Accelerating Proof of Concept - Day 1


Monday, October 4


Short Course:*

8:30 Morning Coffee and Short Course Registration

9:30 - 11:45am Evidence Led Decision Making for Clinical Proof-of-Concepts

Instructors:

  • John Arrowsmith, Ph.D., Science Director, CMR International
  • Richard K. Harrison, Ph.D., Science Director, North America, CMR International

The R&D based Pharmaceutical Industry is currently faced with major issues around revenue generation and productivity. Consequently it has been actively adopting a variety of strategies, aimed at maintaining its long term viability as the provider of new breakthrough medicines to address unmet medical needs. Such strategies include managing cost by downsizing and outsourcing, increasing access to new drugs and technologies through partnering, acquisitions and mergers, bringing a greater internal focus on productivity through re-organizations into Business Units and Research Units combined with a narrowing of scope to fewer therapeutic areas and finally, to extract the maximum value from existing assets through investing in emerging markets, in drug repositioning, in generics/biosimilars and in diversifying the portfolio to encompass biologics/vaccines, consumer health, animal health and diagnostics.

These strategies are being adopted as means to maintain revenue streams and to mitigate some risk, but fundamentally they are being driven by the low output of new medicines from the R&D divisions of Pharma. Later in this paper some science led approaches to cracking this R&D productivity issue will be discussed.

Despite the huge increases in the investments made in Pharma R&D over the last decade or so, productivity, as measured by the NDA approval of new molecular entities, has remained fairly constant at around 20+ per annum. This level of productivity is not sufficient to sustain an R&D based pharmaceutical industry that relies heavily on NME’s to drive future revenue streams. Phase 2 survival is the Achilles Heel of R&D productivity since it has always been the phase with the lowest survival rate. Only about 1 in 4 drugs entered into a Phase 2 proof of concept study (POC) will successfully transition into Phase 3; and of these only about 50% become an approved drug. Companies that claims “improved or high productivity” by bolstering the size of it’s portfolio in Phases 1 and 2, have not addressed the real issues involved in delivering a positive POC and progressing those successes to Phase 3. The short course will discuss not only the key criteria that underpin quality PoC decisions, but also strategies that should be considered in achieving a PoC in a timely and cost effective manner.

*Separate Registration Required


12:30-1:25pm Main Conference Registration

1:25 Welcome Remarks

Kate Skaare, Conference Producer, Cambridge Healthtech Institute

1:30 Keynote Address: Getting to ‘No’: Accelerating Proof of Concept to Speed Development and Manage Risk

Kenneth I. Kaitin, Ph.D., Director, Tufts Center for the Study of Drug Development, Professor of Medicine, Professor of Pharmacology & Exp Therapeutics, Tufts University School of Medicine

Despite concerted efforts by the research-based industry over the years to reduce drug development costs and improve overall R&D efficiency, there continue to be an alarming number of development candidates that fail in late clinical testing, when costs and resource demands are at their greatest. This talk will review current drug development metrics collected by Tufts CSDD and address some of the new strategic and operational approaches to speeding the time to making informed go/no-go decisions.

BIOMARKERS & PHARMACOGENOMICS: EARLY PHASE DEVELOPMENT ACCELERATION

2:30 Chairperson’s Opening Remarks

Walter Janssens, Ph.D., Senior Preclinical Assessor, Preauthorisation, Coordinator Early Phase Development, Federal Agency for Medicines and Health Products

2:40 Risk Assessment in Translational Medicine: Algorithms or Gut Feeling to Prepare for Proof-of-Concept?

Martin Wehling, M.D., Managing Director, Institute for Experimental and Clinical Pharmacology and Toxicology, Director, Clinical Pharmacology Mannheim Medical Faculty, Mannheim Ruprecht-Karls-University Heidelberg

Essential conditions of risk assessment in translational processes:

  • Biomarker evaluation and development
  • Translational planning including early stage planning of phase 0 human studies
  • POC readout planning

Key messages from translational risk assessment, e.g. biomarker development strategies, animal test design in projection of early planning of PoP/C studies and the Critical role of translational toxicology will be discussed

3:10 Network Refreshment Break with Exhibit and Poster Viewing

3:55 Integrating Translational Medicine and Biomarkers in Optimizing the Value for Proof-of-Concept Studies

Jingsong Wang, M.D., Director, Discovery Medicine & Clinical Pharmacology, Bristol-Myers Squibb

4:25 Marching the Pharmacologic Audit Trail: Strategies for
Early Development Trials in Oncology

Roger Luo, Ph.D., Director, Translational Medicine & Early Development, Ortho Biotech Oncology R&D/Centocor R&D, Inc.

New paradigms are needed for oncology drug development in the age of molecularly targeted therapies. One strategic approach to early drug development and proof of concept studies is the concept of the Pharmacological Audit Trail (PhAT), first proposed by Workman and colleagues. The PhAT is a series of key questions queried in sequence regarding the pharmacological behavior of an experimental agent in development. It encompasses evaluation of target expression, pharmacokinetics, pharmacodynamics, target engagement, pathway modulation, biological effects and, ultimately, clinical activity in early clinical trials. This strategy helps to promote scientific evaluation and rational decision making in early oncology drug development, leading up to the demonstration of proof of concept. A thorough discussion of the PhAT and its application to the development of targeted oncological therapeutics will be described.

5:00 Improving Candidate Survival to PoC

John Arrowsmith, Ph.D., Science Director, CMR International

5:30 Networking Cocktail Reception, Exhibit and Poster ViewingSponsored by
Parexel NEW






6:30 Close of Day



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