WEDNESDAY, OCTOBER 6, 2010
7:30am Morning Coffee or Sponsored Breakfast Presentation (Sponsorship Opportunity Available)
8:30 Chairperson’s Remarks
Jeffrey W. Miller, M.D., Director, Exploratory Medicine, Eli Lilly & Company
8:40 Can QbD Accelerate Proof-of-Concept?
Susan Dana Jones, Ph.D., Vice President, Senior Consultant, BioProcess Technology Consultants
QbD (quality by design) principles have been used with success in many industries for several decades. Its business benefits and increased emphasis from regulatory authorities creates a dual motive for implementation. However, its role and benefit for accelerating proof-of-concept needs to be better understood. This presentation will add more clarity around how the concept applies to proof-of-concept studies and steps organizations can take to benefit from QbD.
9:10 Strategic Operations: Accelerating POC - Experience from a Large Pharma Virtual Organization
Coreen Oei, Ph.D., Vice President, Scientific & Clinical Operations, GlaxoSmithKline
Drug development is a costly, risky and lengthy process and is not sustainable in the current situation of economic and development challenges. The industry is looking for “new paradigms” for drug development. In response to this, GSK has set up a Virtual Proof of Concept (VPoC) unit within the drug discovery organization whose aim is to deliver positive proof of concept (PoC) and “Commit to Medicine” decisions for substantially lower cost than is currently expended in the large pharma model.
Sponsored by
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9:40 Using Mechanism-of-Action Repositioning to Accelerate Entry to PoC Studies
Aris Persidis, Ph.D., President, Biovista
Using MoA, it is possible to map all 95,000 pharmacologically active drugs and compounds in the literature against all 29,000 clinical outcomes tracked by medicine. This is a very efficient way to accelerate programs towards PoC, by repositioning drugs in new indications based on a systematic and rational matching of MoA's of drugs, diseases and adverse events. Case studies will be presented in MS, epilepsy, and patient cohort stratification.
10:10 Networking Refreshment Break and Exhibit and Poster Viewing
10:40 Project Planning Methods to Decrease Cycle Time Across Multi-disciplinary Teams
Russell Linderman, Ph.D., Executive Director, Research Science & Technology PGRD, Pfizer Inc.
Project teams are faced with many parallel work-streams that must advance a program at risk or encounter the potential for delays associated with sequential operational efforts. Technical project risks are often viewed independently and teams frequently develop options without consideration of the impact on associated lines. We have developed a tool that provides a comprehensive set of options for project teams to discuss, enabling teams to evaluate the most appropriate path forward while mitigating timeline risks to FIH and POC. The approach provides decision logic trees to integrate API availability and toxicology strategies, evaluate the potential for exploratory approaches, and sample plans for consideration. Early results indicate a positive impact on cycle time reduction in early development.
11:10 EXPERT PANEL: Clinical Operations & Business Models
Moderator: Terri Roberson, MT (ASCP), M.B.A., Sr. Director, Operations, Global External Research and Development, Lilly Research Laboratories, Eli Lilly and Company
Panel members will discuss current operational challenges, including outsourcing and coordination of projects and personnel, that affect “go” and “no go” decision making throughout clinical development.
Panel Members:
Kenneth A. Savin, Ph.D., COO, The Finishing School, Advisor to Special Projects, Global External Research and Development, Eli Lilly and Co.
Coreen Oei, Ph.D., Vice President, Scientific & Clinical Operations,
GlaxoSmithKline
Laura Vessey, Associate Director, Global Trial Management (GTM), Early Stage Development, Global Clinical Trial Operations (GCTO), Merck Research Laboratories
11:40 Sponsored Presentation (Opportunity Available)
12:10pm Luncheon Presentation (Sponsorship Opportunity Available) or
Lunch on Your Own
1:25 Chairperson’s Remarks
John Arrowsmith, Ph.D., Science Director, CMR International
1:30 Development of a Novel Therapeutic for ST Elevation Myocardial Infarction
Robert Daly, Ph.D., Executive Director, Clinical Development, KAI Pharmaceuticals
KAI Pharmaceuticals is an emerging biopharmaceutical company developing peptide therapeutics for significant unmet medical needs. KAI’s lead program, KAI-9803, is a peptide inhibitor of the delta isoform of protein kinase C that demonstrated promising results in a variety of animal models of ischemia and reperfusion. This presentation will describe the first in-man study, DELTA MI, which provided proof-of-concept for this product in subjects with ST elevation myocardial infarction.
2:00 Opportunities and Challenges of Accelerated Proof-of-Concept Studies in Neurodegenerative Diseases
Thierry Sornasse, Ph.D., Director, Translational Medicine, Biomarker Integration, Elan Pharmaceuticals, Inc.
The concept of accelerated proof-of-concept studies is ultimately centered on the conduct of focused and efficient (shorter and/or fewer patients) phase 2 clinical studies that do not solely rely in established clinical end points. This is only made possible by recruiting the right patients (disease sub-type, disease stage, and risk factor), and developing relevant biomarkers of drug activity (target engagement, pharmacodynamic, toxicodynamic). While pre-competitive biomarker initiatives in the field of neurodegenerative diseases constitute a major opportunity to develop sophisticated patient selection criteria, the development of sensitive and relevant biomarker of drug activity in the central nervous system remains a significant challenge.
2:30 Networking Refreshment Break with Exhibit and Poster Viewing
3:00 If I Were a VP…
Cong Chen, Ph.D., Director, Biostatistics, Late-stage Oncology, Merck Research Laboratories
In late-stage oncology drug development, drug developers have to make two critical Go-No Go decisions. The first one is whether to proceed to the definitive Phase III investigation after a Phase II proof-of-concept (POC) trial. The second one is whether to stop a Phase III confirmatory trial for futility after an interim analysis of the data. In practice, the two decisions are heuristically made with limited statistical input, usually amounting to statistical characterization of proposed options. We propose to find the optimal decisions by explicitly maximizing a benefit-cost ratio function, which is often the implicit objective in an otherwise qualitative decision-making process. The numerator of the function represents the benefit (proportional to the expected number of truly active drugs identified for Phase III development in the POC setting; proportional to the expected power for successful completion of Phase III in the interim analysis setting), and the denominator represents the expected total late-stage development cost. The method is easy to explain and simple to implement. The optimal design parameters provide a rational starting point for decision makers to consider. The method developed herein is directly applicable to portfolio management in the oncology therapeutic area where cost-effectiveness of a Go-No Go decision is a major concern. It is also applicable to other disease areas with the same concern or to similar decision issues at any stage of drug development.
3:30 Proof-of-Concept in an Academic Setting
Daniel Rader, M.D., Director, Clinical and Translational Research Center, Associate Director, Institute for Translational Medicine and Therapeutics, University of Pennsylvania School of Medicine
This presentation will address the following: development of an orphan drug for a subset of patients with hyperlipidemica, success in a sector where translational capability is on the rise, and the potential of the academic sector in drug development.
4:00 Close of Conference