Cover Image
Market Research Report

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

Published by GBI Research Product code 251903
Published Content info 66 Pages
Immediate Delivery Available
Price
Back to Top
Pathways to Efficient Drug Development - Advances in Modeling and Simulation Outcomes to Fuel Pipeline Productivity
Published: September 30, 2012 Content info: 66 Pages
Description

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

Executive Summary

Clinical Trials Due an Overhaul to Conquer the Pharma 'Valley of Death'.

Costs associated with drug development have risen from around $138m in 1975 to over $1 billion in 2005, with clinical trials representing a key factor in this increasing expenditure, according to a new report by healthcare industry experts GBI Research.

The new research* looks at why researchers in the pharma industry now refer to the clinical development process as the 'valley of death', which must be crossed to attain drug approval.

Clinical trials have grown longer and more complex, while volunteer enrolment and retention rates have fallen. More than 30% of experimental drugs that reach Phase III fail at this point. Late stage failures are costly to the industry - Pfizer's torcetrapib, which failed its Phase III trial in 2006, led to the value of Pfizer dropping by $21 billion over night, and 10,000 job losses being announced the following year.

Recent analyses show that this problem has grown in recent years. Overall, clinical approval success rates have fallen to approximately 16%, though this figure varies widely by therapeutic area. Pivotal trial and regulatory failures were recorded for 31 drugs in 2011, with Eli Lilly, Bristol-Myers Squibb, AstraZeneca, Merck, Sanofi, Novartis and GlaxoSmithKline all suffering at least one setback.

The most common reason for drug failure during Phase II development is a lack of efficacy as determined by primary endpoints. Phase III failures tend to be in therapeutic areas that may provide a higher chance of approval and reimbursement, such as cancer or neuroscience. Whilst it is tempting to speculate that drug development is more difficult for drugs with novel mechanisms of action in these areas of unmet need, this data implies that the pressure on companies to keep development pipelines full may also have led to compounds advancing into Phase III on the basis of inadequate or marginal proof-of-concept.

Others have argued that the majority of failures in clinical trials are due to the inadequacy of animal models to predict success in humans, implying a fundamental flaw in drug development processes which use animal testing methods.

It is essential that R&D productivity is improved, and the 'quick win, fast fail' model is being touted as a possible way to achieve this. This model argues that investments made early in the process increase the information available on which to base key decisions, enabling the earlier termination of projects prior to huge investments being made for the Phase III program.

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

This 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 data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research's team of industry experts.

Table of Contents
Product Code: GBIHC260MR

Table of Contents

1. Table of Contents

  • 1.1. List of Tables
  • 1.2. List of Figures

2. State of the Industry

  • 2.1. Introduction
    • 2.1.1. The Rising Costs of Drug Development
    • 2.1.2. Drug Attrition
    • 2.1.3. Patent Expiries
    • 2.1.4. Regulatory Hurdles
    • 2.1.5. The Fourth Hurdle: Reimbursement
  • 2.2. Innovation in the Drug Development Paradigm
    • 2.2.1. The Critical Path Initiative
    • 2.2.2. The Innovative Medicines Initiative
  • 2.3. Improving Drug Development
  • 2.4. 2012 and Beyond

3. Collaboration in the Precompetitive Space

  • 3.1. Defining Precompetitive Research
  • 3.2. Building Successful Consortia
    • 3.2.1. Choosing the Research Topic
    • 3.2.2. The Set-Up Phase
    • 3.2.3. Project Management
    • 3.2.4. Measuring Success
    • 3.2.5. Case Study of a Successful Private Public Partnership: The Alzheimer's Disease Neuroimaging Initiative
  • 3.3. Qualification of Biomarkers of Efficacy or Safety
    • 3.3.1. Case Study: Biomarkers of Kidney Injury
    • 3.3.2. Case Study: The Biomarkers Consortium
  • 3.4. Open Innovation Platforms
    • 3.4.1. Case Study: OpenPHACTS
    • 3.4.2. Case Study: Sage Bionetworks
  • 3.5. Data Standards
    • 3.5.1. BioSharing: Standard Cooperating Procedures
    • 3.5.2. Clinical Data Standards
    • 3.5.3. Data Standards and the FDA
  • 3.6. Conclusions

4. Improving Drug Development Efficiency

  • 4.1. Modeling and Simulation
    • 4.1.1. Modeling and Simulation: A View from the Regulators
    • 4.1.2. Model Qualification
    • 4.1.3. Modeling and Simulation Expertise and Consultancy
  • 4.2. Innovative Approaches to Clinical Trials
    • 4.2.1. Adaptive Clinical Trials
    • 4.2.2. Case Study: The I-SPY 2 Trial
    • 4.2.3. Exploratory Clinical Trials
  • 4.3. Engaging Stakeholders
    • 4.3.1. Patients
    • 4.3.2. Regulators
    • 4.3.3. Payers
  • 4.4. Conclusions

