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April 2010

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Combination Drug-Diagnostics: Fueling Growth of Personalized Medicine - Table of Contents

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Author: Lucy Jean Sannes, Ph.D., M.B.A.

EXECUTIVE SUMMARY

CHAPTER 1
INTRODUCTION

CHAPTER 2
OVERVIEW OF PERSONALIZED MEDICINE 
2.1 What is Personalized Medicine?  What are Drug/Diagnostic Combinations? 
Drug/Diagnostic Combinations 
2.2 Selected Terms Used in Personalized Medicine 
Pharmacogenomics 
Pharmacogenetics 
Other Terms Used in Personalized Medicine 
2.3 Disease Applications in Personalized Medicine 
2.4 Diagnostic Technologies Used in Personalized Medicine 

CHAPTER 3
SELECTED EXAMPLES OF PERSONALIZED MEDICINE AVAILABLE TODAY 
3.1 Cytochrome P450 and Drug Metabolism 

CYP2D6 Phenotype and Response to Tamoxifen 
Cytochrome P450 Enzymes and Other Drugs 
3.2 Estrogen Receptor and Progesterone Receptor Status and Hormone Therapy for Breast Cancer 
3.3 HER2 Overexpression and both Herceptin and Tykerb - Tests for HER2 Overexpression Alone and in Combination with Other Biomarkers 
3.4 Multi-Gene/Biomarker Tests for Determining Breast Cancer Prognosis and Treatment 
Agendia‘s MammaPrint 
Applied Genomics’ MammoStrat 
bioTheranostics’ Theros Breast Cancer Index 
Genomic Health’s Oncotype DX 
Ipsogen’s MapQuant Dx Genomic Grade 
3.5 Multi-Gene/Biomarker Tests for Determining Diagnosis, Prognosis and Treatment of Other Cancers 
Applied Genomics‘ PulmoType 
Aureon Laboratories’ Prostate Px 
bioTheranostics’ Theros CancerTYPE ID 
Pathwork Diagnostics’ Pathwork Tissue of Origin Test 
3.6 Epidermal Growth Factor Receptor (EGFR) 
Currently Available Therapies That Target EGFR 
Testing for Expression of EGFR 
Testing for EGFR Mutations 
DxS (now part of Qiagen) 
Genzyme Genetics 
AutoGenomics 
3.7 KRAS Mutations and Anti-EGFR Therapy for Colorectal Cancer 
3.8 BRAF Mutations and Anti-EGFR Therapy for Colorectal Cancer 
3.9 UGT1A1 Genetic Variants and Treatment with Camptosar (irinotecan) 

Hologic 
AutoGenomics  
3.10 DPYD and TYMS Variants and 5-Flurouracil Therapy 
Myriad Genetics 
AutoGenomics 
Others 
3.11 Biomarkers in Leukemia and Lymphoma 
Gleevec and Other Tyrosine Kinase Inhibitors for Treatment of Philadelphia Chromosome Positive CML 
Philadelphia Chromosome and Busulfan 
PML/RAR and Vesanoid (tretinoin) 
Rituxan and Other Antibodies That Target CD20 Antigen for Treatment of Lymphoma or Leukemia 
Many Additional Biomarkers for Leukemia and Lymphoma 
3.12 Personalized Medicine and HIV Therapy - Moving Beyond HIV Viral Load and Drug Resistance Testing 
HLA-B*5701 Testing and Hypersensitivity to Abacavir 
CCR5-Tropic HIV and Selzentry (maraviroc) 
3.13 CYP2C9 and VKORC1 Variants and Warfarin Dosing 
3.14 Thiopurine Methyltransferase and Metabolism of Thiopurine Drugs 
3.15 Selected CNS Applications of Personalized Medicine 

HLA-B*1502 and Carbamazepine 
PGxPredict:CLOZAPINE 
3.16 Selected Other Personalized Medicine Tests 

