Welcome to Leaders' Forum

Oncology Leaders' Forum 2009 | Monday, November 9th, 2009
Agenda | Day 1

Morning plenary session
(to be shared with the R&D Leaders' Forum 2009)
Proving the value of R&D innovation in an era of healthcare reform and financial stricture

Analyst's perspective: What sort of prices will the market support moving forwards, especially for traditionally expensive targeted small molecule drugs and biologics?
Public and private sector payers’ perspectives: How will health economics and reimbursement criteria continue to evolve under President Obama's administration?
- How will value be assessed?
- To what extent will an evidence-based medicine model be adopted and how high will the reimbursement hurdle be?
- Managing complexity: Will reimbursers pay different amounts for different outcomes for different indications?
Keynote industry perspective: The pharma and biotech industries are increasingly being asked to deliver more innovation and value for lower cost - How are our R&D strategies going to meet this challenge?
- What are our key initiatives for reducing R&D spend while maintaining/increasing innovation?
Multiple stakeholders' panel discussion & extended Q&A with the audience: What is the best way to conduct healthcare reform in order to preserve R&D innovation?
- What are the various potential scenarios for the US healthcare environment in the mid- to long-term
  - What will each mean for industry R&D strategists
  - Which model is most likely to prevail?
- What have been the pros and cons of NICE in terms of its impact on industry R&D innovation in the UK, particularly in the oncology field?
- What are the implications of a universal healthcare model on clinical trial costs and timelines?
- Is the industry's traditional value-generating model broken? What do we mean by value? What experimental models are being tested to better understand and defend the value of your drug?
  - Getting buy-in from payers much earlier in development – how early should the dialogue start and what form should it take?
  - Generating evidence at the R&D stage that will justify the economic value of the drug
    - Using public data to focus on the differentiating features of a product
- Comparative effectiveness in oncology: What benefit is worth reimbursement moving forwards, particularly in the context of adding to/replacing the Standard of Care?
  - Is survival going to be enough (and if so, how long?) or will curative treatments be the only reimbursable option?
  - What would the ideal model for oncology pricing and reimbursement look like?

Followed by your choice of two breakout sessions:

Focus session 1
Steering oncology R&D models down the path to competitive advantage, and to the required level of innovation at manageable cost
• How do you accurately define the value proposition of your oncology drug candidate at each stage of the discovery and development process?

Reviewing the key market approvals/failures and commercial stories of the past 12 months in oncology
- Market size aside, where are we seeing the greatest innovation in oncology?
Understanding the market: How quickly can one really change the Standard of Care by adding a targeted agent?
- Is the current model of seeking to add to, or replace an element of, the Standard of Care a viable long-term option for the sector?
  - What innovative approaches can one take in this regard?
- What can be done to better meet challenges facing novel-novel combination therapies?
Short presentation...
- Case study: What does the orphan approach to an oncology indication really look like in commercial terms?
...followed by extended discussion/Q&A with the audience
- How to leverage smaller oncology markets like kidney?
- How can these niche indications be used to drive innovation and market development?
Panel discussion & extended audience Q&A: Large, mid-sized and small pharma and biotech perspectives: Defining key target selection criteria in a rapidly changing world
- As markets/indications become more fragmented, how are the criteria and processes for valuing technologies in oncology R&D changing?
- What steps can you take to accurately assess at an early stage whether or not your drug candidate will have a viable path to regulatory approval and reimbursement?
- How should the increasingly critical aspects of product differentiation and labelling be integrated into R&D decision making at an early stage?
- How can biotechs make sure they are more product-oriented from the get-go?
  - How should they decide on the target indication for their platform technology?

Or | Workshop
Troubleshooting oncology clinical development on a global basis to overcome patient recruitment challenges in the Western hemisphere
• What are the pros and cons of accessing conducting trials in the US/Europe versus developing regions of the world?

Optimizing clinical trial designs and tactics to gain competitive advantage in the patient recruitment space on a global basis
- What is the optimal Phase II trial design in terms of efficiently accessing sufficient numbers of suitable patients?
- What designs have resulted in the highest and lowest success rates for subsequent Phase III trials?
- Capitalizing on potential collaborative strategies (eg. with patient advocacy groups and online patient affinity groups)
- What are the trade-offs in choosing a global clinical development program in terms of speed, quality of data and cost?
What is the US FDA's current view with regard to the acceptability of clinical data derived abroad?
- What are the specific criteria that must be met in order for the data to be deemed to be relevant in the US?
- Discussing issues of ethics and informed consent in international clinical trials
Case study: What steps may be taken to overcome the challenge of varying Standards of Care in different nations involved in an oncology trial, and the potential impact on the relevance of the data in the US and Europe?
Working party (Interactive panel & audience brainstorming session tasked with creating a 'road map' in 30 minutes
- Building a 2-3 tier clinical development model for a small biopharma company to share risk, speed development (as much as possible), and possibly bring in an international aspect, without compromising data quality

