Section 3. Where to Start: The Protocol
- Chapter 9. No Patients—No Data: Patient Recruitment in the Twenty-first Century
  - 1. The Need
  - 2. The Solution
  - 3. SWOT: Strengths—Planning
  - 4. SWOT: Weaknesses—Site Performance
  - 5. SWOT: Opportunities—The Enrollment Plan
  - 6. SWOT: Threats—Sites and Regulations
  - 7. Applicable Regulations
  - 8. Take Home Message
- Chapter 10. The Impact of Bad Protocols
  - 1. A Critical Need to Address Rising Protocol Design Complexity
  - 2. Impact of Bad Design on Direct Procedure Costs
  - 3. Impact of Bad Design on Study Performance
  - 4. The Solution: Optimizing Study Designs
  - 5. The Necessity to Optimize Protocol Design
  - 6. Take Home Message
- Chapter 11. Data Mining for Better Protocols
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 12. It’s All in the Literature
  - 1. The Need
  - 2. The Solution
  - 3. Applicable Regulations
  - 4. Take Home Message
- Chapter 13. What Makes a Good Protocol Better?
  - 1. The Need
  - 2. The Solution
  - 3. Table/Checklist
  - 4. SWOT
  - 5. Applicable Regulations
  - 6. Take Home Message
- Chapter 14. The Clinical Trial Site
  - 1. The Need
  - 2. Enabling Staff at Clinical Trial Sites to Perform Well
  - 3. The Solution
  - 4. Take Home Message
  - 5. SWOT
  - 6. Applicable Regulations
Section 4. Alternative Study Designs
- Chapter 15. Do We Need New Endpoints in Clinical Trials: Surrogate and Biomarkers
  - 1. The Need
  - 2. The Solution
  - 3. Applicable Regulations
  - 4. Take Home Message
  - 5. SWOT
  - 6. Conclusion
- Chapter 16. On the Measurement of the Disease Status in Clinical Trials: Lessons from Multiple Sclerosis
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Take Home Message
  - 5. Applicable Regulations
- Chapter 17. Generating Evidence from Historical Data Using Robust Prognostic Matching: Experience from Multiple Sclerosis
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Take-Away Message
- Chapter 18. Studies with Fewer Patients Involved—The Adaptive Trial
  - 1. The Need
  - 2. The Solution
  - 3. SWOT Analysis
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 19. Connected Health in Clinical Trials: The Patient as Sub-Investigator
  - 1. Connected Health
  - 2. Application of Connected Health in Clinical Research
  - 3. Consumer Devices in Clinical Trials
  - 4. The Trial Subject as Sub-Investigator
  - 5. Take Home Message
  - 6. SWOT
  - 7. Applicable Regulatory Guidance
- Chapter 20. Studies Without Sites: The Virtual Trial
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Take Home Message
Section 5. From Data to Decisions
- Chapter 21. Re-Engineering Clinical Research with Data Standards
  - 1. The Need
  - 2. The Solution
  - 3. Applicable Regulations
  - 4. SWOT
  - 5. Take Home Message
- Chapter 22. Data Management 2.0
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 23. What Do the Sites Want? The Trial Master File
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Take Home Message
  - 5. Applicable Regulations
- Chapter 24. From Data to Information and Decision: ICONIK
  - 1. The Need
  - 2. The Solution: ICONIK Integrated Clinical Data Platform
  - 3. The Solution: Data, Information, and Knowledge: Quality as It Relates to Risk
  - 4. The Solution: Using the Information/Knowledge to Manage the Risk in Real Time
  - 5. The Solution: Risk Assessment
  - 6. The Solution: Integrated Risk Management Plan
  - 7. The Solution: Centralized Monitoring Activities
  - 8. The Solution: Adaptive Site Monitoring Activities
  - 9. SWOT
  - 10. Applicable Regulations
  - 11. Take Home Message
- Chapter 25. Knowledge Management: Looking after the Know-How
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 26. Taking Control of Ever-Increasing Volumes of Unstructured Data
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. COP
  - 5. A Little Warning
  - 6. Applicable Regulations
  - 7. Take Home Message
- Chapter 27. Share the Knowledge Based on Quality Data
  - 1. The Need
  - 2. The Solution
  - 3. Identifying Critical Data
  - 4. The Knowledge Structure in Practice
  - 5. The Data Team
  - 6. The Knowledge Team
  - 7. The Boiler Room
  - 8. The Action Team
  - 9. An Example from Real Life
  - 10. Organizational Psychology
  - 11. SWOT
  - 12. Take Home Message
Section 6. You Need Processes, Systems, and People
- Chapter 28. You Need Processes, Systems, and People—It's All about the People (and Their Competences)
  - 1. The Need
  - 2. The Solution
  - 3. SWOT Analysis
  - 4. Take Home Message
- Chapter 29. Managing the Change—You Need Processes, Systems, and People
  - 1. The Need
  - 2. SWOT
  - 3. The Solution
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 30. How Quality Performance Metrics Enable Successful Change
  - 1. The Need
  - 2. The Solution
  - 3. SWOT
  - 4. Applicable Regulations
  - 5. Take Home Message
- Chapter 31. Conclusion
  - 1. Take Home Message
Index

