Tuesday, October 4
7:30am-5:00pm Conference Registration
7:30 - 8:15 Breakfast Presentation (Sponsorship Opportunity Available) or Morning Coffee
8:15-9:30 Roundtable Discussions
Work with your colleagues in a dynamic discussion/working group to create the ideal definition of PoC for the pharmaceutical industry, while addressing the following in a resultant read-out for the group:
Sponsored by9:30-9:35 Chairperson’s Opening Remarks
Graham Anthony, M.B.A., CFO, Biovista
9:35-10:05 PoC: What Concepts? How Much Confidence?
Oranee T. Daniels, Ph.D., Vice President, Clinical Pharmacology, Theravance, Inc.
One of the big debates in early clinical drug development is what the ‘proof-of-concept’ study should look like. What concepts do the study have to prove? Is demonstrating mechanism of action in humans sufficient? Do we need to differentiate our new chemical entity from standard of care or existing therapy? We’ve heard confusing terminology from PoC (proof-of-concept), PoM (proof of mechanism) to PoR (proof of relevance). Is there a one-size fits all answer for every program? The presenter will walk the audience through a proposed systematic approach that considers unique challenges of each program yet follows logical steps to identify potential pitfalls and desired confidence in decision making. Logistics and operational challenges will also be discussed briefly.
10:05-10:35 The Case for Early Failure Studies
Doina Roman, M.D., Senior Medical Director, Translational Medicine Sciences, Takeda Global Research & Development Center, Inc.
Compound failure in Phase III is costly, but lessons can be learned from these studies and applied to the proof-of-concept phase. Identifying mistakes, patterns, and decision-making criteria can be a successful tool in both establishing PoC or assigning a go/no-go decision in earlier phases. Case studies will be presented and discussed.
10:35-11:00 Networking Coffee Break with Exhibit and Poster Viewing
DATA STRATEGY: SPEEDING UP
PHASE II TO SLOW DOWN PHASE III?
11:00-11:30 Soluble Ligand PK/PD Modeling and Its Application to the Development of a Monoclonal Antibody
Thomas A. Puchalski, Pharm.D., Director, Oncology PK/PD , Pharmacokinetic and Pharmacometrics Group, Biologics Clinical Pharmacology, Centocor R&D, a division of Johnson & Johnson Pharmaceutical Research & Development, LLC
Over the past decade, regulatory agencies have emphasized the importance of integrating pharmacokinetic and pharmacodynamic (PK/PD) data. The concept is also known as model-based drug development, involves building mathematical models to characterize the relationship between drug concentration (PK) and drug effect (PD). Recent literature has shown this can streamline drug development. I will describe the application of PK/PD modeling to an antibody targeting a soluble target. The mechanistic PK/PD model simultaneously described free target, total target and antibody serum concentration data. I will also discuss how the modeling and simulation results were used for dose selection.
11:30-12:00pm Acceleration in PoC, Speed or Confidence?
Xiaoyin (Frank) Fan, M.S., Ph.D., Director, Biometrics, Vertex Pharmaceuticals
Proof-of-Concept (PoC) studies are often underpowered because of the lack of sufficient prior clinical efficacy information, limited trial size, and time pressure, unless the treatment effect is unexpectedly overwhelming. Thus the Go/No-Go decision based on PoC results often depends not just on a single hypothesis being tested but takes a more wholistic assessment of the trial data. These include assessment of efficacy, safety, pharmacometrics, and biomarker data if available. It is important to recognize that the primary goal of a PoC study is to move the drug candidate into late phase development with both speed and reasonable confidence. Speeding up PoC only makes sense if it can accelerate the entire drug development program and improve the probability of success (PoS). We will use some real PoC examples as illustrations in this presentation.
Sponsored by12:00-12:30 How Systematic Indication Expansion can Accelerate Proof of Concept
Graham Anthony, M.B.A., CFO, Biovista
Methods and technologies that can reduce the time it takes for an advanced drug candidate to enter the clinic and provide an efficacy signal are widely needed. Using mechanism of action to systematically explore potential efficacy/risk scenarios for a drug helps accelerate development towards Phase IIa and proof of concept/mechanism trials. Biovista's Clinical Outcome Search Space Technology maps all drugs against all 23,000 indications and 6,000 adverse events tracked by medicine and is used to accelerate proof of concept. Case studies will be discussed on MS, epilepsy and oncology.
12:30-2:00 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch On Your Own
2:00-2:05 Chairperson’s Remarks
Terri Roberson, Senior Director, Global External Research and Development, Lilly Research Laboratories
2:05-2:35 Pharmacometrics and PoC: Preventative Care for Drug Development
Thaddeus Grasela, Pharm.D., Ph.D., Adjunct Professor, University at Buffalo
The synthesis of data and experience is a critical function of Pharma R&D teams. The synthesis process generally consists in the compilation of relevant study results by separate functional areas. Cross-functional, interdisciplinary analysis is often minimal or lacking. This lack of cross-functional synthesis has two important consequences: 1) unintended or unrecognized knowledge gaps and 2) research plans that rely on intuition rather than an explicit synthesis of knowledge. This presentation will describe a strategy for using pharmacometric methods to perform a comprehensive and interdisciplinary synthesis of available data that can play a central role in R&D planning and in the design, analysis, and interpretation of study results.
2:35-3:05 The Utility of Decision Analysis in Early Clinical Development
Eyas Abu-Raddad, Ph.D., Research Advisor, PK/PD Chorus, Eli Lilly & Co
Sponsored by3:05-3:35 Evaluating Clinical Research Sites, Let’s Get Scientific about Data Quality
Neil K. Singla, M.D., CEO, Lotus Clinical Research, LLC
When speaking about data quality, most sponsors focus on data cleanliness and not data accuracy. Data cleanliness has to do with appropriate recording and transcription of data with the avoidance of queries. Data accuracy is much more important! Did the site reveal the treatment effect that the study was designed to show? By using a simple metric, the standardized effect size, the contribution of individual centers toward or away from the primary endpoint can be evaluated.
3:35-4:20 Networking Refreshment Break with Exhibit and Poster Viewing
4:20-5:20 EXPERT PANEL: How to Carry a Phase II trial design to Phase III?
Moderator: Terri Roberson, Senior Director, Global External Research and Development, Lilly Research Laboratories
• What’s the biggest stumbling block from Phase II to III?
• What’s the ideal Phase III study? How does it differ from the Phase II?
• Phase II to III decision making: who wins? Good science or good business?
5:20-6:20 Networking Happy Hour with Exhibit and Poster Viewing
6:20 End of Day Two
Day 1 | Day 3
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