Personalized Medicine 2010
Arrowhead Publishers, May 2010, Pages: 261
Assessing the Opportunities and Challenges in Commercializing Companion Diagnostics and Targeted Therapies
The era of Personalized Medicine is set to revolutionize the healthcare industry. As our knowledge of genetics and molecular biology rapidly expands, a personalized approach to medical care is becoming increasingly important. Not only does this paradigm promise to improve an individual’s treatment outcome and reduce healthcare expenditure, personalized medicine is set to transform the pharmaceutical/biotechnology and companion diagnostic markets.
The era of Personalized Medicine is set to revolutionize the healthcare industry. As our knowledge of genetics and molecular biology rapidly expands, a personalized approach to medical care is becoming increasingly important.
Not only does this paradigm promise to improve an individual’s treatment outcome and reduce healthcare expenditure, personalized medicine is set to transform the biopharmaceutical and molecular diagnostics markets.
Arrowhead’s Personalized Medicine 2010 report provides an in-depth assessment of this burgeoning market, and examines the major challenges that need to be overcome to maximize success.
Key features of Arrowhead’s Personalized Medicine 2010 include:
- Case studies of leading pharmacogenomic products
- Analysis of the personalized medicine market, including seven major market sales forecasts to 2016
- Regulatory and legal issues surrounding personalized medicine
- Payer reimbursement challenges
- Marketing strategies to capitalize profits
Key questions answered by Personalized Medicine 2010 include:
- What are the major challenges in this market and how can these be overcome?
- How will pharmacogenomics transform clinical trials?
- What are the leading pharmacogenomic products on the market?
- Who are the key players involved in the market and what strategies are they using to enhance product performance?
- What pharmaceutical/biotechnology and diagnostic companies are seeking alliances in 2010?
- How can company alliances maximize commercial success?
Key Findings
Rather than developing blockbuster drugs that target broad populations, in an era of personalized medicine, industry must discover and develop tailored therapies for smaller markets and the development of new therapeutics based on genomics and proteomics will require an entirely new level of tailoring.
There are many benefits associated with the pursuit of personalized medicine, among them the ability to understand risks and benefits at the level of the individual, the expanded use of biomarkers to identify unpromising drugs early in the R&D process and to run smaller, accelerated clinical trials that can be modified in mid-stream as evidence emerges.
By targeting subpopulations, the market for personalized medicine may follow the niche orphan drug structure. Currently, orphan drug status is awarded to therapeutics that target rare diseases or conditions, where the target population is less than 200,000 people. With the FDA offering funding grants for clinical research in rare diseases, this could be further incentive for personalized medicine companies wishing to gain commercial success.
Personalized medicine may breathe new life into drug programs that were abandoned during the development process or withdrawn from the market due to safety concerns.
Recommendations for Moving Forward
Articulate the benefits of products in the pipeline to payers and regulators as early as possible in the R&D process to win their support and avoid the time and expense of developing a drug or diagnostic that cannot be commercialized.
To help make the business case for reimbursement, develop health economic models that project the potential cost savings of products in development.
Work proactively with regulators to educate them about the need for a reasonable period of IP exclusivity as well as new pathways for approval of targeted diagnostics, therapeutics and MTT companion diagnostics. Assist in offering novel solutions.
Collaborate with payers in developing novel reimbursement strategies that minimize your risks and costs, such as an accelerating schedule of reimbursements as products progress through their lifecycle and demonstrate their efficacy.