Peter Schueler

Peter Schüler is Senior Vice President, Global Medical & Safety Services, ICON Plc. Peter is board certified in neurology (1996; German Medical Board) and in Pharmaceutical Medicine (2001; Swiss Medical Association). In 1999 in obtained a Certificate in Business Administration by Henley Management College, UK. After his medical education he started is professional career in 1987 at the Medical Imaging Application Center of Siemens AG Medical Solutions. The same year he started at the Department of Experimental Neuropsychiatry of the Neurological University Hospital Erlangen, Germany. During the coming 8 years he was involved in basic and clinical CNS research. He held the position of Secretary General of the German section of the International League Against Epilepsy (ILAE; 1991-1995) and was awarded two scientific awards (Alfred-Hauptman epilepsy prize and ILAE Young Investigator Award). In 1995 he joined Pharmacia. During that period, he participated in the launch of two NCEs in the indications Parkinson’s and major depression. In October 2000 he moved into the CRO business, building a department for Medical Affairs and also taking over responsibilities for clinical operations in Germany, Austria and Switzerland. In 2007, Peter joined ICON, now overseeing over 360 people located in 11 major offices in Argentina, Brazil, Germany, India, Japan, Latvia, Mexico, Poland, Singapore, UK and US. He issued several publications on drug safety and authored an eBook about clinical trial design and conduct (Zenosis Ltd publisher) and continues to lecture Pharmaceutical Medicine at the Universities of Duisburg-Essen, Germany and Aveiro, Portugal.

Affiliations and expertise

MD, Senior VP Medical and Safety Services, ICON Clinical Research, Frankfurt, Germany and Auxiliary Professor for Pharmaceutical Medicine, Universities Duisburg-Essen, Germany and Aveiro, Portugal

Brendan Buckley

Brendan Buckley is Chief Medical Officer of ICON Plc. He was previously a co-founder of Firecrest Clinical. He is a Honorary Clinical Professor in the School of Medicine at University College Cork Ireland. A medical graduate of the National University of Ireland and a doctoral graduate in Biochemistry of Oxford University, he has over 30 years’ experience in clinical pharmacology research. Prior to joining ICON, he was Director of the European collaborative Centre for Clinical Trials in Rare Diseases at Cork. He was a member of the EMA Committee for Orphan Medicinal Products (COMP) from 2000-2003 and a member of the EMA Scientific Advisory Committee on Diabetes and Metabolism until 2011. He was a member of the Board of Directors of the Irish Medicines Board, the national competent authority for Ireland, 2004-2011 and chaired its statutory Advisory Committee for Human Medicines. He is chairman of the independent data and safety monitoring boards for a number of large clinical trials programmes with several sponsors. He is a member of the scientific advisory boards of a number of research charities and honorary Director of the Cork Cancer Research Centre. Professor Buckley is a Fellow of the Royal College of Physicians of Ireland, of the College’s Faculty of Pathology and a Fellow of the Faculty of Sports and Exercise Medicine.

Affiliations and expertise

MD DPhil FRCPI, Chief Medical Officer ICON plc, Dublin, Ireland and Honorary Clinical Professor, School of Medicine, University College Cork, Ireland

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