9.15

Examining the economics of stratifying patient populations and launching companion diagnostics

• What do recent case study-based models tell us about the new market realities and the potential impact on your business model?
• Satisfying regulators and payers in simultaneous clinical development of therapeutic and diagnostic
• The new commercialization and market adoption dynamics of stratified medicines
• Stratified medicine is not inevitable. Achieving its potential requires reasonable economics and decisions by all

Mark R. Trusheim, Executive in Residence & Visiting Scientist, Massachusetts Institute of Technology Sloan School of Management; President, Co-Bio Consulting LLC

How can 'personalized medicines' make the transition to licensed products in the eyes of physician, healthcare assessor and regulator?

9.35

Physician's perspective

Dr José Baselga, Chief, Division of Hematology/Oncology & Associate Director, Massachusetts General Hospital Cancer Center

9.55

AHRQ perspective on practical and policy aspects of personalized medicine moving forward

• How will comparative effectiveness research impact aspects of personalized medicine such as genetic testing?
• How can individual biology, environmental and other factors bee assessed to ensure the right treatment to the right patient at the right time?
• What are the research and data infrastructure needs to achieve this going forward?
  

Jean R. Slutsky, Director, Center for Outcomes & Evidence, Agency for Healthcare Research & Quality (AHRQ), US Department of Health & Human Services

10.15

FDA perspectiveHow is the FDA addressing regulation of targeted therapeutics with companion diagnostics?

• The path to personalized medicine
• Drug-diagnostic co-development
• Oversight of laboratory developed tests

Dr Abraham Tzou, Division of Immunology & Hematology, Office of In Vitro Diagnostic Device Evaluation & Safety, The Food & Drug Administration

10.35

Industry perspectives & public-private sector roundtable discussion
How can companion diagnostics be more effectively and efficiently co-developed and commercialized?

• What is the model for successfully commercializing Rx/Dx products?
  • How do you value your product?
  • How to reconcile the differences between commercialization models for therapeutic and diagnostic?
  • How will they integrate with the IT aspects of targeted healthcare? (Eg with Electronic Patient Records)
  • How should the industry approach the education of the physician and patient communities moving forward to ensure the widespread adoption of Rx/Dx products?
• What should the modern drug-diagnostic development organization look like?
  • How is the fundamental oncology R&D organization changing to allow for an increased focus on stratified patient populations?
  • How do you decide on the best timing with regard to the co-development of therapy and diagnostic?
  • Which is the current optimal regulatory pathway for launching your diagnostic test alongside your drug?
• Evaluating the diagnostics toolbox: What will be needed at each stage of R&D moving forward?

Panellists:
Dr Alan Barge, Vice President, Head of Clinical Oncology, AstraZeneca
David M. Epstein, PhD, Senior Vice President Oncology, Chief Scientific Officer, OSI Pharmaceuticals, Inc
Dr Gary J. Kelloff, Special Advisor, DCTD, CIP, National Cancer Institute
Jens Oliver Funk, MD, Senior Vice President, Merck Serono R&D, Global Head of TA Oncology, EMD Serono, Inc

11.20

Morning coffee in the exhibition area

12.10

How can the oncology R&D community drive the migration away from the single pathway/somatic mutation approach?

• How are novel enabling technologies (eg in silico, imaging) facilitating the search for synergies between compounds/molecules at the bench?
• What are the keys to translating these insights successfully into the clinic?

Dr Mike Burgess, Head, Oncology Early Development Group, Roche

Case studies: Examining R&D strategies focused on the development of two or more unlicensed compounds

• What is the optimal development strategy, given uncertainty with regard to both the regulatory environment and the swiftly evolving marketplace?

12.30

Rationally combining investigational agents

Dr Louise B. Grochow, Executive Director of Global Medical Science, AstraZeneca

12.50

Questions & discussion

12.55

A pathway-directed approach to combination therapy with unapproved targeted agents

Dr Stuart Lutzker, Vice President, BioOncology Exploratory Clinical Development, Genentech, Inc

1.15

Questions & discussion

1.20

Buffet lunch in the exhibition area

2.30

Case study

• When should co-development of 2 unlicensed drugs be considered?
• What technical and regulatory hurdles must be overcome?
• What are the challenges of commercializing drug combinations in the currently evolving marketplace?

