Day 2

Tuesday, June 14, 2005

7:45 Morning Coffee

Technological and Diagnostic Perspectives

8:15 Chairperson's Opening Remarks and Roundtable Report Outs

8:45 Moving Microarrays into the Clinic - The Need for Standard Controls and Clinical Guidelines
Dr. Janet A. Warrington, Vice President, Emerging Markets and Molecular Diagnostics Research and Development, Affymetrix, Inc. 
Success in moving Affymetrix GeneChip® technology into clinical and diagnostic laboratories requires building a research and development foundation in multiple areas including standardization across industry, development of clinical guidelines via accredited processes, satisfaction of clinical and diagnostic customer requirements, driving technology into new applications areas, generation of validation data, diagnostics suitable platform delivery, influencing regulatory approval for each intended use as well as certification for array and instrument manufacture. In this presentation Dr. Warrington will describe the approaches Affymetrix is taking to achieve these goals including, strategic objectives, key projects, partnerships and collaborations. 

9:20 Strategies for the Creation of Partnerships - Predicting and Overcoming Challenges in Creating Drug Response Profiling Diagnostics 
Angus Hastie, Director, Global Marketing and Business Development, Roche Molecular Diagnostics
Partnership Genesis - aligning disparate Rx/Dx business principles to initiate the contract process for a successful outcome 
• The bottom-line numbers. What is the value of incorporating a diagnostic? If early? If post-approval? 
• Strategies for collaborative Rx/Dx programs with Pharma - avoiding the "submarine" diagnostic 
• Applying translational research from prototype assays to optimized, market-compatible tests 
• Incorporating optimized, scalable tests into pivotal regulatory trials 
• Gaining early market penetration with screening tests; pulling new drug choice through diagnostic metrics

9:55 Networking Coffee Break

10:25 The Rx/Dx Paradigm - Diagnostic and Genetic Technologies
Douglas E. Onsi, Vice President, Business Development, Genzyme Corporation
With the approval and success of Herceptin, the path has been blazed for a new generation of targeted therapeutics that are indicated and approved in conjunction with a diagnostic test. This presentation will review the Herceptin story with the comparison of what would have happened to the drug without the diagnostic test. Then the presentation will include some background information on the diagnostic and genetic technologies that are available to be used to diagnose cancer, such as FISH, IHC and DNA sequencing. Finally, the presentation will discuss some products in development in for which a diagnostic test is either being used as part of the clinical trial strategy or should be used based on the emerging genetic and clinical data. The presenter predicts that diagnostic testing will become a normal part of the clinical and marketing strategy for oncology drugs to increase the likelihood of clinical success in terms of increased efficacy and reduced side effects and to enhance marketing success in terms of market penetration and premium pricing over less well defined agents.

11:35 Best Practice for Diagnostics
Interactive Panel Discussion With Session Speakers

Regulatory Applications and Options

1:35 Chairperson's Remarks
Mr. Thomas Soriano, President and & CEO, Diagnostics Oncology CRO Inc.  

1:45 Clinical Applications of Genomics: The Use of Evidence Based Frameworks by Decision-Makers
Dr. Gurvaneet Randhawa, Senior Service Fellow, Center for Outcomes and Evidence, Agency for Healthcare Research and Quality 
All healthcare interventions (diagnostic or therapeutic) have potential benefits and harms. Decision-makers assess whether the magnitude of potential benefits of using a new intervention outweighs that of harms. An evidence-based framework helps in identifying the relevant outcomes of interest and ascertaining the strength of evidence for these outcomes. This talk will provide a brief overview of the potential applications of gene-based technologies in clinical prevention (primary and secondary) and disease management and discuss an evidence-based approach to clinical decision-making using examples of recommendations on clinical preventive services from the U.S. Preventive Services Task Force (USPSTF). 

2:20 Internal Impact on R&D Strategies: Using Cost Effective Solutions to Make Clinical Go/No-Go Decisions 
Dr. Melissa Paoloni, DVM, DACVIM, NIH/NCI, Comparative Oncology Program
Cancer drug development is a costly, linear and inefficient process. The two most common causes of drug failure are toxicity or lack of efficacy. These failures are most costly once a drug enters phase I human trials but are even more costly if they occur after phase II trials, and beyond. It is therefore essential that "go no go" decisions focus on the issue of toxicity and efficacy as early in the development path as possible. An opportunity to better inform the drug development path may emerge through the use and integration of relevant non-clinical systems for evaluating new agents into the later steps of the drug development path. The use and integration of companion animals with cancer into the development path of new human cancer drugs is receiving greater attention in the pharmaceutical industry, by academic groups, and regulatory bodies. The new paradigm of cancer drug development that includes naturally occurring cancer models in "go-no-go" decisions will reduce costs of drug development by providing earlier signals of toxicity and efficacy and by informing the design of human trials such that active agents will be evaluated with the greatest opportunity 
for success. 
• Overview of obstacles involved in traditional non-integrated drug development
• A new paradigm: An integrated and comparative approach to drug development
• Examples and cost-benefit analysis of this integrated drug development approach

2:55 Orphan Drug Designation and Pharmacogenomics: Options and Opportunities 
Dr. Marlene E. Haffner, MD, MPH, Director, Office of Orphan Products Development, FDA 
The Orphan Drug Act was designed to offer benefits and encourage drug development in areas of small opportunity and great need. With the knowledge obtained from the Human Genome Project, personalized medicine is becoming more of a reality, and different markets are becoming available to take the best advantage of the knowledge gained. As our knowledge base increases, more Targeted Therapies will become available, many of which will be orphan products. The Office of Orphan Products Development is beginning to see applications based on pharmacogenomics.
Please join Dr. Haffner as she discusses:
• The opportunities and benefits of Orphan Drug Designations
• The impact of pharmacogenomics on Orphan Drugs
• Case examples of Targeted Therapeutics presented for Orphan Drug Designation

3:30 Afternoon Coffee Break

5:00 Conference Wrap - Up 

5:15 Close of Summit