Then | Afternoon plenary session
Delivering alternative funding and co-development models to drive oncology R&D

Roundtable discussion & extended audience Q&A
- How and when should large companies get involved at early stages in biotech research? (Eg. through option agreements, big pharma/big biotech venture arm investment)
- What are the keys for biotech to capitalize on the increasing opportunities to develop products from big pharma portfolios?
- What do the economics of co-development in oncology look like?
  - How are partnering trends and strategies between pharma and biotech evolving in oncology R&D, and what are the keys to securing the optimal partner in an increasingly competitive environment?
  - Foundation funding: What are the potential issues/opportunties?
  - Defining the role of consortia and other public-private partnering models in achieving efficient, successful development of oncology therapeutics
    - What can be done by public and private partners alike to incentivize each other to enter such partnerships?

Oncology Leaders' Forum 2009 | Tuesday, November 10th
Agenda | Day 2

Morning plenary session
Targeting patients and therapies to reduce clinical trial sizes and late-stage attrition rates in oncology
• Optimizing the role of patient selection for activity, and of biomarkers in that patient selection

What are the interim signs of personalized medicine’s impact on attrition rates?
- How is big pharma integrating personalized medicine into its existing R&D structures/strategies?
- What evidence is there for personalized medicine generating better or worse net present values (NPV) and ROI for the pharma industry?
FDA keynote perspective: What are the priorities in the oncology area for the FDA under President Obama's administration?
- How has the Critical Path performed in reducing risk and cost in drug development to date and will existing concepts be modified?
- Evolving regulatory thinking on:
  - Endpoints for oncology trials
  - Required data to drive the regulatory validation of biomarkers
KRAS: A biomarker for our time? Driving the development and validation of biomarkers to predict patient response
- Multiple stakeholders' (pharma/biotech, diagnostic company, regulator, payer) short presentations, followed by panel discussion & extended audience Q&A
  - Why has KRAS impacted clinical practice so quickly and how typical will it prove to be in this regard?
  - Will it present an opportunity for the future, or is it just an associated epiphenomenon which will prove to be a red herring for future researchers seeking related markers?
  - How has the labelling changed and has this met the hopes and expectations of each stakeholder?
  - How should we discover and develop the "next KRAS"? Assessing progress in the development of the next generation of predictive biomarkers of response
  - What lessons can be/are being taken from the original?
  - How will the FDA approach the validation of future biomarkers where data is conclusive without the necessity for a randomized trial?
Updates on imaging biomarker validation trials
- What is the latest data from ongoing imaging consortia trials and data standardization initiatives?
- What are the pros and cons of RECIST 1.1 criteria in practice?

Followed by your choice of two breakout sessions:

Focus session
How are trial population enrichment and novel biomarker development strategies impacting clinical costs, timelines and the quality of go/no go R&D decision-making?

What are the components for deciding how many patients you are going to need, whether you will realistically enrol the right patients, and what a realistic clinical trial timeline would be?
Case studies: What is the latest progress with trial patient pre-selection and biomarker development for novel oncology therapeutics at various stages of development?
- Are these strategies impacting go/no go criteria through the phases in practice?
- How do you decide if your patient selection criteria are robust enough, and whether you can create a pathway to registration?
  - Translating preclinical biomarker-related insights into clinic candidates and trial design
  - Phase I/IIa
  - Phase IIb/III
    - How much data do you need from an oncology Phase II to get into pivotal trials with real prospects of success?
    - Is there really any value in randomized, single arm Phase II trials in oncology?
      - Do they provide any sort of worthwhile indication whether or not you should proceed to Phase III?
Short presentation...
- What biomarker platform(s) should one invest in?
  - Identifying and balancing the key criteria to be met
...followed by panel discussion & extended audience Q&A
- What type of platform is suited to a given indication/therapeutic approach?
  - Are there broader platforms that can cover all your needs across the portfolio, or do you need to piece together individual solutions to cover all bases: Which approach is optimal?
- What are the pros and cons of imaging in terms of its clinical utility in oncology, especially for Phase II dose selection and as a predictor of Phase III success?
  - Addressing the challenges in the standardization of imaging data and the integration of imaging/non-imaging data
- What are the latest breakthroughs in terms of discovering and developing biomarkers that can distinguish between the more and less malignant cancer cells?

Or | Workshop
Making the business case for personalized medicine: What will be the keys to ensuring reimbursement for, and a viable ROI from, novel therapeutics with companion diagnostics?