Dr Rafael Amado, Vice President, Head of Solid Oncology Medicines Development, GlaxoSmithKline

2.50

Questions & discussion

Case studies: How are developers designing and implementing their combination strategies in different therapeutic modalities?

• What studies will be necessary to demonstrate their effectiveness?
• Addressing issues of timing and sequentiality
• How do you adapt your trial design where different therapeutic modalities are being combined?
• How to best outline a dose-finding trial for oncology therapeutics in combination?

2.55

Next generation kinase inhibitor

David M. Epstein, PhD, Senior Vice President Oncology, Chief Scientific Officer, OSI Pharmaceuticals, Inc

3.15

Questions & discussion

3.20

Active immunotherapeutic: Sipuleucel-T, an autologous cellular immunotherapy for prostate cancer

• Introduction to prostate cancer
• Active immunotherapy of prostate cancer – step on the gas
• Countering negative regulation – take your foot off the brake

Dr David Urdal, Chief Scientific Officer, Dendreon Corporation

3.40

Questions & discussion

3.45

Afternoon tea in the exhibition area

12.25

Questions & discussion

12.30

Preclinical models and tools for early-stage stratification efforts - progress and perspectives

• Overview on different approaches to identify and characterize biomarkers in preclinical in vitro and in vivo models
• Translation of different biomarkers into clinical settings and developing decision criteria - challenges and opportunities
• Case studies on biomarker approaches as part of novel drug development programs
  

Dr Nicholas C. Dracopoli, Vice President, Biomarkers, Centocor R&D, Johnson & Johnson

12.50

Questions & discussion

12.55

The Reverse Feasibility Program: A novel approach to conducting oncology clinical research

• Cancer – incidence and impact
• Bio-pharmaceutical industry response to impact
• Challenges related to clinical research and oncology clinical research specifically
• What it means to the industry and patients
• Need for innovative operations to support innovative drug development
• Value Added – what to expect

Denise Deakin, President, Scimega Research

1.15

Questions & discussion

1.20

Buffet lunch in the exhibition area

2.30

Strategies for optimizing patient segmentation during development

• What are the strategic alternatives in evolving from 'retro-fitting' biomarkers to pivotal clinical studies to a more efficient prospective model?
  • What can be done 'in-trial' and how should you address the potential regulatory and commercial implications?

Roy Baynes, MD, PhD, Vice President, Global Development & Therapeutic Area Head Hematology/Oncology, Amgen, Inc

2.50

Short presentation & panel discussion
Comparing and contrasting the various screening and sequencing tools and technologies now available: Which ones have real practical utility relating to patient stratification?

• What benefits can they actually deliver at both early and late stages of R&D today? What will they be able to deliver in five years' time?
• Are emerging high-throughput technologies going to be more useful than more targeted approaches in terms of their impact on R&D decision-making? What will people actually make out of the additional data?
• How far away is the '$1,000 sequence' in reality, and what will its impact be on the oncology drug development model?

Speaker: Dr Daniela S. Gerhard, Director, Office of Cancer Genomics, National Cancer Institute

3.45

Afternoon tea in the exhibition area

4.30

How can preclinical research help remedy the very high attrition rates for oncology therapeutic candidates entering the clinic?

• What can industry do proactively at the preclinical stage to provide insight into:
  • Drug target and compound mechanism of action?
  • Pharmacodynamic markers?
  • Appropriate patient populations?
  • Promising potential combinations?
  • Testable clinical hypotheses?

Dr Karen Zinkewich-Peotti, Vice President, Oncology Research, UCB

4.50

Questions & discussion

4.55

Short presentations & panel discussion
How to select your target indication? Examining IGF-1 receptors as an example

• What were the decision-making criteria for different companies?
  • What is their rationale and strategy in each case?
• How to weigh up the pros and cons of focusing on the investigation of a particular biomarker versus pursuing a pathway to regulatory approval?
  • Can we live with biomarkers for internal decisions without always wondering if the regulatory authorities will accept them?

