Abstract
Introduction
Pharmacogenomics (PGx) is an evolutionary-not a revolutionary-process. The
"blockbuster" business model is, of course, still relevant to the
pharmaceutical industry, but at the same time, companies must adopt
technologies that will enable them to develop better drugs-value-added drugs
that can command the prices capable of providing an acceptable return on
investment for the companies that develop them. Many companies, pharmaceutical
and diagnostic alike, have come to understand that they need to use PGx to
their advantage, a step that requires changing the status quo of drug
development to produce targeted medicines together with diagnostic tests.
Get the Answers You Need to Shape Your Strategy
- The development of targeted medicines and PGx-based testing has been
slower than anticipated, and the promise of PGx is still largely to be
realized. Why? What is the key to unlocking the enormous potential of
personalized drug development?
- The industry needs to implement technologies that can be used to produce
safer and more effi cacious drugs. What are the challenges associated with
embracing the PGx model and the codevelopment of targeted drugs and their
companion diagnostic tests (RxDx codevelopment)? How will PGx improve this
process? Why is collaboration between pharmaceutical and diagnostic companies
so important?
- The pharmaceutical, diagnostic, and regulatory communities need to work
together effectively to develop more personalized medicines. What are the
bottlenecks delaying these interactions? What still needs to be done to make
this process work better?
- Pharmaceutical companies have come to recognize the current value and
future commercial potential of diagnostics, but they need a path to follow for
integrating PGx into drug development. How does a company begin? What is a
reasonable action plan to follow, and why is educating stakeholders so very
important?
Scope
- Industry experts: Twelve interviews and/or commentaries from
industry leaders who share their insights and experiences in implementing
personalized drug development and PDx testing.
- Targeted drugs and companion diagnostics: "personalized medicine"
targets the right patient for the right medicine; updating drug labels with
PGx information; the role of regulators in personalized medicine; complexities
in the underlying biology.
- Building a business case for RxDx codevelopment: a cultural change
for the industry; bottlenecks and stumbling blocks; evolving relationships
between big pharma and diagnostics companies; return on investment;
cost-benefi t analysis; clinical trial effi ciencies, value-added drugs;
raising the safety/ effi cacy bar.
- Pharmacodiagnostic test development: biomarker discovery and
research; CLIA-regulated versus FDA-approved tests; pricing/reimbursement
environment; CPT codes; clinical validity and clinical utility.
Table of Contents
- Executive Summary
- Strategic Considerations
- Stakeholder Implicationss
- The Pharmacogenomics Advantage
- The Diagnostic Component
- Expert Commentary -Building a Business Case for RxDx Codevelopment
- Need for Stakeholder Education
- Outlook for RxDx Codevelopment
- Expert Commentary -Establishing a Pharmacodiagnostics Model
- Evolving Pharmaceutical Industry Dynamic
- PDx Development
- Steps to Get Ahead
- Expert Commentary -Producing Targeted Drugs: A Cultural Change for Industry
- Cultural Change
- Using PGx: Cost and Time Savings
- Four Changes That Must Be Made
- Expert Commentary-Getting Ahead with Personalized Medicine One Step at a
Time
- A New Era of Drug Development
- HIV Coreceptor Tropism Test and CCR5 Antagonist Therapy Codevelopment
- Multiplying Targeted Markets
- Impact of Pricing/Reimbursement
- Personalized Medicine
- Expert Commentary -We Have an Obligation to Always Think About Markers
- New Tools for Decision Making
- Clinical Development
- Diagnostic Tests
- Other Industry Stakeholders
- Expert Commentary -Encouraging Personalized Medicine Without Inhibiting
Its Development
- The Role of Regulators
- Raising the Effi cacy/Safety Bar: Effect on PGx Uptake
- Medical Education
- RxDx Codevelopment
- Expert Interviews: Matters to Be Resolved
- Clinical Validity and Clinical Utility
- Cost-Benefi t Analysis
- Test Codes/Reimbursement
- Labels: A Clearer Direction for PGx Use
- CLIA-Regulated vs. PMA-Approved Tests
- Ten-Point Action Plan for Getting Ahead with PGx
- Moving Ahead
Experts Featured:
Mara G. Aspinall, M.B.A., President, Genzyme Genetics; Trustee, Dana-Farber
Cancer Institute Michael P. Bates, M.D., Vice President, Clinical Research,
Monogram Biosciences, Inc Rolf Ehrnström, M.Sc., Corporate Vice
President, Research & Development, Dako A/S Felix W. Frueh, Ph.D., Associate
Director for Genomics, Offi ce of Clinical Pharmacology, Center for Drug
Evaluation and Research, U.S. Food and Drug Administration Peter Keeling,
M.A., CEO, Diaceutics Klaus Lindpainter, M.D., M.P.H., Head, Roche Genetics,
and Director, Roche Center for Medical Genomics Marisa Papaluca, M.D., Deputy
Head of Safety and Effi cacy of Medicines, European Medicines Agency Christos
J. Petropoulos, Ph.D., Vice President R&D, Virology, and Chief Scientific
Officer, Monogram Biosciences, Inc Munir Pirmohamed, Ph.D., Professor of
Clinical Pharmacology, the University of Liverpool; Chair, Pharmacogenetics,
National Health Service Carol Reed, M.D., Executive Vice President and Chief
Medical Offi cer, Clinical Data, Inc
Additional interviews with staff members at the Secretary' s Advisory Committee
on Genetics, Health, and Society (SACGHS) and a program leader at a European
molecular diagnostics company
Expert Commentaries:
- Building a Business Case for RxDx Codevelopment
- Establishing a Pharmacodiagnostics Model
- Producing Targeted Drugs: A Cultural Change for Industry
- Getting Ahead with Personalized Medicine-One Step at a Time
- We Have an Obligation to Always Think About Markers
- Encouraging Personalized Medicine Without Inhibiting Its Development
Tables:
- 1. Select Targeted Drugs and Their Companion Diagnostics
- 2. Effect of HER2 Testing on the Development of Herceptin
- 3. Drug Marketability and Value: A Larger Share of a More Segmented Market
- 4. Public-Private Consortia
- 5. FDA Publications and Guidance Documents Relating to Pharmacodiagnostics
in Drug Development
- 6. PGx Markers: Valid Genomic Biomarkers in the Context of Approved Drug
Labels
- 7. Select Biomarker Discovery Companies, Their Technologies, and
Pharmaceutical Alliances
Figures:
- 1. Drug Decision Making Based on Pharmacogenomic Testing
- 2. Diagnostic Assay Development in Concert with Drug Development
- 3. Select Therapy Areas: Patient Response Rates to Major Drugs
