Pathways to Efficient Drug Development - Advances in Modeling and Simulation Outcomes to Fuel Pipeline Productivity

Pathways to Efficient Drug Development - Advances in Modeling and Simulation Outcomes to Fuel Pipeline Productivity

Summary

GBI Research, the leading business intelligence provider, has released its latest research report, “Pathways to Efficient Drug Development - Advances in Modeling and Simulation Outcomes to Fuel Pipeline Productivity”. The report examines the reasons why the pharmaceutical industry is looking for improvements in efficiency whilst acknowledging that pharmaceutical R&D remains a long and risky process. It looks in detail at precompetitive research and evaluates how the industry is pulling together to research solutions to problems that are common to all companies. The report investigates innovation in the clinical drug development arena, documenting modeling and simulation based approaches to improving efficiency, as well as novel clinical trial designs. Lastly, the report examines innovation in business models within the industry that aim to help the industry to achieve its mantra of “doing more with less”, which will be critical for its future success.

The report is built using information from primary and secondary research including interviews with experts in the field.

GBI Research’s analysis shows that collaboration and open innovation will play increasingly important roles in the future by enabling research that would not be possible for companies to undertake individually. Many examples exist, driven in large part by the FDA’s Critical Path Initiative and the European Innovative Medicines Initiative, and experiences gained by early consortia will help facilitate the logistical challenges of setting up new collaborations. Within companies, innovations including the increased use of modeling and simulation throughout the drug development process, adaptive clinical trials and exploratory clinical trials have all been studied for some years, suggesting that innovation is hard, but important. Innovation is also occurring in the business models applied within individual companies to enable them to achieve “more with less”. Through adoption of new scientific approaches and business models, companies are hoping not only to refuel their pipelines but to regain the confidence of investors and the public in their ability to deliver meaningful treatments for patients at the same time as generating profits in the coming years.

Scope

- Detailed analysis of the reasons for the industry to be looking closely at improving efficiency
- Definition of precompetitive collaboration, analysis of areas in which precompetitive collaboration is occurring, and discussion of the expansion of this space in the future
- Explorations of the key challenges facing consortia and the factors that make them successful
- Case studies of key innovations in drug development including model-based drug development, adaptive clinical trials and exploratory clinical trials
- Detailed insights into innovation in business models, including virtual networks, open innovation and extensive academic collaborations

Reasons to buy

- Identify key projects in the precompetitive space
- Learn the most important factors for successful precompetitive collaborations
- Develop strategies and priorities for participating in precompetitive collaborations
- Understand current thinking on innovative areas of drug development, including model-based drug development, adaptive clinical trials and exploratory clinical trials, from the viewpoints of companies and regulators
- Explore the business models and partnerships of the largest pharmaceutical companies to support new drug development strategies

1 Table of Contents
1 Table of Contents 5
1.1 List of Tables 7
1.2 List of Figures 8
2 State of the Industry 9
2.1 Introduction 9
2.1.1 The Rising Costs of Drug Development 10
2.1.2 Drug Attrition 12
2.1.3 Patent Expiries 14
2.1.4 Regulatory Hurdles 15
2.1.5 The Fourth Hurdle: Reimbursement 16
2.2 Innovation in the Drug Development Paradigm 16
2.2.1 The Critical Path Initiative 16
2.2.2 The Innovative Medicines Initiative 18
2.3 Improving Drug Development 19
2.4 2012 and Beyond 20
3 Collaboration in the Precompetitive Space 21
3.1 Defining Precompetitive Research 21
3.2 Building Successful Consortia 23
3.2.1 Choosing the Research Topic 23
3.2.2 The Set-Up Phase 23
3.2.3 Project Management 24
3.2.4 Measuring Success 25
3.2.5 Case Study of a Successful Private Public Partnership: The Alzheimer’s Disease Neuroimaging Initiative 26
3.3 Qualification of Biomarkers of Efficacy or Safety 26
3.3.1 Case Study: Biomarkers of Kidney Injury 29
3.3.2 Case Study: The Biomarkers Consortium 31
3.4 Open Innovation Platforms 32
3.4.1 Case Study: OpenPHACTS 33
3.4.2 Case Study: Sage Bionetworks 34
3.5 Data Standards 35
3.5.1 BioSharing: Standard Cooperating Procedures 35
3.5.2 Clinical Data Standards 36
3.5.3 Data Standards and the FDA 36
3.6 Conclusions 37
4 Improving Drug Development Efficiency 38
4.1 Modeling and Simulation 38
4.1.1 Modeling and Simulation: A View from the Regulators 39
4.1.2 Model Qualification 40
4.1.3 Modeling and Simulation Expertise and Consultancy 40
4.2 Innovative Approaches to Clinical Trials 41
4.2.1 Adaptive Clinical Trials 42
4.2.2 Case Study: The I-SPY 2 Trial 44
4.2.3 Exploratory Clinical Trials 45
4.3 Engaging Stakeholders 46
4.3.1 Patients 46
4.3.2 Regulators 47
4.3.3 Payers 47
4.4 Conclusions 47
5 Business Models 48
5.1 Introduction 48
5.2 R&D Reorganization 48
5.2.1 Mimicking the Biotech Environment 48
5.2.2 The Fully Integrated Pharmaceutical Network Model 49
5.3 Open Innovation 50
5.3.1 Case Study: An Open Innovation Incubator 51
5.3.2 Case Study: Open Innovation Drug Discovery at Eli Lilly 52
5.4 Funding for External Innovation 53
5.4.1 Collaborative Commercialization 54
5.5 Academic Partnerships and Translational Medicine 55
5.5.1 Translational Science in the US 57
5.5.2 Case study: Medical Research Council/AstraZeneca 58
5.6 Conclusions 59
6 Appendix 60
6.1 Abbreviations 60
6.2 Methodology 61
6.2.1 Primary Research 61
6.2.2 Secondary Research 62
6.3 References 62
6.4 Contact Us 66
6.5 Disclaimer 66