Panellists:
Dr Robert A. Kramer, Vice President, Oncology & Immunology Drug Discovery, Bristol- Myers Squibb
Dr Markus Warmuth, Executive Director, Head of Oncology Drug Discovery, Cambridge, Novartis Institutes for BioMedical Research

5.35

Panel discussion
Reassessing the major unmet needs and 'undruggable' targets in oncology: What sort of collaborations/consortia involving industry, academia and other stakeholders could deliver breakthroughs, and what would be the role of each party in enabling such activities?

• What are the utility and future of such collaborations and what specific elements should each party take ownership of?
• How to address the disconnect between each party's goals and expectations?

Panellists:
Wayne A. Rosenkrans, Jr, PhD, Distinguished Fellow, MIT Center for Biomedical Innovation; Program in Ethics and Systems Medicine, Georgetown University; Chairman, Personalized Medicine Coalition; Vice President Strategic Consulting, Fuld and Co; Chief Scientific Advisor, Expertech Solutions; Chief Applications Officer, SciTech Strategies
Dr Karen Zinkewich-Peotti, Vice President, Oncology Research, UCB

6.00

Close of day 1, followed by a themed cocktail reception, sponsored by KEW Group, in the exhibition area

8.55

Presentation reserved

Judgement of value and the reward for innovation: How are the perspectives and practices of payers/healthtech evaluators evolving with regard to the assessment, pricing and reimbursement of novel therapeutics, and in terms of improving healthcare standards in general?

• How do we define 'value' in oncology, CNS and other therapeutic areas? Will there be a long-term trend towards value-based reimbursement on a global basis?
• How are we viewing methods/considerations for reducing the cost of delivered care (eg. comparative effectiveness research)?
• How can payers make best use of their data streams externally? How can biopharma benefit?
• What role will real life studies play in reimbursement?

9.15

Payer's perspective

Speaker to be announced

9.35

Questions & discussion

9.40

Healthtech evaluator's perspective

Dr Naomi Aronson, Executive Director, Technology Evaluation Center (TEC), BlueCross & BlueShield Association

10.00

Questions & discussion

10.05

Industry keynote address
How can the industry make the case in R&D for the reimbursement of premium-priced medications in an increasingly cost-averse environment?

Jay P. Siegel, MD, Chief Biotechnology Officer & Head, Global Pharmaceutical Regulatory Affairs, Johnson & Johnson, Pharmaceuticals

10.25

Questions & discussion

10.30

Roundtable discussion
Comparative effectiveness: How might it be employed and what will be the implications for, and the impact on, drug development models?

• What will be the key differences between US and European payers’ incorporation of comparative effectiveness research?
  • What impact will it have on reimbursement?
  • What impact will it have on milestones in deal making / licensing?
    • How will it be applied?
• How does clinical development play in to healthcare authorities, and how can the industry provide the sorts of outcomes-related data that will satisfy them?
  • What comparative trials does it make sense for pharma and biotech companies to run?
  • How to evaluate health economic outcomes earlier in development?
• How to approach the future with regard to fast-followers in oncology?

Panellists:
Roy Baynes, MD, PhD, Vice President, Global Development & Therapeutic Area Head Hematology/Oncology, Amgen, Inc
Dr Michael Krams, Vice President - Head of Neurology Franchise, Johnson & Johnson
David Weiner MD, Senior Medical Director, Head of Early Clinical Development,
Neurology Global Clinical Development Unit, EMD Serono, Inc

11.10

Morning coffee in the exhibition area

Big pharma and big biotech perspectives: What are our short-, mid- and long-term strategies for replenishing our oncology pipelines?

11.55

Big pharma perspective
Which technologies and indications will we focus on, and how are our oncology R&D organizations and core capabilities evolving as a consequence?

• How can criteria for selecting new targets improve success rates in the clinic?
• Technologies in target ID, validation and stratification – promises and pitfalls
• Internal vs. external target identification – how much do we need to do?