What are the latest developments in the terms of a regulatory pathway to licensure for companion diagnostics? What issues still need to be addressed?
- Regulator's perspective
- Industry perspective
Case study: Examining the latest clinical trial design strategies for novel oncology therapeutics with companion diagnostics
Co-presented case study: Dissecting a pharma-diagnostic company partnership model - What are the key obstacles to successful collaboration and what are the strategic solutions to making such relationships mutually beneficial from both sides of the deal?
- How to integrate the disparate corporate and R&D models involved?
Multiple stakeholders' roundtable debate & extended audience Q&A
- How will therapeutics with companion diagnostics be reimbursed?
  - Who will pay for them?
- Debating the correct apportioning of value between therapeutic and companion diagnostic components
  - What are the various industry (pharma/biotech and diagnostic) and public sector stakeholders’ (regulators, payers, physicians, patients) perspectives?
    - Where is the balancing point?
    - How does the money flow?

Then | Afternoon plenary session
Re-defining our fundamental approach to oncology target identification

Keynote address: How and when will oncology drug discovery migrate from its traditional focus on tumor type/location to a pathway-based model?
Panel discussion & extended audience Q&A
- What would be the key adaptations required to the current oncology R&D model to allow us to examine multiple pathways, and to use this as scientific hypothesis to guide the selection of therapeutic agents for combination?
- How will recently discovered phenomena such as circulating tumor cells impact R&D strategy moving forwards?
  - How will tumor microenvironment-targeting strategies develop?

Oncology Leaders' Forum 2009 | Wednesday, November 11th
Agenda | Day 3

Morning plenary session
Which new targets, technologies and scientific breakthroughs will drive oncology R&D over the coming decade and beyond?

In which specific oncology indications are we seeing advances?
- What progress is being made with the most difficult cancers (eg. pancreatic)?
What will follow monoclonal antibodies?
- Examining key novel therapeutic modalities: What is the data to support claims that they will provide the next breakthrough in oncology?
Short presentations & presentation-specific Q&A
- RNAI and other oligonucleotide therapeutics
  - Delivering early clinical trial designs and data: What's the state of the art and chances of success for the field, and who are the biggest players now?
  - How are delivery issues being addressed?
- Active immunotherapeutics: What lessons has the sector learned in recent years and how is the pathway towards licensure being shaped?
  - How will they fit into the reimbursement picture given the likely need to combine them with other agents
- Stem cells
- Antibody drug conjugates
- Stable peptides
...followed by panel discussion & extended audience Q&A
- What is going to fuel oncology discovery pipelines in 5 years time?
- Is it really time to move on from kinases?
  - Has the area been fully mined, or have we not yet explored deeply enough?
- How must big pharma look to work with academia moving forwards given the potential shortfall in technologies entering their drug discovery engines over the coming years?
  - Is it time for a fundamental rethink on the pharma-academia relationship? Does pharma need to start partnering more aggressively with academia now?
  - Will/can Government help with combined pharma/academic discovery projects? What is pre- and what is pro-competitive in oncology in reality, and how should this enable more fruitful interactions between pharma, biotech, academia and other stakeholders?

Followed by your choice of two breakout sessions:

Focus session
What is the clinical/regulatory model that allows oncology combination therapies to be successfully developed in a realistically cost-effective manner?

What are the key changes that can be made today to the traditional, monotherapy-based oncology R&D model to facilitate the efficient development of novel therapies that will be licensed and reimbursed as part of a combination?
- Addressing regulatory issues: What data will the regulators require for two unapproved agents?
- What would be the pathway for combination therapies incorporating a companion diagnostic?
Short presentation...
- Case study: How do you incorporate a combination strategy into the early stages of R&D?
...followed by extended discussion & Q&A with the audience
- Given the financial repercussions, what is the optimal timing for commencing this work for both pharma and biotech companies?
- What predictive enabling technologies can assist in decision-making regarding combination strategy in the translational phases?
Short presentations...
- Case studies: What are the latest trial designs and data for combination therapies in development? How are the challenges in proving both safety and efficacy being addressed?
...followed by extended discussion/Q&A with the audience
- What is the optimal process for deciding on the best combination for your product?
- How should you meet the challenges presented by combinations of chemical and biological agents?

Or | Workshop
Designing an R&D roadmap for a novel technology area: Cancer stem cells

Working party (Interactive panel & audience brainstorming session tasked with creating a 'road map' in 120 minutes)
- Given the economic, healthcare and R&D trends, issues and advances discussed over the previous 2 days, what should the full R&D process, from basic research through to pivotal trials, licensure and reimbursement, look like for a cancer stem cell therapeutic entering the pipeline in the near future? (Session ends with the Moderator's summary of the 'road map' and its key action points)

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