5. Business Models

  • 5.1. Introduction
  • 5.2. R&D Reorganization
    • 5.2.1. Mimicking the Biotech Environment
    • 5.2.2. The Fully Integrated Pharmaceutical Network Model
  • 5.3. Open Innovation
    • 5.3.1. Case Study: An Open Innovation Incubator
    • 5.3.2. Case Study: Open Innovation Drug Discovery at Eli Lilly
  • 5.4. Funding for External Innovation
    • 5.4.1. Collaborative Commercialization
  • 5.5. Academic Partnerships and Translational Medicine
    • 5.5.1. Translational Science in the US
    • 5.5.2. Case study: Medical Research Council/AstraZeneca
  • 5.6. Conclusions

6. Appendix

  • 6.1. Abbreviations
  • 6.2. Methodology
    • 6.2.1. Primary Research
    • 6.2.2. Secondary Research
  • 6.3. References
  • 6.4. Contact Us
  • 6.5. Disclaimer

List of Tables

  • Table 1: Pathways to Efficient Drug Development, Clinical and FDA Approval Times across Therapeutic Classes (2005-2009)
  • Table 2: Pathways to Efficient Drug Development, Transition Probability at Each Stage of Clinical Drug Development
  • Table 3: Pathways to Efficient Drug Development, Overall FDA Approval Success Rate for New Chemical Entities by Therapeutic Area
  • Table 4: Pathways to Efficient Drug Development, Loss of US Sales Revenues Due to Patent Expiries ($m; 2010-2013)
  • Table 5: Pathways to Efficient Drug Development, Drugs Withdrawn from the Market in the US (1992-2010)
  • Table 6: Pathways to Efficient Drug Development, Ongoing Projects of the Critical Path Institute
  • Table 7: Pathways to Efficient Drug Development, Proposed Network for Evaluating PPPs in the Pharmaceutical Sciences
  • Table 8: Pathways to Efficient Drug Development, Biomarkers qualified by the FDA for use in drug development
  • Table 9: Pathways to Efficient Drug Development, Biomarkers qualified by the EMA for use in drug development
  • Table 10: Pathways to Efficient Drug Development, Ongoing Public-Private Partnerships for Biomarker Identification and Qualification
  • 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
  • Table 12: Pathways to Efficient Drug Development, Ongoing and Completed Projects being Undertaken by The Biomarkers Consortium
  • Table 13: Pathways to Efficient Drug Development, Open Innovation Platforms to Enhance Drug Discovery
  • Table 14: Pathways to Efficient Drug Development, Disease Specific Models Developed by the FDA
  • Table 15: Pathways to Efficient Drug Development, Pharmacometric Consultancies
  • Table 16: Pathways to Efficient Drug Development, Examples of Companies Offering Accelerator Mass Spectrometry Services
  • Table 17: Pathways to Efficient Drug Development, Eli Lilly's Long-Term Service Providers
  • Table 18: Pathways to Efficient Drug Development, Open Innovation Business Models that Place Research Results in the Public Domain
  • Table 19: Pathways to Efficient Drug Development, Examples of Pharmaceutical Corporate Venture Capital Funds
  • Table 20: Pathways to Efficient Drug Development, New Companies Launched by Enlight Bioscience
  • Table 21: Pathways to Efficient Drug Development, Projects Funded by Pfizer's Centers for Therapeutic Innovation
  • Table 22: Pathways to Efficient Drug Development, Examples of Recent Collaborations Between Academia and the Pharmaceutical Industry
  • Table 23: Pathways to Efficient Drug Development, AstraZeneca Compounds Made Available for Research (December 2011)

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)
  • Figure 2: Pathways to Efficient Drug Development, Drivers for Innovation in the Pharmaceutical Industry
  • Figure 3: Pathways to Efficient Drug Development, The Rising Cost of Drug Development 1975-2005
  • Figure 4: Pathways to Efficient Drug Development, Changes in Clinical Trial Parameters between 2000-2003 and 2004-2007
  • Figure 5: Pathways to Efficient Drug Development, Failure Rates According to Therapeutic Area in Phase II and Phase III/Submission
  • Figure 6: Pathways to Efficient Drug Development, History of the European Innovative Medicines Initiative and its Strategic Research Agenda
  • Figure 7: Pathways to Efficient Drug Development, European Innovative Medicines Initiative: Strategic Research Agenda (updated 2012)
  • Figure 8: Pathways to Efficient Drug Development, New Drug Applications Filed with the FDA Centre for Drug Evaluation and Research (1996-2011)
  • Figure 9: Pathways to Efficient Drug Development, Key Areas of Precompetitive Research
  • Figure 10: Pathways to Efficient Drug Development, Disease, Drug and Trial Models: Pharmacometrics in Drug Development
  • Figure 11: Pathways to Efficient Drug Development, The I-SPY2 Adaptive Clinical Trial
Back to Top