Dr Markus Warmuth, Executive Director, Head of Oncology Drug Discovery, Cambridge, Novartis Institutes for BioMedical Research

12.15

Questions & discussion

12.20

Big pharma perspective
Overcoming the challenges in balancing internal and external innovation in the oncology R&D pipeline

• Examples of collaborations with Exelixis, Adnexus and Medarex - illustrating the challenges and opportunities of balancing internal and external innovation

Dr Robert A. Kramer, Vice President, Oncology & Immunology Drug Discovery, Bristol- Myers Squibb

12.40

Questions & discussion

12.45

Big biotech perspective
Biogen Idec’s approach in oncology: Internal vs external development

• Core capabilities
• Criteria for external opportunities
• Examples of key partnerships and collaborations

Elaine Caughey, Senior Director, Business Development, Biogen Idec

1.05

Questions & discussion

1.10

Buffet lunch in the exhibition area

OR

Lunch workshop
(Highly interactive optional session for a maximum of 50 participants)
Sponsored by

Comparative effectiveness: Strategies, trends, the impact on healthcare reform and the tools to empower key stakeholders in informed decision-making

Speakers:
Wendy Hamilton, Senior Vice President, Product Strategy & Operations, Healthcare & Science, Thomson Reuters
Tami L. Mark, PhD, MBA, Director Analytic Strategies, Healthcare & Science, Thomson Reuters

» Click here for more information

Case studies: Exploring R&D strategies for key novel target areas/novel modalities with the marketplace of the future in mind

• What steps are being taken to provide the data that healthtech evaluators, physicians and patients will want to see moving forward? How are we preparing to demonstrate comparative effectiveness?
• What are our expectations for the shape and value of the marketplace for each respective technology/modality/indication at the projected time of regulatory approval, and how is this being translated into risk and cost control measures in R&D?
• What clinical trial designs are we using and why? What is the latest clinical data?
• What aspects of global strategy are we utilizing? What are the drivers behind this, what challenges are we facing and how are we overcoming them?

2.20

Issues and challenges in the development of PARP-inhibitors for BRCA-related cancers

• Background to DNA repair failure in cancer
• Early pre-clinical and clinical data
• Future perspectives

Dr Alan Barge, Vice President, Head of Clinical Oncology, AstraZeneca

2.40

Antibody drug conjugates: Changing the paradigm for early drug development in cancer

• Can we expect efficacy during phase 1 trials?
• Will companion diagnostic tests be the rule, rather than the exception?

Speaker to be announced

3.00

Discovery and early development of XL184, an oral TKI targeting MET and VEGFR

• Biological advantage of dual targeting approach for MET and VEGFR
• Development of pharmacodynamic biomarkers during discovery for use in clinical studies
• Clinical development strategy based on strong biological rationale and broad signal-searching efforts

Dana Aftab, PhD, Senior Vice President, Translational Research, Exelixis, Inc

3.20

Drug targeting of cancer stem cells

• Where do cancer stem cells really exist to be targeted?
• What targets might select cancer stem cells from normal stem cells?
• What outcomes might we expect if we are successful?

Dr Stephen M. Kelsey, Executive Vice President & Chief Medical Officer, Oncology, Geron Corporation

3.40

Afternoon tea in the exhibition area

12.15

Questions & discussion

12.20

Case study
How do you translate data from late-stage/marketed drugs that work in a given pathway back to early R&D to look for synergies with other products and/or to inform development of early-stage products targeting the same (or a similar) pathway?

• What can we learn from drugs on the market?
• How broad (technology-wise) do we have to start biomarker exploration at discovery stage
• How to best utilise tissue samples for translational research
• Determine the quality of the biomarker assay dependent on the clinical stage of the program
• Strategic collaboration between Roche Pharma and Diagnostics beyond the pure project level

Dr Gerd Maass, Global Head, Biomarker & Pathway Analysis, Translational Research Sciences, Roche Pharma Research & Early Development

12.40

Questions & discussion

12.45

Panel discussion
Phase 0 trials in oncology: How useful are they in practice?

• What can/should they be used for?
  • Are they really a worthwhile substitute for a phase I?
  • What are the pros and cons?
  • Are they really informative, or just adding a little refinement to what you basically already know?
• What are PD biomarkers really worth in targeted oncology therapeutic R&D?
  • Should industry continue to spend money on them and if so, what do they expect to get out of it?
    