1 EXECUTIVE SUMMARY
1.1 SCOPE OF PERSONALIZED MEDICINE: MARKET TRENDS AND R&D INSIGHTS
1.2 METHODOLOGY OF PERSONALIZED MEDICINE: MARKET TRENDS AND R&D INSIGHTS
1.3 KEY CHALLENGES IN THE COMMERCIALIZATION OF PERSONALIZED MEDICINE
1.4 OVERVIEW OF THE OPTIMIZATION OF R&D
1.5 OVERVIEW OF THE POTENTIAL PERSONALIZED MEDICINE MARKET
1.6 STRATEGIC ALLIANCES ARE NEEDED TO GAIN SUCCESS
2 AN INTRODUCTION TO PERSONALIZED MEDICINE
2.1 BIOMOLECULAR ADVANCES AND THE REVOLUTION OF THE HEALTHCARE INDUSTRY
2.2 WHAT IS PERSONALIZED MEDICINE?
2.2.1 The Definition of Personalized Medicine
2.2.2 The Definition of Pharmacogenetics and Pharmacogenomics
2.2.3 The Aims of Personalized Medicine
2.2.4 The History of Personalized Medicine
2.3 PERSONALIZED MEDICINE AND THE CHANGING HEALTHCARE PARADIGM
2.4 THE POTENTIAL BENEFITS OF PERSONALIZED MEDICINE
2.4.1 A Preventative Outlook to Healthcare
2.4.2 Optimal Therapy Selection
2.4.3 Safer Drugs with Less Adverse Drug Reactions
2.4.4 Improve Patient Compliance
2.4.5 Optimize Clinical Trials to Improve R&D
2.4.6 Reduce Healthcare Costs
2.5 PERSONALIZED APPROACH VERSUS CURRENT PRACTICE PARADIGMS
3 OVERVIEW OF PHARMACOGENOMICS: THE SCIENCE BEHIND PERSONALIZED MEDICINE
3.1 AN INTRODUCTION TO GENOMICS AND PROTEOMICS
3.1.1 What is DNA?
3.1.2 What is RNA?
3.1.3 What is a Gene?
3.1.4 What is a Genome?
3.1.5 What are Proteins?
3.1.6 Mechanisms of Gene Expression…From Genes to Proteins
3.1.7 Genetic Mutations
3.1.8 What is a Proteome?
3.1.9 The Human Proteome versus the Human Genome
3.2 THE CREATION OF HUMAN GENETIC RESEARCH DATABASES
3.3 IDENTIFICATION OF THE HUMAN GENOME
3.3.1 An Overview of the Methodology of Genome Sequencing
3.3.2 The Human Genome Project (HGP)
3.3.3 The International HapMap Consortium
3.4 GENOMIC RESEARCH INTO GENETIC POLYMORPHISMS
3.4.1 The 1000 Genomes Project
3.4.2 The SNP Consortium
3.4.3 The dbSNP
3.4.4 The Pharmacogeneomics Research Network and Knowledge Base (PharmaGKB)
3.4.5 The Future Outlook of Human Genetic Databases
3.4.6 The Race for the $1000 Human Genome Sequence
3.4.7 The Market Potential of Sequencing the Human Genome
4 BIOMARKERS: LEADING THE PATH TO PERSONALIZED MEDICINE
4.1 THE DEFINITION OF BIOMARKERS
4.1.1 Applications of Biomarkers
4.2 CASE STUDY OF THE USE OF BIOMARKERS TO MIMIMIZE ADRS
4.2.1 Biomarkers of ADR Susceptibility in US FDA-Approved Product Labels
4.2.2 Steps in the Development & Utilization of Genomic Biomarkers for ADRs
4.3 VALIDATING BIOMARKERS WITH REGULATORS
4.3.1 Joint Cooperation of the FDA and EMEA to Validate Kidney Damage Biomarkers
4.4 BIOMARKER PROGRAMS CREATED BY PHARMACEUTICAL/BIOTECHNOLOGY COMPANIES AND THE RISE OF THE BIOMARKER MARKET
5 OVERVIEW OF THE COMPANION DIAGNOSTICS MARKET
5.1 INTRODUCTION TO COMPANION DIAGNOSTICS
5.1.1 The Definition of Companion Diagnostics
5.1.2 The Need for Companion Diagnostics
5.2 SELECTED CASE STUDIES OF COMPANION DIAGNOSTICS
5.2.1 Roche/Affymetrix’s AmpliChip Cytochrome P450 Genotyping Test (AmpliChip CYP450)
5.2.3 Agendia’s MammaPrint
5.2.4 Genomic Health’s OncoType DX Test
5.2.5 Genentech/Roche/Dako’s HercepTest
5.3 GOVERNING STANDARDS OF GENETIC TESTS
5.3.1 Diagnostic Performance: Specificity and Sensitivity Effectiveness
5.3.2 The ACCE Framework Model
5.4 REGULATION OF COMPANION DIAGNOSTICS
5.4.1 FDA Regualtion of IVDMIAs
5.4.2 Genentech’s Push for all Diagnostics to be FDA Approved
5.4.3 CLIA Regulation of Laboratories
5.5 REIMBURSEMENT OF COMPANION DIAGNOSTICS
5.6 THE COMPANION DIAGNOSTIC PIPELINE
5.7 THE COMPANION DIAGNOSTICS MARKET
6 PERSONALIZED MEDICINE R&D OPTIMIZATION
6.1 OVERVIEW OF CLINICAL TRIALS
6.2 ISSUES SURROUNDING THE DESIGN OF CLINICAL TRIALS
6.2.1 Patient Selection
6.2.2 Trial Duration
6.2.3 Trial Comparator Drug Choice
