Impact of Pharmacogenomics on Public Healthcare Policy: Educating patients, payors and healthcare providers

Table of Contents

Executive Summary

• Pharmacogenomics defined
• Application & implementation of PGx by the industry
• Pharmacogenomic strategies, alliances & licensing opportunities
• Pharmacogenomic regulation & implementation
• Reimbursement - coverage, coding & payment
• Future challenges and opportunities

Chapter 1 Pharmacogenomics defined

• Summary
• Introduction
• What is pharmacogenomics & pharmacogenetics?
• Why is pharmacogenomics important?
• Rising R&D costs
• Long development times
• High risk drug development
• Current pharmacogenomic environment
• PGx testing for targeted therapies
• PGx diagnostics to improve safety
• Conclusions

Chapter 2 Implementation of PGx by the industry

• Summary
• Introduction
• Current applications
• Efficacy studies
• Safety and toxicity studies
• Dosing studies
• Implications
• Target identification
• Pre-clinical development
• Clinical development
• Phase 4 development
• Conclusions

Chapter 3 Pharmacogenomic strategies, alliances and licensing opportunities

• Summary
• Introduction
• Pharmaceutical strategies to PGx
• AstraZeneca
• Bayer Schering Pharma
• Eli Lilly
• GlaxoSmithKline
• Pfizer
• Roche
• Conclusions- Pharma PGx strategies
• Biotech strategies to PGx
• Amgen
• Genentech
• Millennium Pharmaceuticals
• Conclusions- Biotech PGx strategies
• Diagnostic strategies to PGx
• Clinical Data
• Dako
• Diaceutics
• Genomic Health
• Genzyme Genetics
• Monogram Biosciences
• Conclusions- Diagnostic PGx strategies
• Interactions between stakeholders
• Consortia
• Intellectual property
• IP conclusions
• Conclusions

Chapter 4 Adoption of pharmacogenomics by regulators

• Summary
• Introduction
• Regulators: US, Europe and Japan
• USA
• Europe
• Japan
• Global PGx harmonization: ICH
• In vitro diagnostics
• The US
• Europe
• PGx guidance on IVD devices
• Conclusions

Chapter 5 PGx and Health Economics

• Summary
• Introduction
• Reimbursement criteria
• Coverage
• Coding
• Payment
• Reimbursement drivers
• Reimbursement in the US
• Reimbursement in Europe
• Eligibility of coverage
• Medical support for PGx coverage
• Dx coding
• Payment and pricing
  • Case study 1: DPD testing
• Cost-effectiveness of PGx
• Commercially viable PGx testing
  • Case Study 2: HLA B*5701 testing
• Industry incentives
• Conclusions

Chapter 6 Future challenges and opportunities

• Summary
• Introduction
• Challenges
• Opportunities
• Market trends
• Market sales
• Conclusions

Chapter 7 Appendix

• Acknowledgements
• Acronyms
• Bibliography
• Glossary
• Index

List of Figures

• Figure 1.1: A genomic timeline
• Figure 1.2: The concept of pharmacogenetics
• Figure 1.3: Costs, time and success rates associated with drug development
• Figure 1.4: R&D costs savings following the application of pharmacogenetics
• Figure 1.5: R&D time savings following the application of genomics technologies in drug discovery
• Figure 2.6: Applications of PGx/biomarkers in drug development timelines
• Figure 2.7: Applications of new technologies to develop new medicines
• Figure 2.8: Response rates to current medicines
• Figure 2.9: Factors affecting efficacy and outcome of drug treatment
• Figure 2.10: Response rates to Iressa
• Figure 2.11: US Reported Adverse Drug Reactions (1995-2001)
• Figure 2.12: Relationship of drugs, disease, genes and gene products
• Figure 2.13: Roche AmpliChip CYP450 array technology
• Figure 2.14: Dose response curves to A) penicillin and B) 6MP
• Figure 2.15: Warfarin mode of action, metabolism and inter-individual variation
• Figure 3.16: Pharmacodynamic biomarkers for dose-selection
• Figure 3.17: PGx and biomarkers to aid decision making
• Figure 3.18: Bayer' s high tech research platform
• Figure 3.19: Obesity patients stratified according drug response and weight loss/weight gain
• Figure 3.20: Pfizer- Applying PGx
• Figure 3.21: Genotyping an integral part of diagnosis and treatment
• Figure 3.22: Roche -Pharmaceuticals' and diagnostics' approach
• Figure 3.23: Amgen - PGx approach in R&D and Drug Therapy
• Figure 3.24: Genentech - development history of Herceptin/HercepTest
• Figure 3.25: Genentech - multiple targeted cancer therapy
• Figure 3.26: Millennium - molecular classifiers of Velcade response
• Figure 3.27: Millennium - Survival classifiers stratifies risk groups
• Figure 3.28: Diaceutics Library - Rationale for its CMR model
• Figure 3.29: Genomic Health Product Pipeline 2008-2009
• Figure 3.30: Genzyme Genetics Product Pipeline 2008-2009
• Figure 3.31: Monogram Biosciences molecular diagnostics approach
• Figure 4.32: The organization of the Interdisciplinary Pharmacogenomic Review Group (IPRG)
• Figure 4.33: FDA' s voluntary PGx submission pathway
• Figure 4.34: The organization of the EMEA
• Figure 5.35: CMS a dominant third-party payor A) 2002A and B) 2008E
• Figure 5.36: Roche - US & EU approval & reimbursement of new tests
• Figure 6.37: Market trends and drivers for the industry
• Figure 6.38: Challenges in PGx
• Figure 6.39: Opportunities in PGx
• Figure 6.40: Key stakeholder in PGx
• Figure 6.41: Future PGx Landscape
• Figure 6.42: Future PGx Landscape
• Figure 6.43: PGx Sales Forecasts 2005-2015

List of Tables

• Table 1.1: PGx - Gene Association Studies
• Table 1.2: Targeted Oncology Products and PGx based tests
• Table 1.3: PGx tests to aide prescription and reduce the risk of ADRs
• Table 2.4: Patient stratification for Herceptin trials
• Table 2.5: Drug withdrawals 1996-2004
• Table 2.6: PharmGKB Knowledge Base annotated PGx genes
• Table 2.7: Warfarin dosage versus genetic variant
• Table 2.8: Disease-associated alleles underlie complex disease phenotypes
• Table 3.9: SNP Coverage per candidate gene
• Table 3.10: Recent strategic alliances and collaborations in PGx
• Table 3.11: PGx driven consortia
• Table 5.12: PGx Dx value based pricing
• Table 5.13: Types of economic evaluations in health care
• Table 5.14: Assessment of the potential cost-effectiveness of PGx interventions
• Table 5.15: Potential cost-effective PGx applications