CHAPTER 4 
DEVELOPMENT OF DRUG/DIAGNOSTIC COMBINATIONS - PERSONALIZED MEDICINE PRODUCTS
4.1 Identification of Personalized Medicine Biomarkers 
4.2 Validation of Diagnostic Tests for Personalized Medicine 
4.3 Development and Regulatory Issues Facing Diagnostic Companies in Personalized Medicine 
Regulatory Issues and Consideration for Clinical Laboratories with Laboratory Developed Tests for Personalized Medicine 
Regulatory Issues and Consideration for Diagnostics Companies in Personalized Medicine 
The two basic review processes 
4.4 Development and Regulatory Issues Facing Therapeutic Companies in Personalized Medicine 
4.5 Selected Personalized Medicine Examples - What Tests are Used by Physicians? 
Why Isn’t Warfarin Sensitivity Testing Performed Routinely Now? 
Screening for HLA-B*5701 Hypersensitivity to Abacavir 
Genomic Health and Validation of OncoType DX 
Agendia and Validation of MammaPrint 

CHAPTER 5
STRATEGIC ISSUES FACING PHARMACEUTICAL AND DIAGNOSTIC COMPANIES DEVELOPING PERSONALIZED MEDICINE PRODUCTS 
5.1 Need for More Research and Understanding 
Biomarkers and Other Factors Influencing Response to Therapy 
Validation Studies and Outcome Studies 
Other Research and Information Needs 
5.2 Models/Options for Pharmaceutical Companies Developing Drug/Diagnostic Combinations 
5.3 Models/Options for Diagnostic Companies Developing Personalized Medicine Tests - Test Development 
5.4 Delivery of New Diagnostic Tests - IVD versus CLIA Laboratory 
5.5 Health Economics, Reimbursement, and the Payers’ Perspective 

Health Economic Studies for Personalized Medicine Tests 
Perspectives Expressed in Expert Interviews 
Payer Perspectives and Policies 
Medco Health Solutions 
Generation Health 
5.6 Intellectual Property Issues 
5.7 Penetrating the Market 
Does the Test Affect Physicians’ Decisions? 
Acceptance by Experts; Incorporation into Guidelines 
Physician Education 

CHAPTER 6
COMPANY PERSPECTIVES ON PERSONALIZED MEDICINE 
6.1 Potential Benefits, Risks, and Challenges for Biopharmaceutical and Pharmaceutical Companies 
6.2 Potential Benefits, Risks, and Challenges for Diagnostic Companies

CHAPTER 7
POTENTIAL OPPORTUNITIES FOR THE FUTURE 
7.1 Tests in Development for Prediction of Breast Cancer Prognosis and Determining Treatment 
Agendia 
Applied Genomics (Subsidiary of Clarient) 
Celera 
Genomic Health 
Nuvera Biosciences 
Prediction Sciences and Clarient 
Additional Tests in Development for Prediction of Breast Cancer Prognosis and Determining Treatment 
7.2 Tests in Development for Determining Prognosis and Treatment of Other Cancers 
Agendia 
Applied Genomics (Subsidiary of Clarient) 
Genomic Health 
Additional Tests in Development for Prediction of Prognosis and Determining Treatment for Other (Non-Breast) Cancers 
7.3 Other (Non-Cancer) Personalized Medicine Tests in Development and/or Used in Pharmaceutical Research 
7.4 Pharmaceutical/Diagnostic Company Agreements 

CHAPTER 8
EXPERT INTERVIEWS 
Howard C. Coleman, Chief Executive Officer, Genelex Corporation 
Cornelius Diamond, CEO, Prediction Sciences 
Richard Ding, Chief Executive Officer and bioTheranostics (A bioMerieux Company) & Vice President of Strategy and Business Development at bioMerieux (the parent company) 
Stephen Eck, M.D., Ph.D., Vice President, Translational Medicine & Pharmacogenomics, Eli Lilly 
Paul Harkin, President & Managing Director, Almac Diagnostics 
Fareed Kureshym, Founder, Chairman, President and CEO, AutoGenomics 
Stephen Little, Vice President of Personalized Medicine, Qiagen, Former CEO and Founder of DxS (which was acquired by Qiagen) 

REFERENCES

COMPANY INDEX WITH WEB ADDRESSES