6.2.4 Trial End-Point
6.3 THE COST OF R&D
6.4 PERSONALIZED MEDICINE APPLICATIONS IN R&D
6.4.1 The Preclincal Stage: Development of Targeted Drugs for Specific Genetic Disorders
6.4.2 The End of Traditional Phase I and Phase IIA Trials
6.4.3 The Introduction of Adaptive Phase IIB and Phase III Trials
6.4.4 Continuing Phase IV Studies
6.4.5 The Use of Biomakers to Aid R&D
6.4.6 Arrowhead’s Recommendations for Maximizing Personalized Medicine R&D
7 REGULATORY HURDLES IN THE COMMERCIALIZATION OF PERSONALIZED MEDICINE
7.1 INTRODUCTION TO THE REGUALTION OF PERSONALIZED MEDICINE
7.2 FDA PERSONALIZED MEDICINE SUBMISSIONS
7.2.1 Submission of Personalized Medicine Data to an IND
7.2.2 Submission of Personalized Medicine Data to a New NDA, BLA or Supplement
7.2.3 Submission of Personalized Medicine Data to an Approved NDA, BLA or Supplement
7.2.4 FDA Voluntary Exploratory Data Submissions (VXDSs)
7.2.5 FDA Voluntary Genomic Data Submissions (VGDSs)
7.2.6 FDA Labeling Regulations
7.3 EMEA PHARMACOGENOMIC SUBMISSIONS
7.4 ...PROCESSING JOINT FDA -EMEA VOLUNTARY GENOMIC DATA SUBMISSIONS (VGDSS) WITHIN
THE FRAMEWORK OF THE CONFIDENTIALITY ARRANGEMENT
7.5 JAPANESE REGULATION
7.6 GLOBAL PERSONALIZED MEDICINE HARMONIZATION: THE INTERNATIONAL CONFERENCE ON HARMONISATION OF TECHNICAL REQUIREMENTS FOR REGISTRATION OF PHARMACEUTICALS FOR HUMAN USE (ICH)
7.6.1 Structure of the ICH
7.6.2 ICH Personalized Medicine Regulations
7.6.3 The Outlook for Personalized Medicine Regulation
8 PERSONALIZED MEDICINE LEGAL AND REIMBURSEMENT CHALLENGES
8.1 US LEGISLATION AND GOVERNMENT INITIATIVES
8.2 INTELLECTUAL PROPERTY ISSUES
8.3 PAYER REIMBURSEMENT ISSUES
8.3.1 Personalized Medicine Potentially Offers Cost Savings and Quality Improvements
8.3.2 The CMS Reimbursement Process
8.3.3 Modernizing Reimbursement to Support Personalized Medicine
9 PERSONALIZED MEDICINE ETHICAL CONCERNS
9.1 INTRODUCTION TO ETHICAL ISSUES SUROUNDING PERSONALIZED MEDICINE
9.2 HEALTH INEQUALITIES
9.3 HEALTHCARE INFORMATION TECHNOLOGY (HIT) AND ADOPTION OF ELECTRONIC HEALTH RECORDS (EHRS)
9.4 PATIENT CONFIDENTIALITY
9.5 DISCRIMINATION ISSUES
9.6 RACIAL ISSUE, CASE STUDY OF NITROMED’S BIDIL (ISOSORBIDE/HYDRALAZINE)
10 PERSONALIZED MEDICINE THERAPEUTICS
10.1 INTRODUCTION TO PERSONALIZED MEDICINE THERAPEUTICS
10.2 ONCOLOGY PERSONALIZED MEDICINE THERAPEUTICS
10.3 LUNG CANCER
10.3.1 OSI Pharmaceuticals/ Roche’s Tarceva (erlotinib)
10.3.2 AstraZeneca’s Iressa (gefitinib)
10.4 BREAST CANCER
10.4.1 Genentech/Roche’s Herceptin (trastuzumab)
10.4.2 Clinical Efficacy
10.5 GLAXOSMITHKLINE’S TYKERB/TYCERB (LAPATINIB)
10.5.1 Roche’s Avastin (bevacizumab)
10.6 COLORECTAL CANCER
10.7 IMCLONE/BRISTOL-MYERS SQUIBB/MERCK KGAA’S ERBITUX (CETUXIMAB)
10.8 LEUKEMIA
10.8.1 Novartis’ Gleevec/Glivec (imatinib)
10.8.2 Clinical Efficacy
10.8.3 Safety Profile
10.8.4 Bristol-Myers Squibb’s Sprycel (dasatinib)
10.8.5 Novartis’ Tasigna (nilotinib)
11 PERSONALIZED MEDICINE’S MARKET IMPACT
11.1 THE END OF THE TRADITIONAL BLOCKBUSTER BUSINESS MODEL…
11.2 …AND THE PURSUIT OF NICHE BLOCKBUSTERS
11.3 REVIVING FAILED DRUG CANDIDATESAND WITHDRAWN DRUGS
11.4 EXPANDING INDICATIONS FOR LAUNCHED AND GENERIC DRUGS
11.5 SWOT ANALYSIS OF THE PERSONALIZED MEDICINE MARKET
11.6 ARROWHEAD’S PERSONALIZED MEDICINE REVENUE FORECAST, 2004-2019
12 PERSONALIZED MEDICINE COMMERCIALIZATION STRATEGIES
12.1 THE NEED TO COLLABORATE
12.2 MAJOR PHARMA/BIOTECH COMPANY STRATEGIES
12.3 COMPANION DIAGNOSTIC COMPANY STRATEGIES
12.4 ARROWHEAD’S RECOMMENDATIONS
13 APPENDIX
14.1 EPIDEMIOLOGY ANALYSIS
14.2 BIBLIOGRAPHY
- AstraZeneca
- Bristol-Myers Squibb
- Genentech
- Glaxosmithkline
- Novartis
- OSI Pharmaceuticals
- Roche
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