Abstract
The application of pharmacogenomics in drug development is widespread today, in differing degrees of intensity. Indeed, industry leaders have stated that:
"Pharmacogenomics is now an integral part of drug development. It is an emerging part, but it is already taking place within all the drug companies."
The focus of pharmacogenomics is currently selecting targets that are validated from a genetics perspective before looking into the entire genome, attacking attrition early in development (Phase I and II), addressing adverse reactions in Phase III, and supporting a standardized system of post-launch surveillance with regulatory oversight.
Addressing attrition earlier in the drug development process can have a huge impact on development cost. Indeed, moving just 10% of Phase III failures to Phase I reduces the average total cost of developing a drug by $98 million.
There is considerable concern in the industry, and on Wall Street, that the net impact of the introduction of pharmacogenomics will be negative. Yet, the economic and the logistical arguments for not implementing pharmacogenomics are not sound. The average sales of a conventional drug are approximately $63 million lower than for a pharmacogenomics-developed drug.
Assuming a 30-year revenue lifecycle, the Net Present Value (NPV) of the pharmacogenomics drug is approximately $85 million higher than the conventional drug.
Table of Contents
Chapter 1: Executive Summary 1--1
- Overview 1--1
- Key Pharmacogenomics Players 1--2
- Cost Opportunities 1--4
- Regulatory Impact 1--6
- References 1--8
Chapter 2: Introduction 2--1
- Genetic Distinction and the Impact on Medicine 2--1
- Opportunities and Threats Impacting Companies and Patients 2--3
- Trends in Pharmacogenomics 2--4
- Driving Force for Leading Technologies and Tools 2--5
- References 2--7
Chapter 3: Companies Realizing The Pharmacogenomics Opportunity 3--1
- Market Opportunities 3--1
- Large Pharma 3--1
- Late-stage Compound or Whole Company Acquisition 3--1
- Early-stage Compound Acquisitions 3--1
- Roll Up of Individual Business Units 3--1
- Biotechnology 3--2
- Microarray Chip 3--2
- Services and Diagnostic Kits 3--2
- Platform Technology 3--2
- Bundled Drug and Test 3--2
- Requirements for Market Adoption in the Pharmaceutical Industry 3--3
- Substantiation of Polymorphism to Drug Reaction Relationship 3--3
- Sufficiently Low Cost of SNP Testing 3--3
- Diagnostic Testing Defined in Trial Protocol 3--4
- Reimbursement Coverage for SNP Testing 3--5
- Healthcare and Doctor Acceptance of Diagnostic Testing 3--5
- Mandatory Diagnostic Acceptance for Industry Acceptance 3--6
- Economic Viability of Using Diagnostic in the Drug Sales Model 3--6
- Breakeven Analysis of Diagnostic-Enabled Drug Model 3--7
- Implications for Industry 3--8
- References 3--9
Chapter 4: Industry Forecast 4--1
- Forms of Pharmacogenomics Success 4--1
- Drug Prescriptions Aligned with Patient Needs 4--2
- Drug Distribution Augmentation and Expansion 4--3
- Blockbuster Extension via Price and Volume 4--4
- Key Elements of the Pharmaceutical Value Chain 4--5
- Pharmaceutical and Biotech Companies 4--6
- NCE (New Chemical Entity) Introduction 4--6
- Marketing Strategy and Execution 4--7
- Clinical Trial Management 4--8
- Molecular Diagnostics and Testing 4--9
- Payer-Provider 4--10
- Physicians and Healthcare Delivery 4--11
- Adverse Events and Negligence 4--11
- Negligence and Litigation 4--12
- Relative Cost of Adverse Reactions 4--13
- References 4--14
Chapter 5: Regulatory Update 5--1
- Current Regulatory Process for Drug Development 5--1
- Key Issues Facing Industry 5--4
- Issues Facing the FDA 5--5
- Adequate and Appropriate Informed Consent 5--5
- SNP Data Collection in Clinical Trials 5--6
- Impact to Industry from Pharmacogenomics Regulation 5--6
- Implications for Industry 5--8
- Defend Existing Revenue Streams 5--9
- Ensure that the Basis of Differentiation in Your Diagnostic Tests is Valid 5--9
- Understand Market Opportunities Afforded Players with Drug/Test Combinations 5--9
- Conclusion 5--9
- References 5--10
Chapter 6: Summary of FDA Guidance for Industry 6--1
- Guidance for Industry1 Pharmacogenomic Data Submissions 6--1
- Introduction 6--1
- Background 6--2
- Submission Policy 6--3
- General Principles 6--3
- Specific Uses of Pharmacogenomic Data in Drug Development and Labeling 6--5
- Benefits of Voluntary Submissions to Sponsors and FDA 6--6
- Submission of Pharmacogenomic Data 6--8
- Submission of Pharmacogenomic Data During the IND Phase 6--8
- Submission of Pharmacogenomic Data to a New NDA, BLA, or Supplement 6--10
- Submission to a Previously Approved NDA or BLA 6--11
- Compliance with 21 CFR Part 58 6--12
- Submission of Voluntary Genomic Data from Application-Independent Research 6--12
- Format and Content of a VGDS 6--12
- Process For Submitting Pharmacogenomic Data 6--14
- Agency Review Of Voluntary Genomic Data Submissions 6--14
- Glossary 6--17
- Appendix A: Submission of Pharmacogenomic (PG) Data to an IND 6--19
- Appendix B: Submission of Pharmacogenomic (PG) Data to a New NDA, BLA, OR Supplement 6--21
- Appendix C: Submission of Pharmacogenomic (PG) Data to an Approved
- NDA, BLA, OR Supplement 6--23
- Appendix D: Quick Reference on Pharmacogenomic Submissions 6--24
- Appendix E: Voluntary Submission Cover Sheet 6--25
- References 6--26
Chapter 7: Intellectual Property: Compelling Filings By Large And Emerging Players 7--1
- Overview 7--1
- Strategic Considerations & Implications of Patenting 7--1
- Pay close attention to the specific scope and value of claims and how those relate to
- your business model and strategic plan. 7--2
- Ensure the IP issued is central to the value for the customer. 7--2
- Use of NIH federal funds in the research of a product. 7--2
- Evaluate your company's freedom to operate. 7--2
- Trade secrets. Internal value: Yes. External value: Sometimes. 7--2
Chapter 8: Survey Results 8--1
- Summary of Market-Based Survey Results 8--1
- Survey Results 8--1
TABLE OF EXHIBITS
- Exhibit 1.1 Cost of Clinical Drug Development 1--4
- Exhibit 1.2 Avoidable Cost of $177.4B of Drug-related Morbidity and Mortality 1--5
- Exhibit 1.3 Variance Analysis of Increased Cost Originating from Drug-related Morbidity and Mortality 1--6
- Exhibit 4.1 Percentage Price Premium Consumer is Willing to Pay for Tailored Drugs 4--5
- Exhibit 4.2 Diagnostics Versus Therapeutic Value Proposition 4--9
- Exhibit 4.3 Molecular and Pharmacogenomics Diagnostics Players 4--10