1.05

Questions & discussion

1.10

Buffet lunch in the exhibition area

OR

Lunch workshop
(Highly interactive optional session for a maximum of 50 participants)
Sponsored by

Comparative effectiveness: Strategies, trends, the impact on healthcare reform and the tools to empower key stakeholders in informed decision-making

Speakers:
Wendy Hamilton, Senior Vice President, Product Strategy & Operations, Healthcare & Science, Thomson Reuters
Tami L. Mark, PhD, MBA, Director Analytic Strategies, Healthcare & Science, Thomson Reuters

» Click here for more information

2.20

Tumor sampling/banking: Which clinical materials can/should you use in preclinical (eg. for broad screening to predefine characteristics of the tumor, and of given pathways)?

• How do you then leverage these findings in the early phases of development?

Dr Timothy Yeatman, Executive Vice President, Co-Founder & Chief Scientific Officer, M2Gen

2.40

CardioOncology - cardiac safety considerations in oncology drug development

• With the development and ratification of the ICH E14, cardiac safety, specifically QT prolongation has become a primary cardiac safety endpoint in the drug development process. Historically, indications such as oncology have benefited from higher regulatory tolerance levels based on the indication and an overall favorable benefit-risk considerations
• To assist in defining the concept of benefit/risk ratio within oncology, if a compound is efficacious, but also generates a prolongation of the QT interval, the benefit of the compound in treating the disease, outweighs the risk of TdP or sudden death associated with QT prolongation. However, more recently this view has been challenged by the FDA and other regulatory agencies, following high profile reports of oncology drug related cardiotoxicity, primarily with tyrosine kinase inhibitors (TKI) and monoclonal antibodies directed against TKI receptors. This highlighted the responsibility of the pharmaceutical and biotech sponsors to properly define the QT and other cardiovascular safety liabilities during oncology drug development
• This session will provide an overview of cardiac safety within oncology compound development including case study review and perspective from representatives from pharma/biotech organizations

Dr Jeffrey Heilbraun, Senior Manager, Scientific Affairs, CoreLab Partners, Inc

3.40

Afternoon tea in the exhibition area

5.15

Assessing the ongoing progress of cancer genome projects - what benefits and challenges for the oncology R&D community can be expected to emerge from the data?

• What key gaps in our knowledge and understanding of the data's utility will remain?
• Examining issues regarding the accessibility of cancer genome data for pharma/biotech R&D

Dr Matthew L. Meyerson, Professor of Pathology, Dana-Farber Cancer Institute

5.35

Questions & discussion

5.40

Close of day 2, followed by a themed cocktail reception, sponsored by The Institute for Neurosciences at Brigham and Women's Hospital, in the exhibition area

9.05

Industry perspective
The oncology clinical development model cannot operate efficiently in the current regulatory environment: Where do we go next?

Dr Jean-Pierre Bizzari, Senior Vice President, Clinical Oncology/Hematology, Celgene Corporation

9.25

Case study
Sharing risk in cancer drug development - the Cancer Research UK clinical development partnerships model

• Taking on the early development of de-prioritized agents
• Sharing risk in cancer drug development
• Delivery of early phase clinical trials through a network of Experimental Cancer Medicine Centers

Dr Rob Williams, Head of Nonclinical Operations & Chief Development Scientist, Cancer Research UK

9.45

Short presentation & panel discussion
What can be done better pre-phase IV in oncology?

• How can we improve how we examine results (what we look for and when) of earlier phases to improve the prospects of phase IV trials?
• How to go about collecting and using data that may be required for your risk mitigation program pre-phase III?
• Is accelerated approval really worth it in light of issues around post-approval commitments?

Dr Nancy Simonian, CMO, Millennium: The Takeda Oncology Company

10.15

Monitoring reviewer metrics during blinded Independent Central Review (ICR)

• The causes of discordance between radiologists during blinded independent central review for oncology clinical trials
• The monitoring of adjudication rates using statistical modeling
• The monitoring of reviewer performance on a corporate-wide and protocol-specific basis

J. Michael O'Neal, MD, Chief Medical Officer, CoreLab Partners, Inc

10.35

Panel discussion

• What has changed in the oncology clinical development model/process over the past 5 years and what does the future look like if we extrapolate forward?
• How have recent clinical trial results and drug approvals affected the regulator's view of progression-free survival?
  • Will overall survival become a more attractive endpoint to the industry?