1.1 List of Tables
Table 1: Pathways to Efficient Drug Development, Clinical and FDA Approval Times across Therapeutic Classes (2005-2009) 11
Table 2: Pathways to Efficient Drug Development, Transition Probability at Each Stage of Clinical Drug Development 12
Table 3: Pathways to Efficient Drug Development, Overall FDA Approval Success Rate for New Chemical Entities by Therapeutic Area 12
Table 4: Pathways to Efficient Drug Development, Loss of US Sales Revenues Due to Patent Expiries ($m; 2010–2013) 14
Table 5: Pathways to Efficient Drug Development, Drugs Withdrawn from the Market in the US (1992–2010) 15
Table 6: Pathways to Efficient Drug Development, Ongoing Projects of the Critical Path Institute 17
Table 7: Pathways to Efficient Drug Development, Proposed Network for Evaluating PPPs in the Pharmaceutical Sciences 25
Table 8: Pathways to Efficient Drug Development, Biomarkers qualified by the FDA for use in drug development 27
Table 9: Pathways to Efficient Drug Development, Biomarkers qualified by the EMA for use in drug development 27
Table 10: Pathways to Efficient Drug Development, Ongoing Public-Private Partnerships for Biomarker Identification and Qualification 28
Table 11: Pathways to Efficient Drug Development, Pharmaceutical Companies Involved in the Predictive Safety Testing Consortium, IMI SAFE-T Project and the Biomarkers Consortium Kidney Project 30
Table 12: Pathways to Efficient Drug Development, Ongoing and Completed Projects being Undertaken by The Biomarkers Consortium 31
Table 13: Pathways to Efficient Drug Development, Open Innovation Platforms to Enhance Drug Discovery 33
Table 14: Pathways to Efficient Drug Development, Disease Specific Models Developed by the FDA 39
Table 15: Pathways to Efficient Drug Development, Pharmacometric Consultancies 41
Table 16: Pathways to Efficient Drug Development, Examples of Companies Offering Accelerator Mass Spectrometry Services 45
Table 17: Pathways to Efficient Drug Development, Eli Lilly’s Long-Term Service Providers 49
Table 18: Pathways to Efficient Drug Development, Open Innovation Business Models that Place Research Results in the Public Domain 51
Table 19: Pathways to Efficient Drug Development, Examples of Pharmaceutical Corporate Venture Capital Funds 53
Table 20: Pathways to Efficient Drug Development, New Companies Launched by Enlight Bioscience 54
Table 21: Pathways to Efficient Drug Development, Projects Funded by Pfizer’s Centers for Therapeutic Innovation 55
Table 22: Pathways to Efficient Drug Development, Examples of Recent Collaborations Between Academia and the Pharmaceutical Industry 56
Table 23: Pathways to Efficient Drug Development, AstraZeneca Compounds Made Available for Research (December 2011) 58

1.2 List of Figures
Figure 1: Pathways to Efficient Drug Development, Number of New Drug and Biologic FDA Approvals and Global R&D Expenditure by the Pharmaceutical Industry (2004-2011) 9
Figure 2: Pathways to Efficient Drug Development, Drivers for Innovation in the Pharmaceutical Industry 10
Figure 3: Pathways to Efficient Drug Development, The Rising Cost of Drug Development 1975-2005 10
Figure 4: Pathways to Efficient Drug Development, Changes in Clinical Trial Parameters between 2000-2003 and 2004-2007 11
Figure 5: Pathways to Efficient Drug Development, Failure Rates According to Therapeutic Area in Phase II and Phase III/Submission 13
Figure 6: Pathways to Efficient Drug Development, History of the European Innovative Medicines Initiative and its Strategic Research Agenda 18
Figure 7: Pathways to Efficient Drug Development, European Innovative Medicines Initiative: Strategic Research Agenda (updated 2012) 19
Figure 8: Pathways to Efficient Drug Development, New Drug Applications Filed with the FDA Centre for Drug Evaluation and Research (1996-2011) 20
Figure 9: Pathways to Efficient Drug Development, Key Areas of Precompetitive Research 21
Figure 10: Pathways to Efficient Drug Development, Disease, Drug and Trial Models: Pharmacometrics in Drug Development 39
Figure 11: Pathways to Efficient Drug Development, The I-SPY2 Adaptive Clinical Trial 44