バイオマーカーの実用化動向

Abstract

Introduction

As health care moves toward personalized medicine, biomarkers have gained an increasingly important role in the delivery of effi cient, quality care. The FDA has established specifi c defi nitions for biomarkers, and diagnostics, biotechnology, and pharmaceutical companies have woken up to biomarkers' potential following the publicity surrounding biomarkers associated with Genentech' s Herceptin. A large number of companies now have commercialized biomarker tests employing a variety of technologies, and activity is particularly prevalent in the oncology and drug dosing arenas. Although larger companies such as Roche have dominated molecular diagnostics, smaller companies willing to invest in niche product areas have seized the opportunity to develop equally valuable products.

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• Oncology and infectious diseases have proven to be the most popular targets of new biomarker development; however, oncology is favored because it has one specifi c advantage. What is that advantage?
• Although companies have already successfully commercialized oncology markers in breast cancer, new opportunities lie in other oncology areas. Which company is developing biomarker tests to detect signs of prostate and colon cancer, and which company is developing tests to detect the most common causes of cervical cancer?
• Focusing on generating scientifi c publications and clinical trial data throughout the development of a new biomarker test can allow for early commercialization of the test. Which company successfully employed this strategy to develop and commercialize its biomarker test-now reimbursable under the insurance plans of 125 million people?
• Increased regulatory oversight may make marketing some types of diagnostic tests more diffi cult. Which biomarker tests has the FDA specifi cally suggested it will seek to regulate?
• The approval of Genentech' s Herceptin, requiring the co-development of a test for HER2/NEU to qualify patients for treatment, has inspired other companies to try similar co-development strategies. Which companies have attempted to duplicate Herceptin' s success? Have these companies' attempts been successful?
• Companies having platform technologies that can provide simpler biomarker assays capable of being automated have an advantage over those companies that cannot provide such assays. Which companies have this capability?
• Roche is currently the dominant player in the fi eld of molecular diagnostics. What types of diagnostic systems have helped Roche to become the dominant player in this fi eld? What strategies has Roche pursued to improve on its diagnostics position?

Scope

• Current biomarker technologies: in vitro diagnostic tests (IVDs), immunohistochemistry (IHC) tests, fl uorescent in situ hybridization (FISH) tests, microarrays, and circulating tumor cells (CTCs)
• Biomarker diagnostics for targeted cancer therapies: Herceptin and its companion biomarkers; Genomic Health' s Oncotype DX biomarker test and Agendia' s MammaPrint test for breast cancer; Veridex' s CellSearch system to aid monitoring of metastatic breast cancer; biomarker tests to diagnose and monitor chronic myelogenous leukemia and determine initiation of Gleevec
• Companion biomarker diagnostics for drug dosing: biomarkers for Pfi zer' s Camptosar and Bristol-Myers Squibb' s Coumadin
• Commercialized biomarker products: successful products from Dako, Roche Diagnostics, Genomic Health, Agendia, Third Wave Technologies, Genzyme Genetics, and Veridex
• Opportunities: continued commercialization of oncology biomarkers; attractions of NAT technology; increasing importance of drug labeling; small companies' successes; signifi cant room for improvement
• Challenges: the reluctance of drug companies to restrict the use of their drugs through biomarker tests; uncertain regulatory environment; low reimbursement rates for diagnostic products; the diffi culties of developing companion diagnostics for EGFR-targeted drugs
• Outlook: licensing opportunities for smaller players; the advantages of platform biomarker technologies; reimbursement issues; the launch of validation and clinical trials; increasing chances of early adoption by coordinating product development with business strategy

Table of Contents

• Executive Summary
  • Strategic Considerations
  • Stakeholder Implications
• The Increasing Importance of Biomarkers
• A Wide Variety of Biomarker Technologies and Test Formats
• Oncology Biomarkers: Companion Diagnostics for Targeted Cancer Therapies
  • Overview
  • Commercial Success: Biomarker Tests for HER2/NEU/ERB2 Used with Herceptin in Breast Cancer
  • New Biomarker Tests: Possible Solutions for Prognosis and Therapy Selection in Breast Cancer
  • Biomarker Tests for Chronic Myelogenous Leukemia Incorporated Throughout Gleevec Development
• Biomarkers Used for Drug Dosing
  • Overview
  • Camptosar
  • Coumadin
• Profi les of Select Companies
  • Dako (Glostrup, Denmark)
  • Roche Diagnostics (Basel, Switzerland)
  • Genomic Health (Redwood City, California)
  • Agendia (Amsterdam, the Netherlands)
  • Third Wave Technologies (Madison, WI)
  • Genzyme Genetics (Framingham, MA)
  • Veridex (Raritan, NJ)
• Lessons Learned
  • Opportunities
  • Challenges
• Outlook
  • Biomarkers and Target Diseases
  • Advantages of a Platform Technology
  • Reimbursement Issues
  • Validation and Clinical Trials
  • Coordination of Product Development and Business Strategies

Tables

• 1. Major Technologies Currently Used for Biomarker Assays
• 2. Select Biomarkers Included in Drug Labeling
• 3. Select Commercialized Biomarkers, Analyte-Specifi c Reagents, and Products in Advanced Stages of Development

Figures

• 1. Applications of Biomarkers in Cancer Management
• 2. EGFR Family
• 3. Approximate Percentages of Drug Labels in the United States That Include Pharmacogenomic Information or Information Regarding Recommended or Required Tests

Sidebars

• FDA Biomarker Defi nitions
• Targeting EGFR-In Search of Biomarkers
• FDA Guidance on In Vitro Diagnostic Multivariate Index Assays