Panellist:
Dr David Urdal, Chief Scientific Officer, Dendreon Corporation

11.05

Morning coffee in the exhibition area

FOLLOWED BY YOUR CHOICE OF 2 PARALLEL SESSIONS:

12.05

Statistical design strategies for de-risking oncology drug development

• Making the best use of exploratory studies for early development decision-making
• Maximizing the probability of success of your registration trial through novel statistical design

Yannis Jemiai, PhD, Director of Biostatistics, Cytel Inc

Case studies: Analyzing practical examples of novel adaptive clinical trial designs in oncology

• What benefits are they delivering in practice in terms of minimizing costs, timelines and patient exposure?
• How to make the statistics 'user friendly'?

12.25

Bayesian designs for phase I

Dr Jeffrey Helterbrand, Global Head of Biostatistics, Genentech, Inc

12.45

Continuous reassessment dose selection/escalation trials

Jerald S. Schindler, DrPH, Vice President, Biostatistics & Research Decision Sciences - Late Development Statistics, Merck Research Laboratories

1.05

Questions for the speakers & panel discussion

• How to get into phase III by the shortest possible path and with the highest likelihood of success?
  • Are more randomized phase II trials the answer?
• What is the regulator's current view on the application of adaptive trial designs in oncology?
  • What role (if any) is there for aggressive adaptive phase III trial designs in oncology
    moving forward?

1.35

Lunch & close of the Phacilitate Oncology Leaders' Forum 2010

11.50

Case study
How have competitive companies overcome IP-related issues to form collaborations around the development of novel therapeutic candidates in combination?

Dr Joan A. Lasota, Senior Director, Alliance Management, Merck & Co, Inc

12.10

Questions & discussion

12.20

Experience in building Public-Private Partnerships - presentation of case studies

• The value proposition for each stakeholder
• The synergy obtained by bringing together predictive biomarkers, targeted therapies, imaging and adaptive designs
• Data sharing and IP sorting models

Dr Gary J. Kelloff, Special Advisor, DCTD, CIP, National Cancer Institute

12.40

Questions & discussion

Exploring collaborations between both large and small pharma/biotech companies and diagnostic companies: How to integrate a molecular diagnostics function into a drug company through effective partnering?

• What resources are required in-house to enable this?
• How were the deals structured in terms of risk-sharing?
• How much efficiency can you really gain through such collaborations?
• What's the right timing to initiate them?

12.45

Emerging partnership models for CDx

• Abbott Molecular has signed more than 5 CDx deals with large Pharmaceutical companies in the last two years. This presentation will cover the assumptions that underpin CDx agreements and the challenges that have to be overcome to bring a companion product to the marketplace with a focus on regulatory, logistics, reimbursement and commercial. The presentation also outlines a model to facilitate success in the US market

D. Stafford O'Kelly, President, Abbott Molecular Diagnostics

1.05

Questions & discussion

1.10

Improving patient outcomes - the Novartis Molecular Diagnostics (MDx) approach to partnering

• Today's clinical practice in oncology is shifting towards targeted therapies, as access to improved technologies has profoundly transformed the drug R&D process
• At Novartis, we are addressing unmet medical need through the development of new products for both common and rare forms of cancer based on our understanding of the science, the disease and the drug target pathways
• As an independent unit within Novartis Pharma, Novartis MDx leverages its integrated diagnostics capabilities in order to:
  • Increase the value of our medicinesby delivering companion diagnostics
  • Develop"game changing" tests that significantly improve clinical decision making and patient outcomes
• This talk will explore some of the partnering and collaboration models through which Novartis MDx is aiming to transform the way medicine is practiced today

Dr Yves Dubaquie, Director, Business Development & Licensing, Novartis Molecular Diagnostics

1.30

Questions & discussion

1.35

Lunch & close of the Phacilitate Oncology Leaders' Forum 2010